Disc Medicine(IRON) - 2024 Q1 - Quarterly Results

Company Overview Disc Medicine is a clinical-stage biopharmaceutical company focused on developing novel treatments for serious hematologic diseases by targeting red blood cell biology pathways Company Profile Disc Medicine is a clinical-stage biopharmaceutical company developing novel treatments for serious hematologic diseases by targeting heme biosynthesis and iron homeostasis - Disc Medicine is a clinical-stage biopharmaceutical company focused on novel treatments for serious hematologic diseases[2][9] - The company's portfolio targets fundamental biological pathways of red blood cell biology, including heme biosynthesis and iron homeostasis[9] CEO's Strategic Summary The CEO highlighted positive topline data from the AURORA study for bitopertin, confirming its activity in reducing toxic PPIX and improving quality of life in EPP patients, with upcoming data and regulatory interactions planned - Topline data from AURORA strengthened belief in bitopertin's activity, showing significant reduction in toxic PPIX in EPP patients, reduced phototoxic pain reactions, and improved quality of life[3] - Additional data from AURORA and BEACON studies, updated data for DISC-0974 in anemia of myelofibrosis, and initial data on DISC-3405 in healthy volunteers are expected in June (Q2 2024)[3] - Regulatory interactions for bitopertin are anticipated in the second half of 2024[3] Recent Business Highlights and Upcoming Milestones The company reported positive bitopertin AURORA study results, received FDA RPD designation, and anticipates further clinical data and regulatory interactions in 2024 for its pipeline Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Top-line AURORA Phase 2 results for bitopertin in EPP showed significant PPIX reduction and improved phototoxic reactions, with FDA RPD designation received and regulatory interactions planned - Presented top-line results from AURORA, the placebo-controlled phase 2 study of bitopertin in erythropoietic porphyrias (EPP), in April 2024[5] - Received FDA Rare Pediatric Disease Designation (RPD) in May 2024 for the treatment of EPP[6] - Regulatory interactions to define optimal registrational endpoints for EPP are expected in the second half of 2024[6] AURORA Phase 2 Study Results The AURORA trial demonstrated significant, dose-dependent reductions in whole blood protoporphyrin IX (PPIX) levels and improvements in light tolerance and patient global impression of change, with statistical significance for the 60 mg dose group in reducing phototoxic reactions with pain - Significant, dose-dependent, and sustained reductions in whole blood protoporphyrin IX (PPIX) levels were observed[6] - Improvement on measures of light tolerance, but did not meet statistical significance due to a greater than expected benefit in the placebo arm[6] - Dose-dependent reductions in phototoxic reactions with pain, reaching statistical significance for the 60 mg group, and dose-dependent improvements in Patient Global Impression of Change[6] Upcoming Bitopertin Milestones Additional analyses from the BEACON and AURORA trials are on track to be delivered and presented in Q2 2024 - Additional analyses from the BEACON and AURORA trials are on track to be delivered in Q2 2024[5] - Additional analyses from the AURORA and BEACON trials to be presented in Q2 2024[6] DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Updated Phase 1b/2 data for DISC-0974 in anemia of myelofibrosis (MF) patients, including safety and changes in hepcidin, iron, and hemoglobin levels, are expected to be presented in Q2 2024, with NDD-CKD data anticipated in H2 2024 - Updated phase 1b/2 data from DISC-0974 in anemia in MF patients will be presented in Q2 2024, including safety and changes in hepcidin, iron, and hemoglobin levels[12] - Updated phase 1b/2 data from DISC-0974 in anemia in non-dialysis-dependent chronic kidney disease (NDD-CKD) is expected to be shared in the second half of 2024[12] DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Initial pharmacokinetic and pharmacodynamic data from single-ascending dose (SAD) cohorts of DISC-3405 in healthy volunteers are expected to be presented in Q2 2024 - Initial pharmacokinetic and pharmacodynamic data from SAD cohorts of DISC-3405 are expected to be presented in Q2 2024[7] - Initial single-ascending dose (SAD) data from the phase 1 study of DISC-3405 in healthy volunteers is planned for Q2 2024[5] First Quarter 2024 Financial Results Disc Medicine