Astria Therapeutics (ATXS) 2025 Conference May 07, 2025 10:30 AM ET Speaker0 Morning, everybody, and welcome to day one of the Citizens Life Science Conference. My name is John Wallabin, senior analyst here. We're pleased to have Astria Therapeutics and CEO, Jill Millney, joining us today. Astria is a company we launched coverage on, I think, earlier this year in January. We've been covering the hereditary angioedema space for quite some time, and I think this is one of the more compelling pipeline candidat ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 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: 001-37467 Astria Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 26-3687168 (State or ...

Financial Performance - Net loss for 2024 was $94,260,000, up from $72,891,000 in 2023, indicating a 29.2% increase in losses[16] - The net loss for Q4 2024 was $25.6 million, compared to a net loss of $31.4 million in Q4 2023, reflecting a 18% improvement[10] - The operating loss for Q4 2024 was $29.6 million, a decrease of 13% from $34.2 million in Q4 2023[9] - Net loss per share attributable to common shareholders improved to $(1.68) in 2024 from $(2.42) in 2023[16] - The net loss per share for Q4 2024 was $0.44, down from $0.86 per share in Q4 2023, representing a 49% reduction[11] Cash and Investments - As of December 31, 2024, Astria had cash, cash equivalents, and short-term investments of $328.1 million, up from $246.5 million as of December 31, 2023, indicating a 33% increase year-over-year[6] - Cash and cash equivalents decreased to $59,820,000 in 2024 from $175,530,000 in 2023, a decline of 66.0%[18] - Net cash used in operating activities was $(81,212,000) in 2024, compared to $(68,445,000) in 2023, representing a 18.5% increase in cash outflow[20] - Net cash provided by financing activities rose to $157,202,000 in 2024 from $88,398,000 in 2023, an increase of 77.8%[20] Operating Expenses - Total operating expenses for 2024 increased to $111,558,000 from $83,030,000 in 2023, representing a 34.5% increase[16] - Research and development expenses for Q4 2024 were $20.2 million, a 73% increase from $11.7 million in Q4 2023, driven by external expenses for clinical trials[7] - Research and development expenses rose significantly to $77,106,000 in 2024, compared to $42,127,000 in 2023, marking a 83.1% increase[16] - General and administrative expenses for Q4 2024 were $9.4 million, up 29% from $7.3 million in Q4 2023, attributed to stock-based compensation and company growth[8] Assets and Liabilities - Total assets increased to $342,363,000 in 2024, up from $254,666,000 in 2023, reflecting a growth of 34.3%[18] - Total stockholders' equity increased to $319,263,000 in 2024, up from $243,116,000 in 2023, a growth of 31.3%[18] - Total liabilities increased to $23,100,000 in 2024 from $11,550,000 in 2023, a rise of 99.0%[18] Clinical Trials - The ALPHA-ORBIT Phase 3 trial of navenibart commenced in February 2025, with top-line results expected in early 2027, involving up to 135 adult and 10 adolescent patients[3] - Initial efficacy and safety data from the ALPHA-SOLAR long-term extension trial of navenibart are expected in mid-2025[1] - The Phase 1a trial of STAR-0310 is ongoing, with initial results anticipated in Q3 2025, aiming to demonstrate its differentiated profile[2]

Company Performance - Astria Therapeutics, Inc. (ATXS) has gained approximately 25.9% year-to-date, significantly outperforming the Medical sector, which has returned an average of -2.7% [4] - The Zacks Consensus Estimate for ATXS' full-year earnings has increased by 4.5% over the past 90 days, indicating improving analyst sentiment and a positive earnings outlook [3] Industry Comparison - Astria Therapeutics, Inc. is part of the Medical - Biomedical and Genetics industry, which includes 501 individual stocks and currently ranks 69 in the Zacks Industry Rank. This industry has seen an average loss of 9.7% this year, highlighting ATXS's superior performance [5] - In contrast, Abbott (ABT), another stock in the Medical sector, has a year-to-date return of 2.3% and belongs to the Medical - Products industry, which is ranked 85 and has moved up by 11.4% year-to-date [4][6] Sector Overview - The Medical sector consists of 1021 individual stocks and currently holds a Zacks Sector Rank of 3, which measures the strength of the sector groups based on the average Zacks Rank of individual stocks [2] - The Zacks Rank system emphasizes earnings estimates and revisions, with ATXS currently holding a Zacks Rank of 2 (Buy), indicating a favorable outlook compared to the broader market [3]

