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Mirum(MIRM) - 2021 Q1 - Earnings Call Transcript
2021-05-09 18:51
Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q1 2021 Earnings Conference Call May 6, 2021 4:30 PM ET Company Participants Ian Clements ??? Chief Financial Officer Chris Peetz ??? President and Chief Executive Officer Peter Radovich ??? Chief Operating Officer Conference Call Participants Mani Foroohar ??? SVB Leerink Paul Jeng ??? Guggenheim Partners Jack Allen ??? Baird Ryan Deschner ??? Raymond James Operator Welcome to the Mirum Pharmaceuticals Q1 Earnings Call. My name is Rees, and I will be your operator ...
Mirum(MIRM) - 2021 Q1 - Quarterly Report
2021-05-06 20:17
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________to ________ Commission File Number: 001-38981 Mirum Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 83-1281555 ( ...
Mirum Pharmaceuticals (MIRM) Investor Presentation - Slideshow
2021-03-18 19:46
Corporate Presentation March 2021 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward-looking statements are subject to known and unknown risks, uncert ...
Mirum(MIRM) - 2020 Q4 - Annual Report
2021-03-09 21:15
[Special Note Regarding Forward-Looking Statements](index=3&type=section&id=SPECIAL%20NOTE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) The report contains forward-looking statements subject to risks and uncertainties that could cause actual results to differ materially from expectations - The report contains forward-looking statements under the Private Securities Litigation Reform Act of 1995, which are subject to various risks and uncertainties that could cause actual results to differ materially from expectations[9](index=9&type=chunk)[11](index=11&type=chunk) - Key areas of forward-looking statements include regulatory approval, commercialization, sales and marketing, clinical trial timing, market size, intellectual property, regulatory developments, supplier performance, competition, personnel, COVID-19 impact, and financing[10](index=10&type=chunk) [Summary of Risks Associated with Our Business](index=5&type=section&id=SUMMARY%20OF%20RISKS%20ASSOCIATED%20WITH%20OUR%20BUSINESS) Investing in the company's common stock involves a high degree of risk, including a limited operating history, significant losses, and dependence on product candidate success - Investing in the company's common stock involves a high degree of risk, including a limited operating history, significant losses, dependence on product candidate success, potential clinical trial delays, extensive regulatory hurdles, and intense competition[15](index=15&type=chunk)[16](index=16&type=chunk) - Other significant risks include the need for substantial additional financing, reliance on third-party intellectual property, and challenges in obtaining and maintaining intellectual property protection[16](index=16&type=chunk) PART I [Item 1. Business](index=6&type=section&id=Item%201.%20Business) Mirum Pharmaceuticals develops and commercializes novel therapies for orphan cholestatic liver diseases with maralixibat and volixibat [Overview](index=6&type=section&id=Overview) Mirum Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing late-stage novel therapies for debilitating orphan cholestatic liver diseases - Mirum Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing late-stage novel therapies for debilitating liver diseases, specifically orphan cholestatic liver diseases[17](index=17&type=chunk) - The company's pipeline includes two clinical-stage product candidates, maralixibat and volixibat, targeting conditions like Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), biliary atresia (BA), primary sclerosing cholangitis (PSC), intrahepatic cholestasis of pregnancy (ICP), and primary biliary cholangitis (PBC)[17](index=17&type=chunk)[18](index=18&type=chunk)[19](index=19&type=chunk) - Maralixibat's NDA for ALGS cholestatic pruritus was completed in January 2021, with a potential launch in H2 2021. An MAA for PFIC2 has been submitted to the EMA. Volixibat's Phase 2b trials for PSC and ICP have initiated, with a PBC trial planned for H2 2021[18](index=18&type=chunk)[19](index=19&type=chunk) [Our Product Pipeline](index=6&type=section&id=Our%20Product%20Pipeline) Mirum's pipeline features maralixibat and volixibat, novel ASBT inhibitors reducing toxic bile acid accumulation - Mirum owns exclusive worldwide rights to its product candidates, maralixibat and volixibat, which are novel, oral, minimally-absorbed ASBT inhibitors designed to reduce systemic bile acid levels[20](index=20&type=chunk)[21](index=21&type=chunk) - ASBT inhibition works by increasing bile acid excretion in feces, thereby lowering toxic bile acid accumulation in the liver and systemically, aiming to improve long-term outcomes and symptoms[21](index=21&type=chunk) [Maralixibat](index=7&type=section&id=Maralixibat) Maralixibat, a lead oral ASBT inhibitor, has an extensive safety profile and holds Breakthrough Therapy and Orphan Drug Designations - Maralixibat is a lead product candidate, an oral ASBT inhibitor, with an extensive safety dataset from over **1,600 human subjects** and over **120 children** treated in ALGS and PFIC studies[21](index=21&type=chunk) - It has received Breakthrough Therapy Designation for pruritus associated with ALGS and for PFIC2, as well as Orphan Drug Designation for ALGS, PFIC, and BA[21](index=21&type=chunk)[23](index=23&type=chunk)[27](index=27&type=chunk)[28](index=28&type=chunk) [Maralixibat in ALGS](index=7&type=section&id=Maralixibat%20in%20ALGS) Maralixibat for Alagille syndrome (ALGS) shows significant pruritus and sBA reductions, with an NDA submitted for approval - ALGS affects **1 in 30,000 births** in the US and Europe, with an estimated **2,000-2,500 eligible children** in the US. There are no approved therapies, and liver transplant is often the only definitive treatment[22](index=22&type=chunk) - The Phase 2b ICONIC trial showed statistically significant reductions in pruritus (ItchRO(Obs) score, **p<0.0001**), serum bile acids (sBA, **36% reduction**, **p<0.01**), and xanthomas (**44% reduction**, **p<0.01**) at week 48, with improved quality of life[23](index=23&type=chunk) - An NDA for cholestatic pruritus in ALGS patients was completed in January 2021, with a potential FDA review decision and commercial launch in H2 2021[25](index=25&type=chunk) [Maralixibat in PFIC](index=8&type=section&id=Maralixibat%20in%20PFIC) Maralixibat for Progressive Familial Intrahepatic Cholestasis (PFIC) has an MAA submitted to EMA, with a Phase 3 trial ongoing - PFIC affects **1 in 50,000 to 100,000 births** in the US and Europe, with no approved therapies. Surgical options like PEBD and liver transplantation are available but have significant complications[26](index=26&type=chunk) - An MAA for maralixibat in PFIC2 has been submitted to the EMA, based on five-year results from the Phase 2 INDIGO trial, showing improved transplant-free survival, pruritus, and growth in responders[27](index=27&type=chunk) - The Phase 3 MARCH clinical trial is ongoing, evaluating higher doses of maralixibat in a broader PFIC population, with enrollment expected to complete in Q2 2021 and topline data in early 2022[27](index=27&type=chunk) [Maralixibat in BA](index=8&type=section&id=Maralixibat%20in%20BA) Maralixibat is being evaluated in a Phase 2b trial for Biliary Atresia (BA), a rare infant liver disorder - BA is a rare liver disorder in infants, affecting **1 in 10,000 to 15,000 live births** in the US, and is the most common reason for liver transplantation in children[28](index=28&type=chunk) - The standard Kasai procedure has a success rate of **30-40%**, with remaining patients at risk of progressive liver disease requiring transplant. ASBT inhibition is believed to have therapeutic potential by reducing sBA[28](index=28&type=chunk) - Recruitment for the EMBARK Phase 2b clinical trial of maralixibat in BA began in Q1 2021, and maralixibat has Orphan Drug Designation for BA[28](index=28&type=chunk) [Volixibat](index=8&type=section&id=Volixibat) Volixibat, an oral ASBT inhibitor, is being developed for adult cholestatic liver diseases, with Phase 2b trials initiated - Volixibat is a novel, oral, minimally-absorbed ASBT inhibitor being developed for adult cholestatic liver diseases, including ICP, PSC, and PBC[29](index=29&type=chunk) - Clinical trials in over **400 adults** have shown significant activity on ASBT and bile acid markers, demonstrating potent biological activity[29](index=29&type=chunk) - The OHANA Phase 2b trial for ICP and VISTAS Phase 2b trial for PSC initiated in Q1 2021, with a PBC trial planned for H2 2021. There are no approved therapies for PSC or ICP in the US[19](index=19&type=chunk)[31](index=31&type=chunk)[32](index=32&type=chunk)[34](index=34&type=chunk) [Our History](index=9&type=section&id=Our%20History) Mirum Pharmaceuticals was incorporated in May 2018, began operations in November 2018, and completed its IPO in July 2019 - Mirum Pharmaceuticals was incorporated in Delaware in May 2018 and began operations in November 2018, completing its IPO in July 2019[35](index=35&type=chunk) - The management team has significant experience in developing and commercializing liver and orphan disease therapies[35](index=35&type=chunk) [Our