Financial Performance - The net loss for the three months ended March 31, 2025, was $89.3 million, compared to a net loss of $81.5 million for the same period in 2024, resulting in an accumulated deficit of $238.2 million as of March 31, 2025[89]. - Total revenue for the three months ended March 31, 2025, was $8.726 million, an increase of $0.5 million compared to $8.189 million in the same period of 2024[120]. - Total operating expenses for the three months ended March 31, 2025, were $115.355 million, an increase of $15.1 million compared to $100.261 million in the same period of 2024[121]. - Research and development expenses for the three months ended March 31, 2025, totaled $72.743 million, an increase of $4.1 million compared to $68.620 million in the same period of 2024[123]. - Net cash used in operating activities for the three months ended March 31, 2025, was $111.5 million, compared to $99.9 million for the same period in 2024[132][133]. - Cash provided by investing activities was $112.6 million for the three months ended March 31, 2025, compared to $124.6 million in 2024[134]. - Interest income, net for the three months ended March 31, 2025, was $16.087 million, an increase from $8.889 million in the same period of 2024[124]. - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.4 billion[131]. Product Development and Approvals - PYRUKYND® is approved by the FDA for treating hemolytic anemia in adults with PK deficiency, and a supplemental new drug application was submitted for treating alpha- or beta-thalassemia, with a PDUFA goal date of September 7, 2025[81]. - The company is evaluating PYRUKYND® in a phase 3 clinical trial for sickle cell disease and developing other candidates like tebapivat for lower-risk myelodysplastic syndromes[82]. - PYRUKYND® received FDA approval for treating hemolytic anemia in adults with PK deficiency and marketing authorization in the EU and Great Britain[99]. - The company submitted an sNDA to the FDA for PYRUKYND® for treating non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia, with a PDUFA goal date of September 7, 2025[99]. - Tebapivat has been granted orphan drug designation for the treatment of MDS by the FDA, with a phase 2 clinical trial expected to begin in mid-2025[114]. Clinical Trials and Results - In the ENERGIZE trial, 42.3% of patients on PYRUKYND® achieved a hemoglobin response compared to 1.6% in the placebo group, demonstrating statistical significance (p<0.0001) with 194 patients enrolled[102]. - In the ENERGIZE-T trial, 30.4% of patients on PYRUKYND® achieved a transfusion reduction response compared to 12.6% in the placebo group (p=0.0003), with 258 patients enrolled[105]. - The RISE UP study showed that 46.2% and 50.0% of patients in the 50 mg and 100 mg arms, respectively, achieved a hemoglobin response compared to 3.7% in the placebo arm (p=0.0003 and 0.0001) with 79 patients enrolled[109]. - In the ACTIVATE-kidsT study, 28.1% of patients in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 11.8% in the placebo arm[111]. - In the ACTIVATE-kids study, 31.6% of patients in the mitapivat arm achieved a hemoglobin response, while 0% of patients in the placebo arm achieved this response[113]. - The company is evaluating PYRUKYND® in clinical trials for SCD and pediatric patients with PK deficiency[99]. - The company has updated clinical trial protocols to include monthly monitoring of liver tests for the first six months of treatment due to potential hepatocellular injury risks[107]. - The company expects to announce topline data for the phase 3 portion of the RISE UP trial in late 2025, with a potential U.S. commercial launch in 2026 if approved[109]. Business Transactions and Collaborations - The sale of the oncology business to Servier was completed for approximately $1.8 billion in cash, with additional contingent payments based on future approvals and sales[83]. - In September 2024, the company recognized income of $200.0 million from the Vorasidenib Milestone Payment following FDA approval for vorasidenib[86]. - The company recognized income of $889.1 million from the sale of Vorasidenib Royalty Rights to Royalty Pharma, net of fees[87]. - The company entered into a distribution agreement with NewBridge Pharmaceuticals for PYRUKYND® in the GCC region, expanding its market reach[91]. - The company entered a distribution agreement with NewBridge Pharmaceuticals for PYRUKYND® commercialization in the GCC region[100]. Future Outlook and Risks - The company expects to continue generating revenue from product sales and potential milestone payments from collaborations or licensing agreements in the future[92]. - Research and development expenses are expected to increase as product candidate development programs progress, with significant uncertainty regarding costs and timelines[95]. - The company has historically incurred significant operating losses and expects to continue incurring significant expenses until achieving profitable results[89]. - The company expects expenses to increase as it continues research, development, and commercialization of its product candidates, including PYRUKYND®[136]. - Future capital requirements will depend on various factors, including the success of product sales and potential royalty payments from Retained Earn-Out Rights[139]. - The company is exposed to market risks related to interest rates and foreign currency exchange rates, with minimal liabilities in foreign currencies as of March 31, 2025[142][145]. - The company anticipates financing cash needs primarily through cash on hand, potential royalty payments, and product sales until substantial revenue is generated[139]. - The company may need additional capital resources to fund operating plans and capital expenditures, which could involve dilution of stockholder ownership[139].