reported an increased net loss for Q1 2024 due to higher R&D and G&A expenses, maintaining a strong cash position into 2026 Financial Summary Disc Medicine reported a net loss of $26.9 million for Q1 2024, an increase from $22.8 million in Q1 2023, primarily due to increased R&D and G&A expenses driven by portfolio progression and headcount, with $342.6 million in cash and cash equivalents providing a runway well into 2026 Financial Performance Summary | Metric | Q1 2024 (in thousands) | Q1 2023 (in thousands) | Change (YoY) | | :-------------------------- | :--------------------- | :--------------------- | :----------- | | Research and Development | $23,704 | $20,180 | +17.46% | | General and Administrative | $7,758 | $4,945 | +56.89% | | Net Loss | $(26,949) | $(22,781) | +18.38% | | Net Loss per Share (Diluted)| $(1.09) | $(1.20) | -9.17% | - Cash and cash equivalents were $342.6 million as of March 31, 2024, expected to fund operational plans well into 2026[5][12] - Increase in R&D expenses was primarily driven by the progression of Disc's portfolio, including bitopertin's ongoing two phase 2 clinical studies and drug manufacturing, and increased headcount[12] Condensed Consolidated Statements of Operations For the three months ended March 31, 2024, total operating expenses increased to $31.5 million from $25.1 million in the prior year, leading to a net loss of $26.9 million, or $(1.09) per share Condensed Consolidated Statements of Operations (in thousands) | Operating Expenses | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Research and development | $23,704 | $20,180 | | General and administrative | $7,758 | $4,945 | | Total operating expenses | $31,462 | $25,125 | | Loss from operations | $(31,462) | $(25,125) | | Other income (expense), net | $4,518 | $2,367 | | Income tax expense | $(5) | $(23) | | Net loss | $(26,949) | $(22,781) | | Weighted-average common shares outstanding-basic and diluted | 24,809,869 | 18,954,914 | | Net loss per share-basic and diluted | $(1.09) | $(1.20) | Condensed Consolidated Balance Sheets As of March 31, 2024, Disc Medicine reported total assets of $354.2 million, a decrease from $368.0 million at December 31, 2023, with cash and cash equivalents decreasing to $342.6 million and total liabilities decreasing to $16.1 million Condensed Consolidated Balance Sheets (in thousands) | Balance Sheet Item | March 31, 2024 | December 31, 2023 | | :-------------------------------- | :------------- | :---------------- | | Cash and cash equivalents | $342,615 | $360,382 | | Total current assets | $351,948 | $365,662 | | Total assets | $354,182 | $367,996 | | Total liabilities | $16,111 | $22,875 | | Total stockholders' equity | $338,071 | $345,121 | Additional Information This section details how Disc Medicine disseminates material information and provides a cautionary statement regarding forward-looking statements and contact information Available Information Disc Medicine disseminates material information to the public through SEC filings, press releases, public conference calls, webcasts, and its investor relations website to ensure broad distribution and compliance with Regulation FD - Disc announces material information via SEC filings, press releases, public conference calls, webcasts, and its investor relations website (ir.discmedicine.com) for broad, non-exclusionary distribution and Regulation FD compliance[10] Disc Cautionary Statement Regarding Forward-Looking Statements This section warns that the press release contains forward-looking statements regarding clinical trials, timelines, business plans, and financial projections, which are based on current beliefs and assumptions and are subject to risks and uncertainties, with actual results potentially differing materially - The press release contains forward-looking statements regarding clinical studies (AURORA, BEACON, DISC-0974, DISC-3405), timelines, business plans, and financial projections[11][13] - These statements are based on current beliefs and assumptions and are subject to risks and uncertainties, meaning actual results or events could differ materially[13][14] - Investors are cautioned not to place undue reliance on these forward-looking statements, and the company undertakes no obligation to update them[14] Contacts Provides contact information for media and investor relations inquiries - Media Contact: Peg Rusconi at prusconi@vergescientific.com[18] - Investor Relations Contact: Christina Tartaglia at christina.tartaglia@sternir.com[18]