Strategy](index=9&type=section&id=Our%20Strategy) Mirum's strategy focuses on leading liver disease treatment by advancing maralixibat and volixibat, and expanding its pipeline - Mirum's strategy is to be a leader in liver disease treatment by advancing maralixibat for ALGS, PFIC, and BA, and volixibat for PSC, ICP, and PBC[36](index=36&type=chunk) - The company plans to commercialize approved products in North America and Europe with a targeted specialty sales force, and seek strategic collaborations for other territories[36](index=36&type=chunk)[40](index=40&type=chunk) - Mirum also aims to actively manage its product portfolio and expand its pipeline by identifying, acquiring, in-licensing, and advancing additional product candidates for liver diseases[40](index=40&type=chunk) [Overview of Cholestatic Liver Diseases](index=10&type=section&id=Overview%20of%20Cholestatic%20Liver%20Diseases) Cholestatic liver diseases involve impaired bile flow, leading to toxic bile acid accumulation, severe pruritus, and liver injury - Cholestatic liver diseases involve impaired bile flow, leading to accumulation of toxic bile acids in the liver and systemically, causing severe pruritus, liver injury, and potentially liver failure[38](index=38&type=chunk) - ASBT is responsible for recycling **95%** of bile acids from the intestine to the liver; inhibiting it leads to increased fecal excretion and lower systemic bile acid levels[37](index=37&type=chunk) - Symptoms include jaundice, pruritus, progressive liver disease, and growth delay, significantly impacting patient quality of life and caregiver well-being[38](index=38&type=chunk) [Alagille Syndrome](index=10&type=section&id=Alagille%20Syndrome) Alagille Syndrome (ALGS) is a rare genetic disorder causing malformed bile ducts, severe pruritus, and progressive liver disease - ALGS is a rare genetic disorder (**1 in 30,000 births** in US/Europe) characterized by malformed bile ducts, leading to bile accumulation and progressive liver disease[39](index=39&type=chunk) - The addressable patient population in the US is estimated at **2,000-2,500 pediatric ALGS patients**, with severe pruritus being a major symptom and driver for liver transplantation[39](index=39&type=chunk)[40](index=40&type=chunk) - Current options, PEBD and liver transplantation, are invasive and have significant complications, highlighting a substantial unmet medical need[41](index=41&type=chunk) [Progressive Familial Intrahepatic Cholestasis](index=11&type=section&id=Progressive%20Familial%20Intrahepatic%20Cholestasis) Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare genetic disorder causing cholestasis and progressive liver disease - PFIC is a rare genetic disorder (**1 in 50,000 to 100,000 births** in US/Europe) causing cholestasis and progressive liver disease, often leading to liver failure in infancy[42](index=42&type=chunk)[44](index=44&type=chunk) - Mirum's initial focus is on nt-PFIC2 patients (**50% of PFIC population**) who have residual BSEP function, making them potential responders to ASBT inhibition[43](index=43&type=chunk)[45](index=45&type=chunk) - Severe pruritus is a prominent symptom, and current treatments (PEBD, liver transplant) are often the only options, indicating a high unmet medical need[44](index=44&type=chunk)[45](index=45&type=chunk) [Biliary Atresia](index=11&type=section&id=Biliary%20Atresia) Biliary Atresia (BA) is a rare infant liver disorder, the most common reason for pediatric liver transplantation - BA is a rare infant liver disorder (**1 in 10,000 to 15,000 live births** in the US) involving blocked or absent bile ducts, leading to bile accumulation and liver damage, and is the most common reason for pediatric liver transplantation[47](index=47&type=chunk) - The Kasai procedure is the standard surgical treatment, but its success rate is only **30-40%**, with many patients still requiring liver transplantation, often before two years of age[49](index=49&type=chunk) - Elevated serum bile acids (sBA) and bilirubin levels post-Kasai procedure are associated with a higher risk of progression to transplant, supporting the potential of ASBT inhibition[49](index=49&type=chunk) [Intrahepatic Cholestasis of Pregnancy](index=12&type=section&id=Intrahepatic%20Cholestasis%20of%20Pregnancy) Intrahepatic Cholestasis of Pregnancy (ICP) increases perinatal risks and causes severe pruritus, with no FDA-approved therapy - ICP is a liver disorder in pregnant women (estimated **40,000 cases** in US, **100,000 in Europe** annually) that increases risks of stillbirth, preterm labor, and neonatal complications, and is associated with severe pruritus[50](index=50&type=chunk)[51](index=51&type=chunk) - Elevated sBA levels in ICP correlate with worsened perinatal risks. There is no FDA-approved therapy in the US, and off-label UDCA offers minimal improvement[51](index=51&type=chunk)[52](index=52&type=chunk) - ASBT inhibition is believed to offer therapeutic potential for ICP patients due to its impact on sBA levels[52](index=52&type=chunk) [Primary Sclerosing Cholangitis](index=12&type=section&id=Primary%20Sclerosing%20Cholangitis) Primary Sclerosing Cholangitis (PSC) is a rare, chronic cholestatic liver disease leading to liver failure and debilitating pruritus - PSC is a rare, chronic cholestatic liver disease (estimated **29,000 in US**, **50,000 in Europe**) characterized by progressive inflammation and destruction of bile ducts, leading to liver failure[53](index=53&type=chunk) - Up to **70%** of PSC patients suffer from debilitating pruritus. Liver transplantation is the only treatment shown to improve clinical outcomes, but recurrence rates post-transplant can be as high as **25%**[55](index=55&type=chunk)[56](index=56&type=chunk)[57](index=57&type=chunk) - There is an acute need for novel therapies, as off-label UDCA does not improve pruritus. ASBT inhibition is believed to address symptoms and potentially delay liver transplantation[56](index=56&type=chunk)[57](index=57&type=chunk) [Primary Biliary Cholangitis](index=13&type=section&id=Primary%20Biliary%20Cholangitis) Primary Biliary Cholangitis (PBC) is a chronic, immune-mediated cholestatic liver disease causing severe pruritus and liver damage - PBC is a chronic, rare cholestatic liver disease caused by immune-mediated destruction of intrahepatic bile ducts, leading to increased hepatic bile acid concentrations and progressive liver damage[58](index=58&type=chunk) - PBC disproportionately affects women (**10:1 ratio**) and is typically diagnosed between **40-60 years of age**. Up to **70%** of patients experience severe pruritus, significantly impacting quality of life[59](index=59&type=chunk)[60](index=60&type=chunk) - Existing therapies for PBC-associated pruritus are temporary or suboptimal, highlighting an unmet need for effective treatments[61](index=61&type=chunk) [Our Solutions](index=14&type=section&id=Our%20Solutions) Mirum's solutions for cholestatic liver diseases focus on ASBT inhibition to increase bile acid excretion and relieve symptoms - Mirum's solutions for cholestatic liver diseases focus on inhibiting ASBT, which increases bile acid excretion and reduces systemic bile acid levels, leading to improved liver function and symptom relief[62](index=62&type=chunk) [Overview of Maralixibat](index=14&type=section&id=Overview%20of%20Maralixibat) Maralixibat, an oral ASBT inhibitor, shows significant efficacy in ALGS and PFIC, with NDA/MAA submissions underway - Maralixibat is a lead product candidate, an oral, minimally-absorbed ASBT inhibitor with an extensive safety dataset from over **1,600 human subjects**[63](index=63&type=chunk) - In ALGS, Phase 2b ICONIC trial showed statistically significant reductions in pruritus and sBA, leading to an NDA submission for cholestatic pruritus in ALGS patients[64](index=64&type=chunk) - In PFIC, Phase 2 INDIGO trial showed multi-parameter response in nt-PFIC2 patients, forming the basis for EMA MAA submission. Phase 3 MARCH trial is ongoing for broader PFIC subtypes[65](index=65&type=chunk) [Historical Clinical Development of Maralixibat](index=15&type=section&id=Historical%20Clinical%20Development%20of%20Maralixibat) This section details maralixibat's clinical development, including ALGS, PFIC, and BA trials, highlighting efficacy and safety data [Our Clinical Trials of Maralixibat in ALGS](index=15&type=section&id=Our%20Clinical%20Trials%20of%20Maralixibat%20in%20ALGS) Maralixibat's Phase 2b ICONIC trial in ALGS demonstrated statistically significant reductions in pruritus and sBA - The Phase 2b ICONIC trial in ALGS patients met its primary endpoint, showing a statistically significant difference in sBA reduction between treatment and placebo groups (**p<0.05**) during randomized withdrawal[67](index=67&type=chunk)[68](index=68&type=chunk) - Patients treated with maralixibat in ICONIC showed approximately **60% mean reduction** in pruritus (ItchRO(Obs) score, **p<0.0001**) and **31% mean reduction** in sBA levels at week 18, with sustained improvements through week 204[71](index=71&type=chunk)[73](index=73&type=chunk)[74](index=74&type=chunk) - ICONIC trial also demonstrated significant improvements in xanthomas (**44% reduction** at week 48, **p<0.01**) and a clinically meaningful acceleration in height growth (**p≤0.01**) with long-term treatment[79](index=79&type=chunk)[80](index=80&type=chunk) [Additional