Latest news with #Phase1study
Yahoo
5 days ago
- Business
- Yahoo
Schrödinger Announces Discontinuation of SGR-2921 Program
NEW YORK, August 14, 2025--(BUSINESS WIRE)--Schrödinger, Inc. (Nasdaq: SDGR) today announced the discontinuation of the clinical development program for SGR-2921, its CDC7 inhibitor, which was being evaluated in a Phase 1 dose-escalation study in patients with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes. Despite early evidence of monotherapy activity observed in the Phase 1 study, based on the profile observed to date, including two emergent events where SGR-2921 was considered to have contributed to two deaths in patients with AML, Schrödinger believes the path to development as a combination therapy would be difficult to pursue. The Phase 1 study was supported by preclinical data which had demonstrated that CDC7 inhibition resulted in monotherapy and combination anti-leukemic responses in patient-derived AML models, suggesting that SGR-2921 could ultimately be used in combination with standard of care agents. "Patient safety is our first priority, and in light of two treatment-related deaths in the Phase 1 dose-escalation study, we have made the decision to discontinue further development of SGR-2921. While disappointing given the early clinical activity observed, we believe this is the right decision for patients," stated Margaret Dugan, M.D., chief medical officer at Schrödinger. "We had hoped to advance this investigational agent for acute myeloid leukemia as relapse rates are high, the disease progresses rapidly and there are limited therapies available. We are very grateful to the investigators, patients and families who have participated in this clinical study." About SchrödingerSchrödinger is transforming molecular discovery with its computational platform, which enables the discovery of novel, highly optimized molecules for drug development and materials design. Schrödinger's software platform is built on more than 30 years of R&D investment and is licensed by biotechnology, pharmaceutical and industrial companies, and academic institutions around the world. Schrödinger also leverages the platform to advance a portfolio of collaborative and proprietary programs. Founded in 1990, Schrödinger has approximately 800 employees operating from 15 locations globally. To learn more, visit follow us on LinkedIn and Instagram, or visit our blog, Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 including, but not limited to the benefits of its computational platform. Statements including words such as "aim," "anticipate," "believe," "contemplate," "continue," "could," "estimate," "expect," "goal," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," "would" and statements in the future tense are forward-looking statements. These forward-looking statements reflect Schrödinger's current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to the company and on assumptions the company has made. Actual results may differ materially from those described in these forward-looking statements and are subject to a variety of assumptions, uncertainties, risks and important factors that are beyond Schrödinger's control, including the uncertainties inherent in drug development, such as the conduct of research activities and the timing of and its ability to initiate and complete preclinical studies and clinical trials, whether results from preclinical and early clinical studies will be predictive of the results of later preclinical studies and clinical trials, whether initial data from clinical results will be predictive of the final results of the clinical trials, uncertainties associated with the regulatory review of clinical trials, and the ability to retain and hire key personnel and other risks detailed under the caption "Risk Factors" and elsewhere in the company's Securities and Exchange Commission filings and reports, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with the Securities and Exchange Commission on August 6, 2025, as well as future filings and reports by the company. Any forward-looking statements contained in this press release speak only as of the date hereof. Except as required by law, Schrödinger undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events, changes in expectations or otherwise. View source version on Contacts Jaren Madden (Investors and Media) 617-286-6264 Matthew Luchini (Investors) 917-719-0636 Allie Nicodemo (Media) 617-356-2325
Yahoo
12-08-2025
- Health
- Yahoo
Marea Therapeutics Enrolls First Participant in Phase 1 Clinical Study Evaluating MAR002 for Acromegaly