Financial Performance - The company reported net losses of $68.6 million for the nine months ended September 30, 2024, compared to $41.5 million for the same period in 2023[65]. - The company has an accumulated deficit of $649.2 million as of September 30, 2024, and has not generated any product revenues[65]. - Net loss for the nine months ended September 30, 2024, was $68.63 million, an increase of $27.15 million or 65% compared to a net loss of $41.48 million in the prior year[74]. - Other income, net increased by $6.0 million to $13.3 million for the nine months ended September 30, 2024, a 82% increase from $7.3 million in the same period of 2023[77]. - For the nine months ended September 30, 2024, net cash used in operating activities was $63.9 million, compared to $38.2 million for the same period in 2023[83]. - Net cash used in investing activities was $188.1 million for the nine months ended September 30, 2024, primarily due to purchases of short-term investments totaling $3.5 billion[83]. - Net cash provided by financing activities was $157.2 million for the nine months ended September 30, 2024, driven by net proceeds from the February 2024 Financing and ATM Programs[85]. Cash and Funding - As of September 30, 2024, the company had $344.3 million in cash, cash equivalents, and short-term investments, expected to fund operations into mid-2027[65]. - The gross proceeds from the October 2023 Financing were $64.0 million, and from the February 2024 Financing were $125.0 million[64]. - Existing cash, cash equivalents, and short-term investments are expected to fund operations into mid-2027, covering ongoing clinical trials and development activities[86]. - The company expects to finance future cash needs through equity offerings, debt financings, and collaborations, with no committed external sources of funds[88]. - The company sold 1,504,619 shares under the 2024 ATM Program for gross proceeds of $15.6 million in the three months ended September 30, 2024[81]. Research and Development - Research and development expenses increased by $26.5 million to $56.9 million for the nine months ended September 30, 2024, representing an 87% increase compared to the same period in 2023[75]. - Research and development expenses for the three months ended September 30, 2024, were $20.5 million, a 54% increase from $13.3 million in the same period of 2023[72]. - The company expects to incur significant research and development expenses in 2024 related to clinical trials for navenibart and STAR-0310, with higher expenses anticipated over the next several quarters[72]. Clinical Trials and Product Development - Navenibart achieved a 92% reduction in monthly attack rate and a 96% reduction in moderate and severe attacks in Cohort 1 after 6 months of follow-up[57]. - In Cohort 2, a 600 mg dose followed by a 300 mg dose resulted in a 96% reduction in monthly attack rate and 67% of patients were attack-free after 6 months[59]. - The company plans to initiate a Phase 1a clinical trial for STAR-0310 in healthy subjects in Q1 2025, with initial results expected in Q3 2025[63]. - The company aims to start a Phase 3 pivotal trial for Navenibart in Q1 2025, with top-line results expected by year-end 2026[59]. - STAR-0310 exhibited a long mean half-life of 26 days in preclinical studies, compared to 10-14 days for typical non-half-life extended IgG antibodies[60]. - Navenibart has received Fast Track and Orphan Drug designations from the FDA for the treatment of hereditary angioedema[57]. Operating Expenses - General and administrative expenses rose by $6.6 million to $25.0 million for the nine months ended September 30, 2024, a 36% increase from the prior year[77]. - General and administrative expenses for the three months ended September 30, 2024, increased by $1.6 million to $8.5 million, a 23% increase from the prior year[72]. - Total operating expenses for the nine months ended September 30, 2024, were $81.97 million, up $33.14 million or 68% from $48.83 million in the same period of 2023[74]. - Total operating expenses for the three months ended September 30, 2024, were $29.01 million, up $8.78 million or 43% from $20.24 million in the same period of 2023[71]. Internal Controls - The company has not made any changes to its internal control over financial reporting that materially affected its financial reporting during the nine months ended September 30, 2024[92].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 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, 2024 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: 001-37467 Astria Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) | --- | --- | --- | |---- ...