Phase 2 Trials in ALGS](index=19&type=section&id=Additional%20Phase%202%20Trials%20in%20ALGS) Additional Phase 2 trials (ITCH and IMAGO) in ALGS evaluated lower maralixibat doses, showing less pronounced pruritus reduction - Two additional Phase 2 trials (ITCH and IMAGO) in ALGS evaluated lower doses of maralixibat, which showed less pronounced pruritus reduction compared to the ICONIC trial's higher dose[83](index=83&type=chunk) Pruritus Results from Phase 2 ITCH and IMAGO Clinical Trials in ALGS (Week 13) | | ITCH MRX | ITCH PBO | IMAGO MRX | IMAGO PBO | | :--- | :--- | :--- | :--- | :--- | | Change from Baseline ItchRO(Obs) | -1.19 | -0.58 | -0.61 | -0.59 | | % Change from Baseline ItchRO(Obs) | -39% | -21% | -22% | -19% | - Long-term open-label extensions of ITCH (IMAGINE II) and IMAGO (IMAGINE I) showed sustained pruritus relief despite initial non-significant differences from placebo at week 13[84](index=84&type=chunk) [Registrational Path for Maralixibat in ALGS](index=19&type=section&id=Registrational%20Path%20for%20Maralixibat%20in%20ALGS) Mirum completed its NDA submission for maralixibat in ALGS, benefiting from Breakthrough Therapy and Rare Pediatric Disease Designations - Mirum completed its NDA submission for maralixibat for cholestatic pruritus in ALGS patients in January 2021, following Breakthrough Therapy Designation and Rare Pediatric Disease Designation[85](index=85&type=chunk) - The Rare Pediatric Disease Designation may qualify the company for a priority review voucher if the NDA is approved[85](index=85&type=chunk) - Discussions with the EMA regarding submission plans for ALGS are pending further analysis of long-term extension treatment data compared to natural history data[86](index=86&type=chunk) [Our Clinical Trials of Maralixibat in PFIC](index=19&type=section&id=Our%20Clinical%20Trials%20of%20Maralixibat%20in%20PFIC) The Phase 2 INDIGO trial in PFIC2 patients showed profound sBA reductions and pruritus improvements, leading to Breakthrough Therapy Designation - The Phase 2 INDIGO trial in PFIC2 patients showed profound and durable reductions in sBA and significant improvements in pruritus, particularly in nt-PFIC2 patients with residual BSEP function[87](index=87&type=chunk)[91](index=91&type=chunk) - INDIGO data indicated that **100%** of maralixibat patients who achieved the NAPPED sBA response threshold remained transplant-free, demonstrating a clear pattern of response leading to long-term transplant-free survival[95](index=95&type=chunk) - The observed responses in PFIC2 patients led to Breakthrough Therapy Designation for maralixibat in this indication[91](index=91&type=chunk) [Registrational Program for Maralixibat in PFIC](index=21&type=section&id=Registrational%20Program%20for%20Maralixibat%20in%20PFIC) Mirum submitted an MAA to the EMA for maralixibat in PFIC2, with the Phase 3 MARCH trial ongoing to support a future FDA NDA - Mirum submitted an MAA to the EMA for maralixibat for PFIC2 treatment in Q4 2020, based on INDIGO trial results and natural history comparisons, which is currently under review[96](index=96&type=chunk) - The Phase 3 MARCH clinical trial is a randomized, placebo-controlled study evaluating higher doses of maralixibat (up to **570 µg/kg BID**) in up to **30 nt-PFIC2 patients** (aged **1-17 years**), with a primary endpoint of pruritus reduction[97](index=97&type=chunk)[98](index=98&type=chunk) - Topline data from the Phase 3 MARCH trial is expected in early 2022, and if positive, will support a planned NDA submission to the FDA for maralixibat in PFIC[98](index=98&type=chunk) [Safety and Tolerability Data for Maralixibat](index=22&type=section&id=Safety%20and%20Tolerability%20Data%20for%20Maralixibat) Maralixibat's clinical development program showed mild to moderate GI adverse reactions, with no serious drug-induced liver injury - In the ALGS clinical development program (**86 patients**, up to **760 µg/kg/day**), the most common adverse reactions were mild to moderate diarrhea and abdominal pain, which resolved with continued treatment[99](index=99&type=chunk) - Independent safety reviews concluded no serious concern for drug-induced liver injury, though isolated, asymptomatic, and reversible ALT elevations were observed in some ALGS participants, likely disease-related[101](index=101&type=chunk) - No cases of drug-induced liver injury or liver-related morbidity/mortality have been reported in over **1600 adult and pediatric study participants**[101](index=101&type=chunk) [Maralixibat in BA](index=23&type=section&id=Maralixibat%20in%20BA) Maralixibat is being evaluated in the Phase 2 EMBARK trial for Biliary Atresia (BA) infants, supported by preclinical data - Preclinical data in bile duct ligated rats showed maralixibat treatment significantly reduced sBA and liver transaminases, supporting its therapeutic potential in BA[102](index=102&type=chunk)[103](index=103&type=chunk) - Clinical observations indicate that lower bilirubin and sBA levels post-Kasai procedure predict improved transplant-free survival in BA patients[104](index=104&type=chunk)[105](index=105&type=chunk)[107](index=107&type=chunk)[109](index=109&type=chunk) - The Phase 2 EMBARK trial in BA infants (approx. **72 patients** post-Kasai) will evaluate maralixibat's safety and efficacy, with primary endpoint of change in total serum bilirubin and secondary endpoints including sBA reduction and transplant-free survival[110](index=110&type=chunk) [Overview of Volixibat](index=25&type=section&id=Overview%20of%20Volixibat) Volixibat is a novel, oral ASBT inhibitor for adult cholestatic diseases, demonstrating potent biological activity - Volixibat is a novel, oral, minimally-absorbed ASBT inhibitor aimed at treating adult cholestatic diseases by blocking bile acid recycling, thereby reducing systemic and liver bile acid levels[111](index=111&type=chunk) - Phase 1 and Phase 2 clinical trials demonstrated volixibat's potent biological activity through decreases in LDL cholesterol and increases in 7αC4 and fecal bile acid content[111](index=111&type=chunk) [Development of Volixibat](index=25&type=section&id=Development%20of%20Volixibat) Volixibat has been evaluated in over 400 adults, showing general tolerability and dose-dependent ASBT inhibition - Volixibat has been evaluated in over **400 adults**, showing general tolerability with common mild to moderate GI events in Phase 1 trials[112](index=112&type=chunk) - A Phase 1 dose-ranging study in healthy adults confirmed dose-dependent increases in fecal bile acid excretion and bile acid synthesis (7αC4), with **20 mg** and **80 mg BID doses** selected for registrational programs[114](index=114&type=chunk) - Previous development for NASH was discontinued by Shire plc, but results indicated ASBT inhibition, leading Mirum to pursue development for adult cholestatic liver diseases[113](index=113&type=chunk) [On-going and Future Clinical Development of Volixibat](index=26&type=section&id=On-going%20and%20Future%20Clinical%20Development%20of%20Volixibat) This section outlines the ongoing and planned clinical development of volixibat for ICP, PSC, and PBC, including Phase 2a/2b trials [Phase 2a/2b OHANA Trial in ICP](index=26&type=section&id=Phase%202a%2F2b%20OHANA%20Trial%20in%20ICP) The OHANA Phase 2a/2b trial is evaluating volixibat in ICP patients, assessing sBA reduction and pruritus improvements - The OHANA Phase 2a/2b clinical trial is evaluating two doses of volixibat (**80 mg BID or 20 mg BID**) versus placebo in approximately **260 ICP patients** with elevated sBA levels[117](index=117&type=chunk)[118](index=118&type=chunk) - The primary endpoint is the mean change from baseline in total sBA concentration, with secondary endpoints assessing pruritus improvements, perinatal outcomes, safety, and tolerability[119](index=119&type=chunk) [Phase 2b VISTAS Trial in PSC](index=27&type=section&id=Phase%202b%20VISTAS%20Trial%20in%20PSC) The VISTAS Phase 2b trial is evaluating volixibat in PSC patients with pruritus, including an adaptive interim analysis - The VISTAS Phase 2b clinical trial is evaluating volixibat in approximately **120 PSC patients** with pruritus, including an adaptive interim analysis[120](index=120&type=chunk) - The confirmatory phase will compare a single dose of volixibat to placebo, with patients eligible for an open-label extension to assess durability of response and changes in fibrosis markers[120](index=120&type=chunk) [Phase 2b Clinical Trial in PBC](index=28&type=section&id=Phase%202b%20Clinical%20Trial%20in%20PBC) Mirum plans to initiate a Phase 2b clinical trial of volixibat in PBC in H2 2021, focusing on treating cholestatic pruritus - Mirum plans to initiate a Phase 2b clinical trial of volixibat in PBC in the second half of 2021, focusing on treating cholestatic pruritus with elevated or normal alkaline phosphatase[122](index=122&type=chunk) [License, Finance and Royalty Agreements](index=28&type=section&id=License,%20Finance%20and%20Royalty%20Agreements) This section details Mirum's key license, finance, and royalty agreements with various partners, outlining payment obligations [Assignment and License Agreement with Shire International GmbH (Takeda)](index=28&type=section&id=Assignment%20and%20License%20Agreement%20with%20Shire%20International%20GmbH%20(Takeda)) Mirum acquired exclusive