Fulcrum Therapeutics

Epicrispr Biotechnologies Secures $68 Million to Advance First Epigenetic Therapy for FSHD

2 months ago

• Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD). • New Zealand's Medsafe has approved Epicrispr's clinical trial application, with the first-in-human study of EPI-321 expected to begin in 2025 to evaluate its safety and biological activity in adults with FSHD. • EPI-321 uses CRISPR technology to silence the DUX4 gene that drives FSHD progression and has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.

Fulcrum's Pociredir SCD Trial Advances with Key Data Readouts Expected in 2025

3 months ago

• Fulcrum Therapeutics has successfully enrolled 10 patients in the 12mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease, with data expected by mid-2025. • The company maintains a strong financial position with $241 million in cash reserves, providing runway into at least 2027 to support ongoing clinical development programs. • Pociredir, an oral EED inhibitor, shows promise in increasing fetal hemoglobin levels and could potentially transform SCD treatment by offering a differentiated oral therapy option.

Fulcrum's Oral SCD Therapy Pociredir Advances in Phase 1b Trial with Key Data Expected Mid-2024

4 months ago

Fulcrum Therapeutics is progressing with the PIONEER Phase 1b trial of pociredir (FTX-6058), an oral therapy for sickle cell disease that aims to reactivate fetal hemoglobin production. Initial data has shown promising results with dose-dependent increases in fetal hemoglobin levels, and new data from higher dosing cohorts is expected by mid-2024.

C4 Therapeutics Announces 2025 Milestones for Oncology-Focused Degrader Medicines

4 months ago

• C4 Therapeutics is set to advance clinical development of cemsidomide, with Phase 1 data expected in late 2025 for multiple myeloma and non-Hodgkin’s lymphoma. • CFT1946, targeting BRAF V600 mutations in solid tumors, is progressing through Phase 1, with data readouts anticipated in the second half of 2025. • Phase 1 data from CFT8919, aimed at EGFR L858R-mutated non-small cell lung cancer, will inform future development plans outside of China. • C4 Therapeutics' cash runway is projected to fund operations into 2027, supporting ongoing research and clinical programs.

Spero Therapeutics' Tebipenem HBr Phase 3 Trial Enrollment Nears Completion

4 months ago

• Spero Therapeutics' Phase 3 PIVOT-PO trial for Tebipenem HBr has surpassed 60% enrollment as of December 31, 2024, and is on track for completion in the second half of 2025. • The PIVOT-PO trial evaluates Tebipenem HBr's efficacy in treating complicated urinary tract infections (cUTI) and acute pyelonephritis (AP) in hospitalized adult patients. • Spero Therapeutics reported cash and cash equivalents of $52.9 million as of December 31, 2024, expected to fund operations into mid-2026. • Interim leadership changes were announced, with Esther Rajavelu appointed Interim President and CEO, and Frank Thomas as Chairman of the Board.

Accent Therapeutics Doses First Patient in Phase 1/2 Trial of DHX9 Inhibitor ATX-559

5 months ago

• Accent Therapeutics initiated a Phase 1/2 clinical trial for ATX-559, a first-in-class DHX9 inhibitor, focusing on BRCA1/2-deficient breast cancer and MSI-H/dMMR solid tumors. • The trial aims to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ATX-559 in solid tumor patients. • Accent Therapeutics anticipates its second program targeting KIF18A to enter clinical trials in the first half of 2025, aimed at treating patients with chromosomally instable tumors. • Serena Silver, Ph.D., has been promoted to Chief Scientific Officer, succeeding Robert A. Copeland, Ph.D., who will retire effective December 31, 2024.

Sarepta Therapeutics and Arrowhead Pharmaceuticals Announce RNAi Collaboration to Treat Rare Genetic Diseases

6 months ago

• Sarepta Therapeutics and Arrowhead Pharmaceuticals have entered into a global licensing and collaboration agreement to develop RNAi-based treatments for rare genetic diseases. • Sarepta will gain rights to several of Arrowhead’s clinical-stage programs, including treatments for facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, idiopathic pulmonary fibrosis, and spinocerebellar ataxia 2. • Arrowhead will receive an upfront payment of $500 million, a $325 million equity investment, and is eligible for up to $10 billion in milestone payments and royalties. • The collaboration diversifies Sarepta’s portfolio, complementing its gene therapy and gene editing efforts with Arrowhead's RNAi technology, while extending Arrowhead's cash runway into 2028.