Q1 2025 Financial Results and Business Update May 1, 2025 1 Agios Conference Call Participants | TOPIC | PARTICIPANT | | --- | --- | | Introduction | Chris Taylor, VP Investor Relations and Corporate | | | Communications | | Business Update | Brian Goff, Chief Executive Officer | | R&D Update | Sarah Gheuens, M.D., Ph.D., Chief Medical Officer, | | | Head of R&D | | Commercial Update | Tsveta Milanova, Chief Commercial Officer | | First Quarter 2025 Financial Results | Cecilia Jones, Chief Financial Officer ...

Agios Pharmaceuticals (AGIO) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Speaker0 Good morning, and welcome to Agil's First Quarter twenty twenty five Conference Call. At this time, all participants are in a listen only mode. There will be a question and answer session at the end. Please be advised that this call is being recorded at Agios' request. I would now like to turn the call over to Chris Taylor, Vice President of Investor Relations and Corporate Communications for Agios. Speaker1 Thank you, oper ...

Financial Performance - PYRUKYND generated net revenue of $8.7 million in Q1 2025, up from $8.2 million in Q1 2024, representing a year-over-year increase of 6.1%[3] - The net loss for Q1 2025 was $89.3 million, compared to a net loss of $81.5 million in Q1 2024, reflecting an increase of 9.8%[7] - Cash, cash equivalents, and marketable securities totaled $1.4 billion as of March 31, 2025, down from $1.5 billion at the end of 2024[8] Expenses - Research and Development (R&D) expenses for Q1 2025 were $72.7 million, an increase of 5.2% compared to $68.6 million in Q1 2024[5] - Selling, General and Administrative (SG&A) expenses rose to $41.5 million in Q1 2025, compared to $31.0 million in Q1 2024, marking a 33.9% increase[7] Patient Enrollment - The total number of unique patients who completed prescription enrollment forms for PYRUKYND increased by 5% from Q4 2024, reaching 234 patients[3] Regulatory and Clinical Developments - The FDA accepted the supplemental New Drug Application (sNDA) for PYRUKYND for thalassemia treatment, with a PDUFA goal date of September 7, 2025[2] - The Phase 3 RISE UP study for mitapivat in sickle cell disease is on track, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026[2] - Agios plans to file an Investigational New Drug Application for AG-236, targeting TMPRSS6 for polycythemia vera, in mid-2025[5] Strategic Focus - The company is advancing its pipeline with a focus on transformative therapies for rare diseases, aiming to create significant value for shareholders[2]

– U.S. Regulatory Filing Under Active Review for Approval of PYRUKYND® (mitapivat) in Thalassemia, with PDUFA Goal Date of September 7, 2025 – – Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease On Track, with Topline Results Expected in Late 2025; Potential U.S. Commercial Launch in 2026 – – Tebapivat Advancing in Clinical Trials for Lower-Risk Myelodysplastic Syndromes (LR-MDS) and Sickle Cell Disease – – PYRUKYND Net Revenue of $8.7 Million in Q1; Cash, Cash Equivalents and Marketable Securities ...

Here are three stocks added to the Zacks Rank #5 (Strong Sell) List today:Agios Pharmaceuticals, Inc. (AGIO) is a biopharmaceutical company. The Zacks Consensus Estimate for its current year earnings has been revised 19.7% downward over the last 60 days.Chegg, Inc. (CHGG) is an individualized learning support provider. The Zacks Consensus Estimate for its current year earnings has been revised 96.8% downward over the last 60 days.Equifax Inc. (EFX) is a data, analytics, and technology company.The Zacks Cons ...

CAMBRIDGE, Mass., March 05, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced the grant of inducement equity awards outside of the Company's 2023 Stock Incentive Plan to its newly appointed Chief Corporate Development & Strategy Officer, Krishnan Viswanadhan. The grants were approved by the Board of Directors effective as of March 5, 2025, as inducements material to M ...

CAMBRIDGE, Mass., March 05, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced the grant of inducement equity awards outside of the Company’s 2023 Stock Incentive Plan to its newly appointed Chief Corporate Development & Strategy Officer, Krishnan Viswanadhan. The grants were approved by the Board of Directors effective as of March 5, 2025, as inducements material to ...

CAMBRIDGE, Mass., March 04, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that its management team is scheduled to present at the following conferences: Leerink Global Healthcare Conference 2025: Fireside chat on Tuesday, March 11, 2025, at 10:50 a.m. ETBarclays 27th Annual Global Healthcare Conference: Fireside chat on Wednesday, March 12, 2025, at 12:30 p.m. ET The live webcast ...

CAMBRIDGE, Mass., Feb. 20, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that its management team is scheduled to present at the TD Cowen 45th Annual Healthcare Conference on Monday, March 3, 2025, at 10:30 a.m. ET. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast w ...