worldwide rights to maralixibat and volixibat from Shire (Takeda), involving upfront payments, milestones, and royalties - In November 2018, Mirum acquired exclusive worldwide rights to maralixibat and volixibat from Shire (now Takeda) through an assignment and license agreement, including rights to underlying Pfizer, Satiogen, and Sanofi agreements[123](index=123&type=chunk)[124](index=124&type=chunk) - Mirum made an upfront payment of **$7.5 million** and issued **1,859,151 shares** of common stock to Shire. Milestone payments include **$2.5 million** for Phase 3 MARCH trial initiation (July 2019), **$10.0 million** for PFIC2 MAA acceptance (Dec 2020), and **$2.0 million** for volixibat development milestone (Jan 2021)[125](index=125&type=chunk)[127](index=127&type=chunk) - Future obligations include up to **$107.0 million** for maralixibat clinical/regulatory milestones (ALGS, PFIC, BA), **$25.0 million** for each other maralixibat indication, **$30.0 million** for volixibat's first indication, tiered sales milestones up to **$30.0 million**, and tiered royalties (low double-digits to mid-teens) on worldwide net sales, reducible by Sanofi/Satiogen royalties[126](index=126&type=chunk)[128](index=128&type=chunk)[129](index=129&type=chunk) [License Agreement with Pfizer Inc.](index=29&type=section&id=License%20Agreement%20with%20Pfizer%20Inc.) Mirum obtained an exclusive worldwide license to Pfizer's know-how for maralixibat, with obligations for low single-digit royalties - Through the Shire License Agreement, Mirum obtained an exclusive, worldwide license to Pfizer's know-how related to maralixibat for development, manufacture, and commercialization[132](index=132&type=chunk) - Mirum is obligated to pay Pfizer a low single-digit royalty on net sales of products utilizing the Pfizer Know-How, continuing until the eighth anniversary of the first commercial sale[135](index=135&type=chunk) - Mirum has the right to unilaterally terminate the agreement with **90 days' notice**. Pfizer retains rights for internal research[133](index=133&type=chunk)[137](index=137&type=chunk) [License Agreement with Sanofi-Aventis Deutschland GmbH](index=30&type=section&id=License%20Agreement%20with%20Sanofi-Aventis%20Deutschland%20GmbH) Mirum obtained an exclusive worldwide license from Sanofi for volixibat patents and know-how, with milestone and tiered royalty payments - Mirum obtained an exclusive, worldwide license from Sanofi for patents and know-how related to volixibat, with rights to develop, commercialize, and manufacture[138](index=138&type=chunk) - Mirum exercised an option in May 2020 to manufacture volixibat and is transferring manufacturing to a third-party contract manufacturer[138](index=138&type=chunk) - Payment obligations include up to **$36.0 million** in regulatory, commercialization, and sales milestones, tiered royalties (mid to high single-digit) on net sales, and a percentage of sublicense fees (mid-teens to low-thirties)[141](index=141&type=chunk) [License Agreement with Satiogen Pharmaceuticals, Inc.](index=31&type=section&id=License%20Agreement%20with%20Satiogen%20Pharmaceuticals,%20Inc.) Mirum obtained an exclusive worldwide license from Satiogen for ASBTi and TGR5 Technology, with milestone and low single-digit royalties - Mirum obtained an exclusive, worldwide license from Satiogen for ASBTi Technology and TGR5 Technology for human diseases (excluding diabetes/obesity), with sublicensing rights[145](index=145&type=chunk) - Payment obligations include up to **$10.5 million** in milestones (**$0.5 million** development, **$5.0 million** regulatory, **$5.0 million** commercialization) and a low single-digit royalty on net sales, creditable against Shire royalties[148](index=148&type=chunk) - Mirum paid a **$0.5 million** development milestone in July 2019 for the initiation of the Phase 3 MARCH clinical trial[148](index=148&type=chunk) [Revenue Interest Purchase Agreement with Oberland](index=32&type=section&id=Revenue%20Interest%20Purchase%20Agreement%20with%20Oberland) Mirum entered a RIPA with Oberland Capital, receiving **$50.0 million** upfront with potential for **$150.0 million** more, for tiered revenue interest - In December 2020, Mirum entered a Revenue Interest Purchase Agreement (RIPA) with Oberland Capital, receiving **$50.0 million** upfront (less costs)[152](index=152&type=chunk) - Mirum may receive up to an additional **$150.0 million** in installments: **$65.0 million** upon FDA acceptance of ALGS NDA, **$35.0 million** upon FDA approval of ALGS, and up to **$50.0 million** at Purchasers' option for in-licenses/acquisitions[152](index=152&type=chunk) - In return, Purchasers receive tiered Revenue Interest Payments (initially **9.75%** at Tier 1, **2.00%** at Tier 2/3) on annual net sales of maralixibat, terminating when payments reach **195.0%** of Cumulative Purchaser Payments[153](index=153&type=chunk) [Intellectual Property](index=32&type=section&id=Intellectual%20Property) Mirum's success relies on proprietary protection for its product candidates through patents, trade secrets, and exclusivity - Mirum's success depends on obtaining and maintaining proprietary protection for its product candidates, including patents, trade secrets, and know-how[154](index=154&type=chunk) - The company holds rights to pending and issued patents in various jurisdictions covering methods of treating cholestasis using ASBTis with limited systemic exposure, expiring in October 2032[154](index=154&type=chunk)[155](index=155&type=chunk) - Maralixibat lacks composition-of-matter patents, relying primarily on method-of-use and dosage form patents. Both maralixibat and volixibat may be eligible for **5 years** of new chemical entity exclusivity and **7 years** of orphan drug exclusivity in the US upon approval[156](index=156&type=chunk)[158](index=158&type=chunk)[159](index=159&type=chunk) [Sales and Marketing](index=34&type=section&id=Sales%20and%20Marketing) Mirum is building a commercial organization and medical affairs team to support maralixibat and volixibat in North America and Europe - Mirum is building a commercial organization to support maralixibat and volixibat in North America and Europe, targeting a small number of specialists who treat pediatric cholestasis[163](index=163&type=chunk)[164](index=164&type=chunk) - The company is also developing a medical affairs organization to provide scientific and medical education to healthcare providers and patients in the rare liver disease community[165](index=165&type=chunk) - In October 2020, Mirum partnered with Eversana for integrated distribution, specialty pharmacy, medical information, and patient services for maralixibat in the US, if approved[166](index=166&type=chunk) [Manufacturing](index=34&type=section&id=Manufacturing) Mirum relies entirely on third-party contract manufacturers for raw materials, API, and finished products, lacking its own facilities - Mirum does not own manufacturing facilities and relies entirely on third-party contract manufacturers for raw materials, active pharmaceutical ingredients (API), and finished products for its product candidates[167](index=167&type=chunk) - The company currently lacks contractual arrangements for commercial supplies and plans to enter into such agreements with third-party suppliers prior to any FDA approval[167](index=167&type=chunk) [Competition](index=34&type=section&id=Competition) The biopharmaceutical industry is highly competitive, with Mirum facing rivals developing ASBT inhibitors and existing off-label treatments - The biopharmaceutical industry is highly competitive, with Mirum facing competition from major pharmaceutical companies, biotechnology firms, academic institutions, and research institutions, many with greater resources[168](index=168&type=chunk)[169](index=169&type=chunk) - There are no FDA-approved therapies for ALGS, PFIC, BA, PSC, or ICP in the US, but symptomatic treatments like cholestyramine and UDCA are used off-label[171](index=171&type=chunk)[172](index=172&type=chunk)[173](index=173&type=chunk) - Competitors like Albireo Pharma, Inc. and GlaxoSmithKline plc are developing ASBT inhibitors for cholestatic liver diseases, with Albireo's odevixibat having an NDA accepted for PFIC[173](index=173&type=chunk) [Government Regulation and Product Approval](index=35&type=section&id=Government%20Regulation%20and%20Product%20Approval) This section outlines extensive US and international government regulations governing drug development, approval, and commercialization [U.S. Drug Development Process](index=36&type=section&id=U.S.%20Drug%20Development%20Process) The US drug development process is extensively regulated by the FDA, requiring substantial time and resources for preclinical and clinical trials - The US drug development process is extensively regulated by the FDA under the FDCA, requiring substantial time and financial resources for preclinical testing, IND submission, and multi-phase clinical trials (Phase 1, 2, 3)[176](index=176&type=chunk)[177](index=177&type=chunk)[178](index=178&type=chunk)[183](index=183&type=chunk) - Clinical trials must comply with Good Laboratory Practice (GLP) and Good Clinical Practice (GCP) regulations, and require IRB approval and ongoing monitoring[177](index=177&type=chunk)[178](index=178&type=chunk)[179](index=179&type=chunk) - Successful completion of clinical trials is necessary to establish safety and efficacy, followed by NDA submission and FDA review for commercial marketing approval[178](index=178&type=chunk) [U.S. Review and Approval Processes](index=38&type=section&id=U.S.