To support the development and launch of dordaviprone, Chimerix has secured a $30 million credit facility with Silicon Valley Bank (SVB), a division of First-Citizens Bank. This facility provides access to additional capital during the upcoming investment cycle and ensures the company is well-positioned for the potential launch. “We also entered into a credit facility of up to $30 million with Silicon Valley Bank, providing access to additional capital during this critical investment cycle and helping ensure dordaviprone availability to as many patients as possible, as quickly as possible, if approved,” added Michelle LaSpaluto, Chief Financial Officer of Chimerix.
Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma, further underscoring its potential impact on a vulnerable patient population. The company has also applied for a Rare Pediatric Disease Priority Review Voucher (PRV) as part of the NDA submission. With the robust support of strategic partnerships and enhanced operational readiness, Chimerix is committed to bringing dordaviprone to market and improving outcomes for patients facing this devastating disease.
NMD Pharma Receives FDA Orphan Drug Designation for NMD670 in Charcot-Marie-Tooth Disease
NMD Pharma A/S, announced that the FDA has granted Orphan Drug Designation (ODD) for NMD670, its skeletal muscle-specific ClC-1 inhibitor, for the treatment of Charcot-Marie-Tooth disease (CMT). This is the second ODD for NMD670, which previously received the designation for generalized myasthenia gravis (gMG) in 2022.
CMT, a hereditary neuropathy affecting approximately 136,000 individuals in the U.S. and over 3 million worldwide, is characterized by muscle weakness, atrophy, fatigue, and sensory deficits that severely impact quality of life. With no approved therapies available, NMD670 aims to address these unmet needs by targeting muscle weakness and fatigue associated with the disease. “This designation underscores the urgent need for effective treatments and highlights the therapeutic potential of our ClC-1 inhibitor approach,” said Thomas Holm Pedersen, CEO of NMD Pharma.
NMD670 is currently being evaluated in the SYNAPSE-CMT Phase II clinical trial, which began in November 2024. The trial involves 80 adult patients with genetically confirmed CMT Type 1 or Type 2 subtypes, testing a twice-daily oral dose over 21 days across sites in the U.S. and Europe. Additional information and trial details can be found under identifier NCT06482437 at clinicaltrials.gov or by contacting NMD Pharma.
In addition to CMT, NMD Pharma is advancing NMD670 through three global trials targeting rare neuromuscular diseases, including a Phase II study for spinal muscular atrophy (SMA) Type 3 and a Phase IIb study in gMG. Data readouts from these studies are expected between the second half of 2025 and the first half of 2026.
Verastem Oncology Announces FDA Acceptance and Priority Review of Avutometinib and Defactinib for KRAS Mutant Low-Grade Serous Ovarian Cancer
Verastem Oncology announced that the FDA has accepted the New Drug Application (NDA) for avutometinib, a RAF/MEK clamp, combined with defactinib, a FAK inhibitor, for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with KRAS mutations. The NDA, granted Priority Review, has a PDUFA action date of June 30, 2025. No advisory committee meeting is planned for this application.
“This marks a pivotal moment for patients with recurrent KRAS mutant LGSOC, a condition lacking approved treatments,” said Dan Paterson, CEO of Verastem Oncology. “We’re preparing for a potential commercial launch in mid-2025 and are committed to addressing this overlooked disease.”
The submission was based on results from the Phase II RAMP 201 trial, presented at the 2024 IGCS Annual Meeting, which showed durable responses and good tolerability in patients treated with the avutometinib-defactinib combination. Supportive data from the FRAME Phase I trial were also included.
To expand on this progress, Verastem is enrolling patients in the Phase III RAMP 301 trial, which will serve as a confirmatory study and potentially support an indication for recurrent LGSOC regardless of KRAS mutation status.
Capricor Therapeutics Submits Biologics License Application for Deramiocel in Duchenne Muscular Dystrophy
Capricor Therapeutics has completed the submission of its Biologics License Application (BLA) to the FDA for full approval of deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The BLA is supported by data from the Phase II HOPE-2 trials and natural history comparisons, demonstrating deramiocel’s potential to attenuate DMD-related cardiac issues.
“This BLA submission is a milestone for Capricor and the DMD community, bringing us closer to delivering a transformational therapy to patients,” said Linda Marbán, Ph.D., CEO of Capricor. “We are eager to collaborate with the FDA during the review process to advance this therapy toward approval.”
Capricor has requested Priority Review, which could reduce the review timeline from 10 to 6 months. A $10 million milestone payment from Nippon Shinyaku Co., Ltd., under their U.S. Commercialization and Distribution Agreement, is tied to this achievement.
Deramiocel holds Orphan Drug Designation from the FDA and EMA, RMAT Designation in the U.S., and ATMP Designation in Europe. FDA approval could also make Capricor eligible for a Priority Review Voucher, given the therapy’s rare pediatric disease designation.
Axsome Therapeutics Completes Phase III Clinical Program of AXS-05 for Alzheimer’s Disease Agitation
Axsome Therapeutics has announced the successful completion of its Phase III clinical program evaluating AXS-05 (dextromethorphan-bupropion) for treating agitation associated with Alzheimer’s disease. The program included multiple trials, with the ACCORD-2 trial meeting its primary endpoint by significantly delaying the time to relapse of agitation compared to placebo. Patients receiving AXS-05 experienced a 3.6-fold lower risk of relapse (hazard ratio: 0.276, p=0.001). The key secondary endpoints were also met, with AXS-05 reducing the relapse rate (8.4% for AXS-05 vs. 28.6% for placebo, p=0.001) and preventing worsening of disease severity. These results reinforce the therapeutic potential of AXS-05 in addressing agitation, a highly distressing symptom for Alzheimer’s patients and their caregivers.
The ADVANCE-2 trial, another Phase III study, showed numerical improvements favoring AXS-05 over placebo across most endpoints, although the primary endpoint of CMAI score reduction did not achieve statistical significance. Despite this, the trial contributed valuable safety data. In both short-term and long-term studies, AXS-05 was well-tolerated, with no increased risks of sedation, cognitive decline, or mortality. Adverse events were minimal and included dizziness and headache. Long-term safety assessments demonstrated consistent tolerability over extended use, with over 300 patients treated for at least six months and over 100 for at least 12 months.
Axsome plans to submit a New Drug Application (NDA) for AXS-05 to the FDA in the second half of 2025. This follows the drug’s Breakthrough Therapy designation for Alzheimer’s disease agitation, granted in 2020 based on earlier positive trial results. The regulatory submission will leverage data from four Phase III trials, including three that demonstrated statistically significant efficacy. According to Jeffrey Cummings, MD, ScD, the robust clinical data highlight AXS-05’s potential to address a significant unmet need in Alzheimer’s disease care by reducing agitation and improving disease severity.
CEO Herriot Tabuteau, MD, expressed optimism about the drug’s ability to provide meaningful benefits to patients and their families. The company aims to bring this innovative therapy to market, marking a crucial step toward improving the quality of life for those affected by Alzheimer’s disease agitation. With its comprehensive clinical evidence and favorable safety profile, AXS-05 has the potential to become a pivotal treatment for this challenging condition.