SOUTH SAN FRANCISCO, Calif., August 12, 2025--(BUSINESS WIRE)--Marea Therapeutics, Inc., a clinical-stage biotechnology company harnessing the latest advances in human genetics to develop first-in-class, next-generation medicines for cardioendocrine diseases, today announced that the first participant has been enrolled in Marea's Phase 1 study evaluating MAR002 for the treatment of acromegaly. Acromegaly is a chronic, rare, systemic disease most often caused by a growth hormone (GH)-secreting pituitary adenoma, leading to the overproduction of insulin-like growth factor 1 (IGF-1) which mediates the characteristic somatic overgrowth and multisystem involvement observed in affected individuals. The primary objective of medical therapy for acromegaly is to normalize IGF-1 levels according to age and sex. "Dosing our first participant in this Phase 1 study of MAR002 is a critical milestone for Marea Therapeutics and brings us closer to addressing the significant unmet needs of patients with acromegaly," said Josh Lehrer, M.D., chief executive officer of Marea Therapeutics. "We believe MAR002's unique properties, including an allosteric mechanism of action, could make it a differentiated and optimal medical therapy for acromegaly, increasing the effectiveness and reducing the burden associated with current therapies." The MAR-201 study is a first-in-human (FIH), randomized, blinded, parallel-group, placebo-controlled single ascending dose (SAD) study in healthy men. Key objectives of the study include: Primary Objective: To evaluate the safety and tolerability of subcutaneous (SC) administration of MAR002, assessed by treatment-emergent adverse events (TEAEs), vital signs, physical examinations, safety laboratories, and electrocardiograms (ECGs). Secondary Objective: To evaluate the pharmacokinetics (PK) of SC administration of MAR002 Exploratory Objectives: To evaluate the effect of SC administration of MAR002 on serum IGF-1 levels (pharmacodynamics [PD]/efficacy) and to assess the immunogenicity. About MAR002 MAR002 is a potent and selective half-life-extended, allosteric, human monoclonal growth hormone receptor antagonist (GHRA) antibody being developed for the treatment of acromegaly. The in vivo PK and PD properties of MAR002 are predictable and typical of a half-life extended human antibody, showing a long duration of action compatible with infrequent subcutaneous dose administration in humans. These characteristics support its potential to offer an effective and convenient treatment for patients with acromegaly. About Acromegaly Acromegaly is an orphan disease characterized by the excess secretion of growth hormone (GH) from a benign pituitary adenoma. Acromegaly affects approximately 30,000 patients in the U.S. If left untreated, acromegaly is highly morbid, leading to significant comorbidities such as GH-induced insulin resistance and diabetes, and serious cardiovascular pathology. The median lifespan of patients can be shortened by 10 years without effective therapy, and incomplete IGF-1 normalization is associated with increased mortality. Despite its severity, acromegaly is often under or misdiagnosed, with an average time from symptom onset to diagnosis of approximately eight years. The current treatment paradigm for acromegaly often involves surgery, performed in over 90% of patients, which achieves remission in about 50% of cases, though this can degrade over time. Medical therapy is required for approximately 65% (around 20,000 in the U.S.) of patients during their disease journey. Current medical treatments include somatostatin receptor ligands (SRLs) and growth hormone receptor antagonist (GHRA) pegvisomant. However, most patients do not achieve biochemical control with existing therapies. About Marea Therapeutics Marea Therapeutics is a clinical-stage biotechnology company harnessing the latest advances in human genetics to develop first-in-class, next-generation medicines for cardioendocrine diseases. The company's lead therapy, MAR001, is in Phase 2 clinical development for adults with metabolic dysfunction and high risk for atherosclerotic cardiovascular disease. The company is also advancing MAR002 for the treatment of acromegaly. To learn more, please visit and follow us on LinkedIn and X. View source version on Contacts Media:1ABKatie Englemankatie@ Investors:Meru AdvisorsLauren Glaserlglaser@ Sign in to access your portfolio
Yahoo
07-08-2025
- Business
- Yahoo
CytomX Therapeutics Announces Second Quarter 2025 Financial Results and Provides Business Update