Clinical Trial Results - STAR-0215 achieved a 92% reduction in monthly attack rate and a 96% reduction in moderate and severe attacks in Cohort 1 after 6 months of follow-up[79]. - STAR-0215 demonstrated a 96% reduction in monthly attack rate and 98% reduction in moderate and severe attacks in Cohort 2, with 100% of patients being attack-free in the first month after dosing[80]. - STAR-0215 has received Fast Track designation from the FDA for the treatment of hereditary angioedema[78]. - The company plans to initiate a pivotal Phase 3 trial for STAR-0215 in Q1 2025, with top-line results expected by year-end 2026[81]. - STAR-0310 is anticipated to enter Phase 1a clinical trials in Q1 2025, with initial results expected in Q3 2025[85]. - The company plans to expand enrollment in the ALPHA-STAR trial to a total of up to 28 patients to accelerate data collection for STAR-0215[81]. - The company anticipates initiating a Phase 3 pivotal trial for STAR-0215 in Q1 2025 and submitting an IND for STAR-0310 by year-end 2024[102]. Financial Performance - The company reported net losses of $19.9 million for the three months ended March 31, 2024, compared to $11.2 million for the same period in 2023, resulting in an accumulated deficit of $600.5 million[87]. - Research and development expenses totaled $15.7 million for the three months ended March 31, 2024, significantly higher than $8.0 million for the same period in 2023[92]. - General and administrative expenses rose by $2.9 million to $8.4 million for the three months ended March 31, 2024, an increase of 54% year-over-year[102]. - Total operating expenses for the three months ended March 31, 2024, were $24.2 million, up $10.7 million from $13.5 million in the same period of 2023[99]. - Net loss for the three months ended March 31, 2024, was $19.9 million, compared to a net loss of $11.2 million for the same period in 2023, reflecting an increase of $8.7 million[99]. - Other income, net increased by $1.9 million to $4.2 million for the three months ended March 31, 2024, an increase of 83% year-over-year[103]. - Net cash used in operating activities was $19.1 million for the three months ended March 31, 2024, compared to $13.3 million for the same period in 2023[111]. - Net cash provided by financing activities was $141.8 million for the three months ended March 31, 2024, primarily from the February 2024 Financing[114]. Cash Position and Funding - As of March 31, 2024, the company had $369.9 million in cash, cash equivalents, and short-term investments, expected to fund operations into mid-2027[88]. - The company raised an aggregate of $823.3 million through equity financings from inception through March 31, 2024[105]. - The company expects its existing cash, cash equivalents, and short-term investments to fund operations into mid-2027[117]. - Future funding requirements will depend on clinical trial progress, collaboration agreements, and regulatory approval outcomes[117]. - The company does not have any committed external sources of funds and may rely on equity offerings, debt financings, and collaborations for financing[119]. - If additional funds are not raised when needed, the company may have to delay or terminate product development efforts[120]. - Material cash requirements as of March 31, 2024, are primarily related to sublease agreements for office space[121]. Commercialization and Market Competition - The costs of commercialization activities for product candidates that receive marketing approval will be significant and may not be fully covered by collaborators[117]. - The company acknowledges that product candidates may not achieve commercial success, impacting future revenues[118]. - The company’s ability to compete against other approved products will be critical for the success of STAR-0215 and STAR-0310[117]. - The license agreement with Ichnos includes potential milestone payments and tiered royalties contingent upon product development and regulatory milestones[122]. - The company may face dilution of stockholders' ownership interests if additional capital is raised through equity sales[119].