%20Review%20and%20Approval%20Processes) The FDA reviews NDAs for safety, effectiveness, and manufacturing compliance, potentially requiring advisory committee review or post-market studies - The FDA reviews NDAs for safety and effectiveness, manufacturing compliance (cGMP), and may require advisory committee review or additional post-market studies (Phase 4)[184](index=184&type=chunk)[187](index=187&type=chunk)[188](index=188&type=chunk)[190](index=190&type=chunk) - The Pediatric Research Equity Act (PREA) generally requires pediatric clinical trials for new drugs, unless a deferral or waiver is granted, or the drug has orphan designation for that indication[185](index=185&type=chunk) - FDA approval may be limited to specific indications or dosages, or require a Risk Evaluation and Mitigation Strategy (REMS) to ensure safe use[190](index=190&type=chunk) [Orphan Drug Designation](index=39&type=section&id=Orphan%20Drug%20Designation) Orphan Drug Designation provides 7 years of market exclusivity for drugs treating rare diseases, along with financial incentives - Orphan Drug Designation is granted by the FDA for drugs treating rare diseases (fewer than **200,000 individuals** in the US) and, if first approved, provides **7 years** of market exclusivity[191](index=191&type=chunk)[192](index=192&type=chunk) - Exclusivity can be lost under certain circumstances, such as a showing of clinical superiority by a competitor or if the designation criteria are no longer met[192](index=192&type=chunk) - Orphan designation does not expedite the regulatory review process but offers financial incentives like grant funding, tax advantages, and user-fee waivers[191](index=191&type=chunk)[192](index=192&type=chunk) [Rare Pediatric Disease Priority Review Voucher Program](index=39&type=section&id=Rare%20Pediatric%20Disease%20Priority%20Review%20Voucher%20Program) The FDA awards priority review vouchers for drugs approved for rare pediatric diseases, which are transferable and can expedite subsequent applications - The FDA awards priority review vouchers for drugs approved for 'rare pediatric diseases' (serious/life-threatening conditions primarily affecting individuals aged **0-18 years** and meeting Orphan Drug Act criteria)[193](index=193&type=chunk)[195](index=195&type=chunk) - Vouchers can be redeemed for priority review of a subsequent marketing application for a different product and are transferable[193](index=193&type=chunk) - The program is authorized until September 30, 2024, with eligibility for vouchers if designation is received before this date and approval before September 30, 2026[195](index=195&type=chunk) [Expedited Development and Review Programs](index=40&type=section&id=Expedited%20Development%20and%20Review%20Programs) The FDA offers Fast Track, Priority Review, Accelerated Approval, and Breakthrough Therapy programs to expedite development - The FDA offers Fast Track, Priority Review, and Accelerated Approval programs to expedite development and review for drugs treating serious/life-threatening conditions with unmet medical needs or potential for substantial improvement[196](index=196&type=chunk)[197](index=197&type=chunk)[198](index=198&type=chunk) - Breakthrough Therapy designation, granted to maralixibat, provides intensive FDA interaction and guidance, and includes Fast Track features[199](index=199&type=chunk) - These designations do not change approval standards and do not guarantee faster development, review, or approval, nor do they assure a material commercial advantage[198](index=198&type=chunk)[200](index=200&type=chunk) [Post-Approval Requirements](index=41&type=section&id=Post-Approval%20Requirements) Approved drug products are subject to ongoing FDA regulation, including record-keeping, adverse event reporting, and strict promotion rules - Approved drug products are subject to ongoing FDA regulation, including record-keeping, adverse event reporting, product sampling, distribution, and strict promotion/advertising requirements (e.g., no off-label promotion)[202](index=202&type=chunk) - Manufacturing processes must continuously conform to cGMP requirements, and facilities are subject to periodic FDA inspections[203](index=203&type=chunk) - Discovery of post-approval problems or non-compliance can lead to product restrictions, recalls, withdrawal of approval, fines, and other penalties[203](index=203&type=chunk)[204](index=204&type=chunk) [U.S. Patent Term Restoration and Marketing Exclusivity](index=41&type=section&id=U.S.%20Patent%20Term%20Restoration%20and%20Marketing%20Exclusivity) The Hatch-Waxman Amendments allow for patent term restoration and marketing exclusivity for new chemical entities and clinical investigations - The Hatch-Waxman Amendments allow for up to **5 years** of patent term restoration to compensate for regulatory review time, but cannot extend a patent beyond **14 years** from approval[205](index=205&type=chunk) - New chemical entities receive **5 years** of non-patent marketing exclusivity, preventing FDA approval of generic or 505(b)(2) applications for the same active moiety[206](index=206&type=chunk) - Three years of marketing exclusivity is granted for new clinical investigations essential to approval (e.g., new indications), and pediatric exclusivity can add **6 months** to existing exclusivity periods[206](index=206&type=chunk)[207](index=207&type=chunk) [Other U.S. Healthcare Laws and Compliance Requirements](index=42&type=section&id=Other%20U.S.%20Healthcare%20Laws%20and%20Compliance%20Requirements) The company is subject to federal and state anti-kickback, fraud and abuse, false claims, privacy, and transparency laws - The company is subject to federal and state anti-kickback, fraud and abuse, false claims, privacy and security (HIPAA, HITECH), and Physician Payments Sunshine Act laws[208](index=208&type=chunk)[209](index=209&type=chunk)[213](index=213&type=chunk)[214](index=214&type=chunk)[215](index=215&type=chunk)[216](index=216&type=chunk) - Violations can lead to significant civil, criminal, and administrative penalties, including fines, exclusion from government programs, and reputational harm[176](index=176&type=chunk)[221](index=221&type=chunk) - The California Consumer Privacy Act (CCPA) and GDPR impose stringent requirements on personal data processing, with significant fines for non-compliance and challenges for cross-border data transfers[217](index=217&type=chunk)[218](index=218&type=chunk)[219](index=219&type=chunk) [Pharmaceutical Coverage, Pricing and Reimbursement](index=44&type=section&id=Pharmaceutical%20Coverage,%20Pricing%20and%20Reimbursement) Sales of approved products depend on coverage and adequate reimbursement from third-party payors, a costly and time-consuming process - Sales of approved products depend on coverage and adequate reimbursement from third-party payors (federal, state, private insurers), who increasingly challenge prices and medical necessity[224](index=224&type=chunk) - Obtaining coverage and reimbursement is a costly and time-consuming process, requiring scientific, clinical, and cost-effectiveness data, with no assurance of adequate rates[224](index=224&type=chunk)[227](index=227&type=chunk) - Participation in government programs (Medicaid, VHCA) may require significant discounts and rebates, potentially lowering product prices[225](index=225&type=chunk) [Healthcare Reform](index=45&type=section&id=Healthcare%20Reform) Healthcare reform initiatives, including the ACA and drug pricing regulations, create uncertainty and potential downward pressure on prices - The Affordable Care Act (ACA) and subsequent legislative changes have introduced cost-containment measures, including fees on drug manufacturers, increased Medicaid rebates, and Medicare payment reductions[228](index=228&type=chunk)[230](index=230&type=chunk)[231](index=231&type=chunk)[232](index=232&type=chunk) - Ongoing challenges to the ACA and new executive orders/regulations related to drug pricing (e.g., Most Favored Nation rule) create uncertainty and potential downward pressure on drug prices[230](index=230&type=chunk)[234](index=234&type=chunk) - State-level legislation also aims to control pharmaceutical pricing through price/reimbursement constraints, discounts, and transparency measures, which could harm business and profitability[235](index=235&type=chunk)[236](index=236&type=chunk) [The U.S. Foreign Corrupt Practices Act](index=47&type=section&id=The%20U.S.