- May 12, 2025 announcement of positive interim data from ongoing Phase 1 study of CX-2051 (EpCAM Antibody Drug Conjugate) in patients with advanced colorectal cancer (CRC) - - CX-2051 Phase 1 dose expansions on track for data update in Q1 2026. Phase 2 study initiation in CRC anticipated 1H 2026 - - Combination dose escalation initiated of CX-801 masked interferon alpha-2b with KEYTRUDA® (pembrolizumab) in patients with advanced melanoma - - Completed $100 million underwritten offering of common stock - - Company to host conference call today at 5 p.m. EST / 2 p.m. PST - SOUTH SAN FRANCISCO, Calif., Aug. 07, 2025 (GLOBE NEWSWIRE) -- CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of masked, conditionally activated biologics, today announced second quarter 2025 financial results and provided a business update. 'Q2 2025 was an exciting quarter for CytomX as we announced positive initial clinical results for CX-2051 in advanced colorectal cancer, a very challenging disease to treat. Our data highlight CX-2051's intentional design as a first-in-class, masked EpCAM-directed ADC with potential to improve upon the standard of care in late-line CRC. We are also delighted to have completed a financing with top-tier investors that enables CytomX to rapidly advance the CX-2051 development program. Looking ahead, we remain highly focused on our next anticipated CX-2051 clinical data update in Q1 2026 and to potentially launching a Phase 2 study in the first half of 2026,' said Sean McCarthy, chief executive officer and chairman of CytomX. Q2 2025 Pipeline Program Updates: CX-2051 (EpCAM PROBODY Topo-1 ADC) Announced positive interim data from ongoing Phase 1 dose escalation study of first-in-class EpCAM Antibody Drug Conjugate (CX-2051) in patients with advanced colorectal cancer (CRC). Initiated CX-2051 dose expansions at the 7.2 mg/kg, 8.6 mg/kg, and 10 mg/kg doses, administered every three weeks (Q3W). Phase 1 data update in advanced CRC in approximately 70 patients is expected by Q1 2026. Planning underway for CX-2051 Phase 2 study initiation in advanced, late-line CRC in 1H 2026. Potential to initiate CX-2051 combination studies in earlier lines of CRC therapy in 2026. Evaluation ongoing of multiple non-CRC, EpCAM-expressing tumor indications for potential future CX-2051 development. CX-801 (PROBODY Interferon alpha-2b) Phase 1 dose escalation of CX-801 monotherapy continues. Preliminary tumor biomarker, pharmacodynamic (PD) and pharmacokinetic (PK) data evaluating the initial molecular performance of CX-801 monotherapy in the ongoing Phase 1 study are expected in the fourth quarter of 2025. In May 2025, Phase 1 dose escalation of CX-801 in combination with KEYTRUDA® was initiated. Initial clinical data for the combination therapy in advanced melanoma is anticipated in 2026. KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA Corporate and Financial: Financial: Completed $100 million ($93.4 million net proceeds) underwritten offering of common stock. CytomX ended the second quarter of 2025 with $158.1 million of cash, cash equivalents and investments with expected cash runway to the second quarter of 2027. Research collaborations: Presented preclinical data for mRNA encoded masked IL-12 molecule in collaboration with Moderna at AACR Annual Meeting showing potent anti-tumor activity with significantly enhanced tolerability vs. unmasked IL-12 molecule. Multiple drug discovery programs continue across our research collaborations with a focus on bispecific immunotherapies, including T-cell engagers. CytomX has research collaborations with Bristol Myers Squibb, Amgen, Astellas, Regeneron, and Moderna. Second Quarter 2025 Financial Results: Cash, cash equivalents and investments totaled $158.1 million as of June 30, 2025, compared to $79.9 million as of March 31, 2025. Total revenue was $18.7 million for the quarter ended June 30, 2025, compared to $25.1 million for the quarter ended June 30, 2024. The decrease in revenue was driven primarily by the completion of our performance obligations in the Bristol Myers Squibb collaboration, the decision not to further develop the CX-904 program in the Amgen agreement, and a decrease in Moderna activities due to Moderna budget considerations. Total operating expense in the second quarter of 2025 was $19.9 million compared to $33.6 million in the second quarter of 2024, a decrease of $13.7 million. Research and development expenses were $13.3 million for the three months ended June 30, 2025, a decrease of $11.9 million compared to the corresponding period of 2024. Reduced research and development expenses were primarily due to a one-time milestone payment of $5M to Immunogen for the first patient dosed in Phase 1 with CX-2051 in Q2 2024, a reduction in CX-904 spend due to program de-prioritization in 2025, and reduced research expenses following the Q1 2025 restructuring. General and