Core Insights - Astria Therapeutics, Inc. granted stock options to purchase 40,000 shares of its common stock as part of its 2022 Inducement Stock Incentive Plan [1][2] - The stock options were granted to two new employees as an inducement for their employment, in compliance with Nasdaq Listing Rule 5635(c)(4) [2] - The exercise price of the options is set at $14.98, equal to the closing price of Astria's common stock on the grant date, March 1, 2024 [2] Company Overview - Astria Therapeutics is focused on developing therapies for allergic and immunological diseases, with a mission to provide life-changing treatments [3] - The company's lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein, currently in clinical development for hereditary angioedema [3] - The second program, STAR-0310, is a monoclonal antibody OX40 antagonist in preclinical development for atopic dermatitis [3]

Corporate Overview - Astria Therapeutics, Inc. is focused on developing therapies for allergic and immunological diseases, with lead programs STAR-0215 for hereditary angioedema (HAE) and STAR-0310 for atopic dermatitis (AD) [10] Financial Results - As of December 31, 2023, the company had cash, cash equivalents, and short-term investments totaling $246.5 million, an increase from $188.8 million as of September 30, 2023 [6] - Net cash used in operating activities for Q4 2023 was $30.2 million, compared to $10.7 million for Q4 2022, while the full year 2023 net cash used was $68.4 million, up from $43.5 million in 2022 [7][9] - Research and development expenses for Q4 2023 were $11.7 million, compared to $9.6 million in Q4 2022, and for the full year 2023, they were $42.1 million, up from $34.3 million in 2022 [8] - General and administrative expenses for Q4 2023 were $7.3 million, compared to $4.7 million in Q4 2022, with full year expenses of $25.7 million versus $19.2 million in 2022 [8] - The operating loss for Q4 2023 was $34.2 million, compared to $14.3 million in Q4 2022, and the full year loss was $83.0 million, up from $53.5 million in 2022 [9] Product Development Updates - The ALPHA-STAR Phase 1b/2 trial for STAR-0215 is on track, with initial proof-of-concept results expected in Q1 2024, assessing safety, tolerability, and efficacy in HAE patients [3] - STAR-0310 is in preclinical development, with an IND submission expected by year-end 2024 and a Phase 1a trial planned to start in Q1 2025 [4] Recent Financing - In February 2024, Astria closed an underwritten offering of 10,340,000 shares of common stock, raising approximately $125.0 million before expenses [5]

Clinical Development - STAR-0215 is in clinical development for hereditary angioedema (HAE) and has the potential to be a best-in-class monoclonal antibody with administration every three or six months[26]. - The Phase 1a clinical trial of STAR-0215 demonstrated a favorable safety profile, with no serious adverse events and an estimated half-life of up to 109 days[28]. - Initial results from the Phase 1b/2 trial (ALPHA-STAR) are expected in Q1 2024, which will include safety, tolerability, pharmacokinetics, pharmacodynamics, and HAE attack-rate reduction data[30]. - The company plans to initiate a Phase 1a clinical trial for STAR-0310 in healthy subjects in Q1 2025, with initial results expected in Q3 2025[33]. - The Phase 1b trial for STAR-0310 in patients with AD is planned for the second half of 2025, with results expected in Q2 2026[33]. - Initial proof-of-concept data for STAR-0215 in HAE patients is expected in Q1 2024, with a Phase 3 pivotal trial anticipated to start in Q1 2025 if results are positive[40]. - The company plans to submit an IND for STAR-0310 by the end of 2024 and initiate a Phase 1a clinical trial in Q1 2025[51]. - The company has completed process and formulation development for STAR-0215 and is set to start process characterization and subsequent validation, ensuring sufficient material for clinical needs[74]. - The company has initiated cell line, process, and formulation development for STAR-0310, expanding its product pipeline[74]. - The company acknowledges that clinical trials may not yield results predictive of future success, and setbacks in late-stage trials are possible[213]. - Clinical trials for STAR-0215 are expected to be longer due to the dosing schedule of every three months or potentially less frequently[218]. - STAR-0310 clinical trials will be larger and more expensive compared to STAR-0215 due to its indication for a non-rare disease[218]. - Risks of clinical development include failure to demonstrate efficacy, insufficient patient enrollment, and potential regulatory disapproval[218]. - Delays in clinical trials could harm the commercial prospects of product candidates and delay revenue generation[218]. - Adverse events during trials could lead to significant delays or failure to obtain regulatory approval[218]. - The company faces heightened risks in drug development for rare diseases due to smaller patient