%20Foreign%20Corrupt%20Practices%20Act) The FCPA prohibits offering or paying anything of value to foreign officials to influence business decisions, requiring accurate accounting - The U.S. Foreign Corrupt Practices Act (FCPA) prohibits offering or paying anything of value to foreign officials to influence business decisions and requires accurate accounting records[237](index=237&type=chunk)[238](index=238&type=chunk) - Violations can lead to criminal liability and serious consequences, including fines, imprisonment, and reputational harm, impacting the ability to compete internationally[108](index=108&type=chunk)[519](index=519&type=chunk) [Europe / Rest of World Government Regulation](index=48&type=section&id=Europe%20%2F%20Rest%20of%20World%20Government%20Regulation) Foreign operations are subject to diverse regulations for clinical trials, product licensing, pricing, and reimbursement, with penalties for non-compliance - Operations in foreign countries are subject to diverse regulations for clinical trials, product licensing, pricing, and reimbursement, similar to but with important differences from US regulations[239](index=239&type=chunk)[240](index=240&type=chunk) - The European Union offers centralized and national authorization procedures for marketing approval, with orphan drug designation providing **10 years** of market exclusivity (extendable to **12 years**)[241](index=241&type=chunk)[242](index=242&type=chunk)[243](index=243&type=chunk)[244](index=244&type=chunk)[245](index=245&type=chunk) - Failure to comply with foreign regulatory requirements can result in fines, withdrawal of approvals, product recalls, and criminal prosecution[247](index=247&type=chunk) [Human Capital Management](index=49&type=section&id=Human%20Capital%20Management) Mirum employs **68 full-time staff**, prioritizing talent attraction, development, and retention through training and diversity initiatives - As of December 31, 2020, Mirum employed **68 full-time employees** (**61 in US**, **7 in Switzerland**), with **17** holding Ph.D. or M.D. degrees, and expects to add more in 2021[248](index=248&type=chunk)[249](index=249&type=chunk) - The company prioritizes attracting, developing, and retaining key personnel through training, performance management, and engagement surveys, and is committed to diversity and inclusion[250](index=250&type=chunk)[251](index=251&type=chunk)[253](index=253&type=chunk) - In response to COVID-19, Mirum implemented work-from-home policies, enhanced safety measures for in-office work (health attestations, PPE, increased cleaning), and prohibited non-essential travel[250](index=250&type=chunk)[251](index=251&type=chunk) [Corporate Information](index=50&type=section&id=Corporate%20Information) Mirum Pharmaceuticals, Inc. was incorporated in Delaware in May 2018, with principal executive offices located in Foster City, California - Mirum Pharmaceuticals, Inc. was incorporated in Delaware in May 2018, with principal executive offices in Foster City, California[254](index=254&type=chunk) [Emerging Growth Company](index=50&type=section&id=Emerging%20Growth%20Company) Mirum is an 'emerging growth company' under the JOBS Act, benefiting from reduced reporting requirements, but opted out of extended accounting transition - Mirum is an 'emerging growth company' under the JOBS Act, allowing it to take advantage of reduced public company reporting requirements until December 31, 2024, or earlier if certain conditions are met[255](index=255&type=chunk) - The company has irrevocably elected not to use the extended transition period for complying with new or revised financial accounting standards, adhering to the same standards as other public companies[256](index=256&type=chunk) - Mirum also qualifies as a 'smaller reporting company,' enabling it to use scaled disclosures as long as its non-affiliate common stock market value is below **$250 million** or annual revenue is below **$100 million** and market value below **$700 million**[256](index=256&type=chunk) [Item 1A. Risk Factors](index=51&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks to Mirum's business, including operational, financial, intellectual property, and stock ownership challenges [Risks Related to Our Business and Industry](index=51&type=section&id=Risks%20Related%20to%20Our%20Business%20and%20Industry) Mirum faces risks from its limited operating history, significant losses, COVID-19 impacts, dependence on product candidates, and intense competition - Mirum has a very limited operating history since its inception in May 2018 and has incurred significant net losses, totaling **$173.2 million** accumulated deficit as of December 31, 2020, with expectations of continued losses[259](index=259&type=chunk)[260](index=260&type=chunk)[261](index=261&type=chunk) - The COVID-19 pandemic has adversely affected operations, including patient enrollment in clinical trials (e.g., Phase 3 MARCH), potential delays in regulatory approvals, and disruptions to supply chains and financial markets[263](index=263&type=chunk)[265](index=265&type=chunk)[268](index=268&type=chunk)[270](index=270&type=chunk)[271](index=271&type=chunk) - The business is highly dependent on the successful clinical testing, regulatory approval, and commercialization of maralixibat and volixibat, which is a lengthy, expensive, and uncertain process, with potential for delays or failure to demonstrate safety and efficacy[274](index=274&type=chunk)[276](index=276&type=chunk)[284](index=284&type=chunk)[289](index=289&type=chunk) - Market opportunities for rare cholestatic liver diseases may be smaller than anticipated, and competition from other biotechnology and pharmaceutical companies, including those developing ASBT inhibitors, is significant[302](index=302&type=chunk)[349](index=349&type=chunk)[351](index=351&type=chunk) - Even with Breakthrough Therapy and Orphan Drug Designations, there is no guarantee of faster approval or market exclusivity, and product candidates may cause undesirable side effects or fail to gain market acceptance[306](index=306&type=chunk)[322](index=322&type=chunk)[352](index=352&type=chunk)[356](index=356&type=chunk)[360](index=360&type=chunk) [Risks Related to Our Reliance on Third Parties](index=81&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) Mirum is highly dependent on third-party licensed IP, CROs for clinical trials, and contract manufacturers for drug supplies, posing significant risks - Mirum is highly dependent on intellectual property licensed from third parties (Shire, Pfizer, Satiogen, Sanofi), and termination of these licenses due to non-compliance or disputes could severely harm its business[397](index=397&type=chunk)[398](index=398&type=chunk)[448](index=448&type=chunk)[449](index=449&type=chunk) - The company relies on third-party CROs for clinical trials and contract manufacturers for drug supplies, and their failure to perform, comply with regulations (GCPs, cGMPs), or meet deadlines could delay or terminate development and commercialization[399](index=399&type=chunk)[400](index=400&type=chunk)[404](index=404&type=chunk)[405](index=405&type=chunk)[407](index=407&type=chunk) - Reliance on single-source suppliers and manufacturers, and potential disruptions from events like COVID-19, could jeopardize the supply of product candidates and increase costs[405](index=405&type=chunk)[407](index=407&type=chunk)[408](index=408&type=chunk) [Risks Related to Our Financial Position and Capital Requirements](index=83&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Capital%20Requirements) Mirum requires substantial additional financing, with potential for stockholder dilution or restrictive debt covenants, and NOL carryforward limitations - Mirum will require substantial additional financing to fund clinical development, commercialization, and operations, and may need to raise funds sooner than anticipated due to unforeseen expenses or market volatility[410](index=410&type=chunk)[411](index=411&type=chunk)[414](index=414&type=chunk) - Raising additional capital through equity or convertible debt will dilute existing stockholders, while debt financing may impose restrictive covenants. Strategic partnerships could require relinquishing valuable rights[416](index=416&type=chunk)[417](index=417&type=chunk) - The company's ability to utilize its federal and state net operating loss (NOL) carryforwards (approx. **$123.5 million** federal, **$2.6 million** state as of Dec 31, 2020) and tax credits may be limited by ownership changes (Section 382) or state-level suspensions[418](index=418&type=chunk)[419](index=419&type=chunk)[421](index=421&type=chunk) [Risks Related to Our Intellectual Property](index=85&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Mirum's success depends on obtaining and maintaining intellectual property protection, facing challenges with method-of-use patents and infringement claims - Mirum's commercial success depends on obtaining and maintaining sufficient intellectual property protection, including patents, trade secrets, and confidentiality agreements, which is complex and uncertain[422](index=422&type=chunk)[423](index=423&type=chunk)[424](index=424&type=chunk)[426](index=426&type=chunk)[427](index=427&type=chunk)[428](index=428&type=chunk) - Maralixibat relies on method-of-use and formulation patents, which are generally weaker than composition-of-matter patents and do not prevent competitors from marketing identical products for unpatented uses ('off-label')[438](index=438&type=chunk)[439](index=439&type=chunk) - The company faces risks of third-party claims alleging patent infringement, which could lead to costly litigation, development delays, substantial damages, or the need to obtain licenses on unfavorable terms[458](index=458&type=chunk)[460](index=460&type=chunk)[462](index=462&type=chunk)[463](index=463&type=chunk) - Protecting trade secrets is difficult, and unauthorized disclosure or independent development by competitors