administrative expenses were $6.6 million for the three months ended June 30, 2025, a decrease of $1.8 million compared to the corresponding period of 2024. The decrease in general and administrative expenses was primarily driven by personnel costs as well as patent and legal expenses. About CytomX TherapeuticsCytomX is a clinical-stage, oncology-focused biopharmaceutical company focused on developing novel conditionally activated, masked biologics designed to be localized to the tumor microenvironment. By pioneering a novel pipeline of localized biologics, powered by its PROBODY® therapeutic platform, CytomX's vision is to create safer, more effective therapies for the treatment of cancer. CytomX's multi-modality technology platform has produced therapeutic candidates across multiple treatment modalities including antibody-drug conjugates (ADCs), T-cell engagers, and immune modulators such as cytokines. CytomX's current clinical-stage pipeline includes CX-2051 and CX-801. CX-2051 is a masked, conditionally activated ADC directed toward epithelial cell adhesion molecule (EpCAM), armed with a topoisomerase-1 inhibitor payload. CX-2051 has potential applicability across multiple EpCAM-expressing epithelial cancers, including CRC, and was discovered in collaboration with ImmunoGen. CX-801 is a masked interferon alpha-2b PROBODY® cytokine with broad potential applicability in traditionally immuno-oncology sensitive as well as insensitive (cold) tumors. CytomX has established strategic collaborations with multiple leaders in oncology, including Amgen, Astellas, Bristol Myers Squibb, Regeneron and Moderna. For more information about CytomX and how it is working to make conditionally activated treatments the new standard-of-care in the fight against cancer, visit and follow us on LinkedIn and X (formerly Twitter). CytomX Therapeutics Forward-Looking StatementsThis press release includes forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that are difficult to predict, may be beyond our control, and may cause the actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied in such statements, including those related to CX-2051. Accordingly, you should not rely on any of these forward-looking statements, including those relating to the potential benefits, safety and efficacy or progress of CytomX's or any of its collaborative partners' product candidates, including CX-2051 and CX-801, the potential benefits or applications of CytomX's PROBODY® therapeutic platform, CytomX's or its collaborative partners' ability to develop and advance product candidates into and successfully complete clinical trials, including the ongoing and planned clinical trials of CX-2051 and CX-801 and the timing of initial and ongoing data availability for our clinical trials, including CX-2051 and CX-801, and other development milestones. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: the unproven nature of CytomX's novel PROBODY® therapeutic technology; uncertainties around the Company's ability to raise sufficient funds to carry out its planned research and development; CytomX's clinical trial product candidates are in the initial stages of clinical development and its other product candidates are currently in preclinical development, and the process by which preclinical and clinical development could potentially lead to an approved product is long and subject to significant risks and uncertainties, including the possibility that the results of preclinical research and early clinical trials, including initial CX-2051 results, may not be predictive of future results; the possibility that CytomX's clinical trials will not be successful; the possibility that current preclinical research may not result in additional product candidates; CytomX's dependence on the success of CX-2051 and CX-801; CytomX's reliance on third parties for the manufacture of the Company's product candidates; possible regulatory developments in the United States and foreign countries, including China and the European Union; and the risk that we may incur higher costs than expected for research and development or unexpected costs and expenses. Additional applicable risks and uncertainties include those relating to our preclinical research and development, clinical development, and other risks identified under the heading "Risk Factors" included in CytomX's Quarterly Report on Form 10-Q filed with the SEC on August 7, 2025. The forward-looking statements contained in this press release are based on information currently available to CytomX and speak only as of the date on which they are made. CytomX does not undertake and specifically disclaims any obligation to