AbbVie Seeks Accelerated FDA Approval for Telisotuzumab Vedotin in c-Met Overexpressing NSCLC

8 months ago

• AbbVie has submitted a Biologics License Application (BLA) to the FDA for telisotuzumab vedotin to treat NSCLC with c-Met protein overexpression. • The BLA is supported by Phase 2 LUMINOSITY trial data, which showed promising overall response rates in c-Met high and intermediate patients. • Telisotuzumab vedotin, an antibody-drug conjugate, targets c-Met-overexpressing tumors and could be a first-in-class therapy if approved. • The FDA previously granted Breakthrough Therapy Designation to telisotuzumab vedotin, and a Phase 3 trial is underway to confirm its efficacy.

Pfizer Withdraws Sickle Cell Disease Drug Oxbryta Globally Due to Safety Concerns

8 months ago

• Pfizer has voluntarily withdrawn Oxbryta (voxelotor) from all global markets due to safety concerns, citing an imbalance in vaso-occlusive crises and fatal events. • The decision follows a review of clinical data that indicated the overall benefit of Oxbryta no longer outweighs the risks for sickle cell disease patients. • Regulatory agencies, including the EMA and FDA, have alerted healthcare professionals and patients about the withdrawal, advising discussions on alternative treatments. • The withdrawal impacts ongoing clinical trials and expanded access programs, leaving the sickle cell community seeking alternative treatment options and raising concerns about trust.

Apellis' Syfovre Faces Third Rejection by EU's CHMP for Geographic Atrophy

8 months ago

• Apellis Pharmaceuticals' Syfovre (pegcetacoplan) received a third negative opinion from the CHMP for treating geographic atrophy (GA) in the EU. • The decision follows previous rejections and a procedural reset due to a European Court of Justice ruling on EMA's expert group organization. • Syfovre is approved in the U.S. for GA, demonstrating strong initial uptake with $292.1 million in sales in the first half of 2024. • Apellis continues to seek approval for pegcetacoplan in other countries, while also marketing it as Empaveli/Aspaveli for paroxysmal nocturnal hemoglobinuria.

FDA Approves Keytruda for First-Line Treatment of Malignant Pleural Mesothelioma

8 months ago

• The FDA has approved Keytruda (pembrolizumab) in combination with pemetrexed and platinum chemotherapy as a first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma. • The approval was based on the Phase 2/3 KEYNOTE-483 trial, which demonstrated a statistically significant improvement in overall survival compared to chemotherapy alone. • Patients receiving Keytruda plus chemotherapy had a median overall survival of 17.3 months, compared to 16.1 months for those receiving chemotherapy alone, reducing the risk of death by 21%. • The Keytruda regimen also showed significant improvements in progression-free survival and overall response rate, offering a new treatment option for this aggressive cancer.

Fulcrum Therapeutics' Losmapimod Fails Phase 3 Trial in FSHD, Development Suspended

8 months ago

• Fulcrum Therapeutics' Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) did not meet its primary endpoint, showing no significant improvement in reachable workspace compared to placebo. • Secondary endpoints, including muscle fat infiltration and shoulder strength, also failed to achieve statistical significance in the losmapimod arm. • Fulcrum plans to suspend the losmapimod program and shift focus to pociredir for sickle cell disease and other early-stage programs. • The company's stock price plummeted following the announcement, raising concerns about its future prospects despite a solid cash position.

Corvus Pharmaceuticals Launches Phase 3 Trial of Soquelitinib for Relapsed/Refractory PTCL

8 months ago

• Corvus Pharmaceuticals has initiated a Phase 3 clinical trial for soquelitinib in patients with relapsed/refractory peripheral T-cell lymphoma (PTCL). • The trial is a randomized, controlled study comparing soquelitinib to standard of care chemotherapy, aiming to enroll approximately 150 patients across multiple global sites. • Soquelitinib, a first-in-class ITK inhibitor, has shown durable anti-tumor activity and a novel mechanism of action enhancing the host anti-tumor response in earlier trials. • The primary endpoint of the trial is progression-free survival, with secondary endpoints including overall survival and objective response rate.