could harm Mirum's competitive position. Trademark protection is also crucial for brand recognition[481](index=481&type=chunk)[482](index=482&type=chunk)[484](index=484&type=chunk)[485](index=485&type=chunk)[487](index=487&type=chunk) [Risks Related to Ownership of Our Common Stock](index=99&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) The common stock price is highly volatile, stockholder returns depend on appreciation, and anti-takeover provisions could limit market price - The trading price of Mirum's common stock is highly volatile, influenced by clinical trial results, regulatory decisions, competition, financing, and general market conditions, potentially leading to significant fluctuations[489](index=489&type=chunk)[491](index=491&type=chunk) - The company does not intend to pay dividends, so stockholder returns depend solely on stock appreciation. Principal stockholders and management own a significant percentage, allowing them to exert substantial control[493](index=493&type=chunk)[494](index=494&type=chunk) - Payment obligations under the Revenue Interest Purchase Agreement (RIPA) could adversely affect financial position, increase vulnerability to downturns, and restrict cash flow for future operations[499](index=499&type=chunk) - Anti-takeover provisions in charter documents and Delaware law could delay or prevent a change of control, potentially limiting the market price of common stock[507](index=507&type=chunk)[508](index=508&type=chunk)[509](index=509&type=chunk) [General Risk Factors](index=107&type=section&id=General%20Risk%20Factors) Mirum faces risks from cybersecurity incidents, compliance with U.S. and foreign laws, and its status as an emerging growth company - Mirum's computer systems and those of its third-party partners are vulnerable to cybersecurity incidents, which could disrupt development programs, compromise intellectual property, and lead to significant liabilities[513](index=513&type=chunk)[516](index=516&type=chunk) - The company is subject to U.S. and foreign export/import controls, sanctions, anti-corruption laws (FCPA), and anti-money laundering laws, with violations potentially leading to criminal liability and business harm[519](index=519&type=chunk) - As an emerging growth company and smaller reporting company, Mirum benefits from reduced reporting requirements, but this status may make its stock less attractive to some investors and could lead to increased volatility[521](index=521&type=chunk)[522](index=522&type=chunk) [Item 1B. Unresolved Staff Comments](index=111&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) This item states that there are no unresolved staff comments from the SEC - Not applicable[533](index=533&type=chunk) [Item 2. Properties](index=111&type=section&id=Item%202.%20Properties) Mirum Pharmaceuticals leases its headquarters in Foster City, California and an office in Basel, Switzerland, deeming current facilities adequate - Mirum leases **11,200 square feet** for its headquarters in Foster City, California, with the agreement expiring in March 2025[534](index=534&type=chunk) - The company also leases approximately **1,400 square feet** for an office in Basel, Switzerland, under an agreement expiring in May 2024[534](index=534&type=chunk) - Existing facilities are considered adequate for current needs, and additional alternative spaces are expected to be available on commercially reasonable terms[534](index=534&type=chunk) [Item 3. Legal Proceedings](index=111&type=section&id=Item%203.%20Legal%20Proceedings) Mirum Pharmaceuticals may be involved in ordinary course legal proceedings, but management believes no pending claims will have a material adverse effect - The company may become involved in legal proceedings from the ordinary course of business[535](index=535&type=chunk) - Management believes there are currently no claims or actions pending that could materially adversely affect results of operations, financial condition, or cash flows[535](index=535&type=chunk) [Item 4. Mine Safety Disclosures](index=111&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to Mirum Pharmaceuticals - None[536](index=536&type=chunk) PART II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=112&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) This section covers Mirum's common stock market, stockholder matters, dividend policy, and the use of proceeds from its initial public offering [Market Information](index=112&type=section&id=Market%20Information) Mirum Pharmaceuticals' common stock has been listed on the Nasdaq Global Market under 'MIRM' since July 18, 2019 - Mirum Pharmaceuticals' common stock has been listed on the Nasdaq Global Market under the symbol 'MIRM' since July 18, 2019[539](index=539&type=chunk) [Holders of Common Stock](index=112&type=section&id=Holders%20of%20Common%20Stock) As of March 5, 2021, there were **30,419,707 shares** of common stock outstanding, held by approximately **15 record holders** - As of March 5, 2021, there were **30,419,707 shares** of common stock outstanding, held by approximately **15 record holders**[540](index=540&type=chunk) [Dividend Policy](index=112&type=section&id=Dividend%20Policy) Mirum has never paid cash dividends and intends to retain future earnings for business development and operations - Mirum has never declared or paid any cash dividends on its common stock and intends to retain future earnings for business development and operations[541](index=541&type=chunk) - Future dividend policy will be determined by the board of directors based on financial condition, operating results, and other relevant factors[541](index=541&type=chunk) [Securities Authorized for Issuance Under Equity Compensation Plans](index=112&type=section&id=Securities%20Authorized%20for%20Issuance%20Under%20Equity%20Compensation%20Plans) Information regarding equity compensation plans is incorporated by reference from Item 12 of Part III of this Annual Report - Information regarding equity compensation plans is incorporated by reference from Item 12 of Part III of this Annual Report[542](index=542&type=chunk) [Stock Performance Graph](index=112&type=section&id=Stock%20Performance%20Graph) This item is not applicable - Not applicable[543](index=543&type=chunk) [Use of Proceeds](index=112&type=section&id=Use%20of%20Proceeds) Mirum completed its IPO in July 2019, generating **$67.2 million** net proceeds, with management retaining broad discretion over their application - Mirum completed its IPO in July 2019, selling **5,000,000 shares** at **$15.00/share**, generating net proceeds of **$67.2 million** after deducting **$7.8 million** in underwriting discounts and offering expenses[544](index=544&type=chunk)[545](index=545&type=chunk) - As of December 31, 2020, approximately **$19.0 million** of the IPO net proceeds have been used, with funds invested in short- and intermediate-term, interest-bearing obligations[546](index=546&type=chunk) - Management retains broad discretion over the application of net proceeds, which will vary based on factors like additional financing, clinical trial success, and costs[548](index=548&type=chunk) [Item 6. Selected Financial Data](index=113&type=section&id=Item%206.%20Selected%20Financial%20Data) This item states that selected financial data is not applicable - Not applicable[549](index=549&type=chunk) [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=114&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section discusses Mirum's financial condition, operations, and liquidity, highlighting its limited history, losses, financing, and R&D focus [Overview](index=114&type=section&id=Overview) Mirum Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing late-stage novel therapies for orphan cholestatic liver diseases - Mirum Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing late-stage novel therapies for debilitating orphan cholestatic liver diseases[551](index=551&type=chunk) - The company's pipeline includes maralixibat (for ALGS, PFIC, BA) and volixibat (for PSC, ICP, PBC), with regulatory submissions and clinical trials ongoing[551](index=551&type=chunk) - Mirum has a limited operating history since November 2018, has incurred significant operating losses, and has not generated product sales revenue to date, funding operations primarily through debt and equity financings[552](index=552&type=chunk)[553](index=553&type=chunk) [Financing Transactions](index=114&type=section&id=Financing%20Transactions) Mirum completed its IPO and subsequent public offerings, generating significant net proceeds, and entered a Revenue Interest Purchase Agreement - Mirum completed its IPO in July 2019, selling **5,000,000 shares** at **$15.00/share**, yielding **$67.2 million** net proceeds[554](index=554&type=chunk) - In January 2020, a follow-on public offering of **2,400,000 shares** at **$20.00/sh
Mirum Pharmaceuticals (MIRM) Investor Presentation - Slideshow
2021-03-04 20:34
Corporate Presentation February 2021 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward-looking statements are subject to known and unknown risks, unc ...
Mirum Pharmaceuticals (MIRM) Investor Presentation - Slideshow
2020-12-04 19:29
Corporate Presentation December 2020 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward-looking statements are subject to known and unknown risks, unc ...