populations[218]. Market Potential - The global market for HAE therapy is strong and growing, with an unmet medical need for effective treatments[27]. - The HAE treatment market was estimated to be over $2 billion in 2022 and is projected to grow to $4.5 billion by 2027, driven by earlier diagnoses and increased treatment adoption[37]. - The moderate-to-severe AD treatment market was approximately $7 billion in 2022 and is expected to grow to $26 billion by 2030 due to increased drug treatment rates and new therapies[48]. - Market research indicates strong interest from U.S. physicians and HAE patients for a product with the potential profile of STAR-0215[38]. - The competitive landscape for HAE and AD treatments is significant, with many companies developing alternative therapies that may impact market share[56][60]. Product Development - STAR-0310, a preclinical product candidate for atopic dermatitis (AD), is anticipated to submit an investigational new drug application (IND) by year-end 2024[33]. - STAR-0310 is being developed as a potential best-in-class treatment for AD, utilizing YTE half-life extension technology for infrequent dosing[43]. - STAR-0310 aims to address the unmet need for safe and effective therapies for moderate and severe AD patients, targeting multiple T cell pathways[46]. - STAR-0310 has shown potential for better efficacy compared to existing biologics, with preclinical studies indicating lower antibody-dependent cellular cytotoxicity[47]. - The company is exploring STAR-0310 for additional allergic and immunological indications, including asthma and chronic urticaria[52]. - The company is developing a drug device combination product for STAR-0215, indicating ongoing innovation in product offerings[74]. Regulatory Environment - STAR-0215 has received Fast Track designation from the FDA for the treatment of HAE, indicating its potential significance in addressing this condition[27]. - Regulatory processes for drug approval require substantial time and financial resources, including compliance with FDA regulations and obtaining necessary approvals[78]. - The FDA aims to review 90% of applications for New Molecular Entities (NMEs) within ten months of acceptance for filing, and 90% of priority review applications within six months[118]. - The FDA requires at least two adequate and well-controlled clinical trials to establish the effectiveness of a new product, although a single trial may suffice under certain circumstances[121]. - The FDA's acceptance of foreign clinical trial data for U.S. marketing approval is contingent upon compliance with GCP regulations and validation through on-site inspections if necessary[101]. - The FDA issues either a Complete Response Letter (CRL) or an approval letter after evaluating drug applications, determining that the drug is effective and its benefits outweigh risks[122]. - The FDA strictly regulates marketing and promotion of approved products, allowing promotion only for approved indications[140]. - Manufacturers must maintain compliance with regulatory requirements post-approval, or risk withdrawal of approval[139]. Intellectual Property - STAR-0215 and STAR-0310 have patent protections that could extend until 2042 and 2044 respectively, assuming all maintenance fees are paid[65][67]. - The patent term for STAR-0215 and STAR-0310 may be extended under the Hatch-Waxman Act, potentially adding up to five years beyond the original patent expiration[70][71]. - The company has in-licensed multiple patent families related to its product candidates, enhancing its intellectual property portfolio[68]. - Patent term restoration under the Hatch-Waxman Act allows for a limited extension of up to five years for patents lost during product development and FDA review, but cannot exceed a total of 14 years from the product's approval date[150]. Financial Considerations - Significant investment and access to commercial manufacturing capacity will be required for the development and commercialization of STAR-0215 and STAR-0310[207]. - The application user fee for federal fiscal year 2024 is approximately $4.05 million, with an annual program fee of $416,734 per eligible prescription product[116]. - The U.S. government is focusing on pharmaceutical pricing transparency and reducing costs under Medicare and Medicaid[189]. - The IRA caps Medicare out-of-pocket drug costs at $4,000 in 2024 and $2,000 in 2025, shifting some costs to drug manufacturers[194]. Workforce and Culture - As of December 31, 2023, the company employed 59 full-time employees, with 33 primarily engaged in research and development activities, including 14 with Ph.D. degrees[75]. - The company aims to attract, retain, and motivate employees through market-based cash compensation and equity incentive plans, which consist solely of stock options[76]. - The company emphasizes creating an inclusive environment where diversity is valued, considering its workforce as one of its biggest assets[77].