update any forward-looking statements, whether as a result of any new information, future events, changed circumstances or otherwise. PROBODY is a U.S. registered trademark of CytomX Therapeutics, Inc. All other trademarks are the properties of their respective owners. Company Contact:Chris OgdenSVP, Chief Financial Officercogden@ Investor Contact:Precision AQ (formerly Stern Investor Relations)Stephanie Media Contact:Redhouse CommunicationsTeri Dahlmanteri@ THERAPEUTICS, STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME(in thousands, except share and per share data)(Unaudited) Three Months Ended June 30, 2025 2024 Revenues $ 18,658 $ 25,115 Operating expenses: Research and development 13,322 25,172 General and administrative 6,622 8,395 Total operating expenses 19,944 33,567 Loss from operations (1,286 ) (8,452 ) Interest income 1,178 1,971 Other (expense) income, net 17 (2 ) Loss before income taxes (91 ) (6,483 ) Provision for income taxes 63 51 Net Loss (154 ) (6,534 ) Other comprehensive loss: Unrealized gain on investments, net of tax 34 6 Total comprehensive loss $ (120 ) $ (6,528 Net loss per share: Basic $ (0.00 ) $ (0.08 ) Diluted $ (0.00 ) $ (0.08 ) Shares used to compute net income per share Basic 129,075,546 84,880,632 Diluted 129,075,546 84,880,632 CYTOMX THERAPEUTICS, BALANCE SHEETS(in thousands) June 30, December 31, 2025 2024 (unaudited) (1) Assets Current assets: Cash and cash equivalents $ 49,041 $ 38,052 Short-term investments 109,046 62,571 Accounts receivable 1,869 3,103 Prepaid expenses and other current assets 4,665 3,579 Total current assets 164,621 107,305 Property and equipment, net 1,946 2,467 Intangible assets, net 510 583 Goodwill 949 949 Restricted cash 1,028 1,027 Operating lease right-of-use asset 5,947 8,136 Other assets 56 66 Total assets $ 175,057 $ 120,533 Liabilities and Stockholders' Equity (Deficit) Current liabilities: Accounts payable $ 227 $ 1,088 Accrued liabilities 11,503 12,338 Operating lease liabilities - short term 5,443 5,145 Deferred revenue, current portion 22,050 67,201 Total current liabilities 39,223 85,772 Deferred revenue, net of current portion 10,241 26,862 Operating lease liabilities - long term 1,442 4,240 Other long term liabilities 4,241 4,115 Total liabilities 55,147 120,989 Commitments and contingencies Stockholders' equity (deficit): Convertible preferred stock — — Common stock 2 1 Additional paid-in capital 788,083 691,095 Accumulated other comprehensive (loss) income 33 27 Accumulated deficit (668,208 ) (691,579 ) Total stockholders' equity (deficit) 119,910 (456 ) Total liabilities and stockholders' equity (deficit) $ 175,057 $ 120,533 __________________(1) The condensed balance sheet as of December 31, 2024 was derived from the audited financial statements included in the Company's Annual Report on Form 10-K for the year ended December 31, in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
21-07-2025
- Business
- Yahoo
Sarepta Stock Keeps Sliding as Company Rejects FDA Call to Withdraw Drug
Key Takeaways Sarepta Therapeutics rejected the Food and Drug Administration's call to stop shipments of its muscular dystrophy drug. The FDA request came after a third patient in a Phase 1 study died while taking a similar treatment. Shares of the biopharma firm slid Monday, extending last week's Therapeutics (SRPT) shares continued to slide Monday after the biopharmaceutical firm refused a Food and Drug Administration (FDA) request to stop distributing its Elevidys treatment for muscular dystrophy. The FDA called for the halt after a third patient suffering from a form of muscular dystrophy taking a similar gene therapy to Elevidys in a Phase 1 study died of liver failure, possibly related to the drug. The news of the patient death sent shares of Sarepta plunging Friday. Elevidys has already been approved to treat both ambulatory and non-ambulatory patients with Duchenne muscular dystrophy. Sarepta voluntarily stopped shipments for non-ambulatory patients last month following a second death during the study to gauge the efficacy of the drug for Limb-Girdle muscular dystrophy. The company noted the third patient who died suffered from non-ambulant Limb-Girdle muscular dystrophy. However, Sarepta said that because its comprehensive scientific interpretation of the data 'shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population.' Sarepta said it's looking to 'continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care." Shares of Sarepta were down about 3% in recent trading. They've lost nearly 90% of their value since the start of the year. Read the original article on Investopedia Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