Mirum(MIRM) - 2020 Q3 - Quarterly Report
2020-11-12 11:08
[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section presents the unaudited financial statements, management's discussion and analysis, market risk disclosures, and internal controls [Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company reported no revenue for the periods ended September 30, 2020, with net loss increasing to $66.1 million for the nine months ended September 30, 2020, from $34.6 million in the prior-year period, driven by higher R&D and G&A expenses [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2020 (Unaudited) | Dec 31, 2019 | | :--- | :--- | :--- | | Cash and cash equivalents | $48,778 | $11,970 | | Short-term investments | $84,971 | $104,690 | | **Total assets** | **$141,865** | **$146,712** | | Total current liabilities | $18,792 | $13,076 | | **Total liabilities** | **$21,610** | **$16,363** | | Accumulated deficit | $(135,968) | $(69,901) | | **Total stockholders' equity** | **$120,255** | **$130,349** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Operating Results Summary (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2020 | Three Months Ended Sep 30, 2019 | Nine Months Ended Sep 30, 2020 | Nine Months Ended Sep 30, 2019 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $15,984 | $12,159 | $51,879 | $28,611 | | General and administrative | $5,732 | $3,708 | $15,466 | $7,474 | | **Loss from operations** | **$(21,716)** | **$(15,867)** | **$(67,345)** | **$(36,085)** | | **Net loss** | **$(21,506)** | **$(15,087)** | **$(66,067)** | **$(34,601)** | | **Net loss per share** | **$(0.86)** | **$(0.84)** | **$(2.65)** | **$(4.47)** | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for the Nine Months Ended September 30 (in thousands) | Cash Flow Activity | 2020 | 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | $(52,796) | $(25,664) | | Net cash provided by (used in) investing activities | $42,820 | $(122,637) | | Net cash provided by financing activities | $46,780 | $127,177 | | **Net increase (decrease) in cash** | **$36,808** | **$(21,149)** | | Cash and cash equivalents at end of period | $48,778 | $30,814 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) The company is a biopharmaceutical firm focused on developing therapies for debilitating liver diseases, with two main clinical-stage candidates: maralixibat and volixibat[23](index=23&type=chunk) - In January 2020, the company completed a follow-on public offering, selling 2,400,000 shares at $20.00 per share, resulting in **net proceeds of $44.7 million**[26](index=26&type=chunk) - In August 2020, the company initiated an at-the-market (ATM) offering program for up to **$75.0 million**, selling 98,708 shares during Q3 2020 and raising **net proceeds of approximately $2.2 million**[27](index=27&type=chunk) - Management believes that its cash, cash equivalents, and investments of **$133.7 million** as of September 30, 2020, are sufficient to fund operations for at least the next twelve months[28](index=28&type=chunk) - The company has future potential payment obligations to Shire, Satiogen, and Sanofi contingent on achieving clinical, regulatory, and commercial milestones for maralixibat and volixibat, with tiered royalties on net sales[50](index=50&type=chunk)[52](index=52&type=chunk)[54](index=54&type=chunk) - Subsequent to the quarter end, through November 12, 2020, the company sold an additional 189,757 shares under its ATM program, resulting in **net proceeds of approximately $3.6 million**[75](index=75&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on developing maralixibat and volixibat for rare liver diseases, highlighting increased operating losses due to escalated R&D and G&A activities, supported by recent equity financings, and noting the potential impact of COVID-19 on clinical trial enrollment [Overview](index=20&type=section&id=MD%26A%20Overview) - The company is developing maralixibat for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA), and volixibat for primary sclerosing cholangitis (PSC) and intrahepatic cholestasis of pregnancy (ICP)[80](index=80&type=chunk) - A rolling New Drug Application (NDA) submission for maralixibat for cholestatic pruritus in ALGS was initiated in Q3 2020, with completion expected in Q1 2021 and a potential launch in H2 2021[80](index=80&type=chunk) - The company plans to submit a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the treatment of PFIC2 in Q4 2020[80](index=80&type=chunk) - The COVID-19 pandemic has impacted the enrollment and conduct of clinical trials, specifically the Phase 3 MARCH trial[90](index=90&type=chunk) [Results of Operations](index=23&type=section&id=MD%26A%20Results%20of%20Operations) Comparison of Operating Expenses for the Nine Months Ended September 30 (in thousands) | Expense Category | 2020 | 2019 | Change | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Research and development | $51,879 | $28,611 | $23,268 | +81.3% | | General and administrative | $15,466 | $7,474 | $7,992 | +106.9% | | **Total operating expenses** | **$67,345** | **$36,085** | **$31,260** | **+86.6%** | - The **$23.3 million increase in R&D expenses** for the nine months ended Sep 30, 2020 was primarily due to a **$11.2 million increase in manufacturing activities**, an **$8.0 million increase in personnel costs**, and a **$5.1 million increase in clinical trial expenses**[111](index=111&type=chunk) - The **$8.0 million increase in G&A expenses** for the nine months ended Sep 30, 2020 was mainly driven by a **$5.4 million increase in personnel costs** and a **$2.0 million increase in expenses** related to operating as a public company[112](index=112&type=chunk) [Liquidity and Capital Resources](index=25&type=section&id=MD%26A%20Liquidity%20and%20Capital%20Resources) - As of September 30, 2020, the company had **$133.7 million in cash, cash equivalents, and investments** and an **accumulated deficit of $136.0 million**[114](index=114&type=chunk) - The company believes its existing capital is sufficient to fund operations through at least the next 12 months[118](index=118&type=chunk) - Net cash used in operating activities increased to **$52.8 million** for the nine months ended Sep 30, 2020, compared to **$25.7 million** for the same period in 2019, reflecting higher net loss partially offset by non-cash items like stock-based compensation[124](index=124&type=chunk)[125](index=125&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=27&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk exposure is to interest rate changes affecting its cash and investment portfolio, with foreign currency risk also considered not significant - Primary market risk is interest income sensitivity from changes in U.S. interest rates, but the impact is expected to be immaterial due to the short-term nature of the investment portfolio[137](index=137&type=chunk) - Foreign currency exchange rate risk, primarily from the Swiss Franc and Euro, is not currently believed to have a significant impact on operating results[138](index=138&type=chunk)[139](index=139&type=chunk) [Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, evaluated the company's disclosure controls and procedures and concluded they were effective as of September 30, 2020, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that as of September 30, 2020, the company's disclosure controls and procedures were effective[141](index=141&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended September 30, 2020, that have materially affected, or are reasonably likely to materially affect, internal controls[142](index=142&type=chunk) [PART II. OTHER INFORMATION](index=29&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section details legal proceedings, significant risk factors, unregistered equity sales, and other required disclosures [Legal Proceedings](index=29&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings that are expected to have a material adverse effect on its financial condition or operations - Management believes there are currently no pending claims or actions against the company that would have a material adverse effect on its results of operations, financial condition, or cash flows[145](index=145&type=chunk) [Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks, including a limited operating history with substantial losses, dependence on the success of its two product candidates, potential delays in clinical trials due to patient enrollment issues, reliance on third-party manufacturers and licensed intellectual property, the need for substantial additional financing, and competition - The business is highly dependent on the success of its product candidates, maralixibat and volixibat, which require significant further clinical testing and regulatory approval[147](index=147&type=chunk)[161](index=161&type=chunk) - The COVID-19 pandemic has adversely affected clinical trial operations, particularly patient enrollment in the Phase 3 MARCH trial, and could continue to cause delays and disruptions[151](index=151&type=chunk)[154](index=154&type=chunk) - The company depends on intellectual property licensed from third parties like Shire, Pfizer, and Sanofi, and the termination of these licenses could result in the loss of significant rights[152](index=152&type=chunk)[266](index=266&type=chunk) - Substantial additional financing will be needed to continue development and commercialization efforts, and failure to obtain it could force the company to delay or reduce its programs[147](index=147&type=chunk)[277](index=277&type=chunk) - The company faces significant competition from other companies developing therapies for cholestatic liver diseases, including those with ASBT inhibitors like GlaxoSmithKline and Albireo[229](index=229&type=chunk)[231](index=231&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=78&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities during the reporting period, with the company detailing the net proceeds of $67.2 million from its July 2019 IPO, which remain unused and held in cash, cash equivalents, and investments - The company completed its IPO on July 22, 2019, with **net proceeds of $67.2 million** after deducting underwriting discounts and offering expenses[377](index=377&type=chunk)[378](index=378&type=chunk) - Through September 30, 2020, the company has not used any of the net proceeds from its initial public offering, which are being held in cash, cash equivalents, and investments[379](index=379&type=chunk) [Other Information (Items 3-6)](index=78&type=section&id=Other%20Information%20(Items%203-6)) This section confirms there were no defaults upon senior securities, no mine safety disclosures required, and no other material information to report for the quarter - Item 3 (Defaults Upon Senior Securities), Item 4 (Mine Safety Disclosures), and Item 5 (Other Information) are all noted as not applicable or having nothing to report[380](index=380&type=chunk)[381](index=381&type=chunk)[382](index=382&type=chunk)
Mirum Pharmaceuticals (MIRM) Presents At Digital International Liver Congress 2020
2020-09-02 13:21
Serum bile acid control in long-term maralixibat- treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency Richard Thompson,1 Deirdre Kelly,2 Alexander Miethke,3 Sanjay Rajwal,4 Nisreen Soufi,5 Irena Jankowska,6 Cara Mack,7 Alain Lachaux,8 Thomas Jaecklin,9 Pamela Vig,10 Andrew J. Wardle,10 Robert H. Squires,11 Kathleen M Loomes12 1 INDIGO Study Author Disclosures I disclose the following financial relati ...
Mirum Pharmaceuticals (MIRM) Investor Presentation - Slideshow
2020-08-27 21:54
Corporate Presentation August 2020 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward-looking statements are subject to known and unknown risks, uncer ...
Mirum(MIRM) - 2020 Q2 - Quarterly Report
2020-08-06 20:18
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________to ________ Commission File Number: 001-38981 Mirum Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 83-1281555 ( ...