Syndax Pharmaceuticals Receives Approval for REVUFORJ from the FDA
Syndax Pharmaceuticals announced that the FDA has approved REVUFORJ (revumenib) for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. This marks the second FDA approval for REVUFORJ in less than one year, making it the first and only menin inhibitor approved for multiple acute leukemia subtypes in both adults and children.
“We are thrilled to have secured a second indication for REVUFORJ, making it the first and only menin inhibitor that is FDA-approved for multiple acute leukemia subtypes in both adults and children. The breadth of the indicated patient population highlights the compelling and consistent efficacy and tolerability of REVUFORJ in multiple different types of patients,” said Michael A. Metzger, Chief Executive Officer of Syndax.
The approval is supported by efficacy data from the Phase 2 portion of the pivotal AUGMENT-101 trial, in which the rate of complete remission plus partial hematological recovery was 23% (95% CI: 14%, 35%). The median time to response was 2.8 months, with a median duration of 4.5 months. Of 46 patients dependent on red blood cell and/or platelet transfusions at baseline, 17% became independent of both transfusions during any 56-day post-baseline period.
“The expanded FDA approval of REVUFORJ marks a major advancement in the management of acute leukemia patients,” said Joshua F. Zeidner, MD, chief, Leukemia Research at the University of North Carolina, Lineberger Comprehensive Cancer Center. “For the first time, a targeted, oral therapy that is well tolerated and efficacious is approved for R/R NPM1-mutated AML and R/R KMT2A-translocated acute leukemia.”
Mutations in the NPM1 gene are the most common genetic alteration observed in AML, occurring in approximately 30% of adults with the disease. Outcomes are poor for patients who relapse or are refractory to treatment, with high rates of relapse associated with this aggressive form of blood cancer. REVUFORJ was previously approved in 2024 for relapsed or refractory acute leukemia with a KMT2A translocation. On September 18, 2025, revumenib was added to the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology for AML as a category 2A recommended treatment option.
FDA Approves BLENREP for Adults with Relapsed/Refractory Multiple Myeloma
GSK announced that the FDA has approved BLENREP (belantamab mafodotin-blmf) in combination with bortezomib and dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least two prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent. BLENREP is the only anti-BCMA antibody-drug conjugate accessible in community settings, where 70% of patients receive care.
“FDA approval of BLENREP is another significant milestone, providing potential for superior efficacy, including overall survival, to US patients. There is an urgent need for new and novel therapies, as nearly all patients with multiple myeloma experience relapse and re-treating with the same mechanism of action often leads to suboptimal outcomes,” said Tony Wood, Chief Scientific Officer at GSK.
Data from the pivotal DREAMM-7 Phase III trial support the approval. In patients who had two or more prior lines of therapy, BLENREP in combination demonstrated a clinically meaningful 51% reduction in the risk of death (HR 0.49, 95% CI: 0.32-0.76) and tripled median progression-free survival to 31.3 months versus 10.4 months for a daratumumab-based triplet (DVd) (HR 0.31, 95% CI: 0.21-0.47). The safety and tolerability profiles of the BLENREP combination were broadly consistent with those of the individual agents.
“With the approval of BLENREP, we now have a community-accessible BCMA-targeting agent with the potential to improve outcomes for patients following two or more prior lines of treatment, where options are limited. This approval marks an important advance in the US relapsed/refractory treatment landscape,” said Sagar Lonial, MD, Chief Medical Officer at Winship Cancer Institute of Emory University.
Multiple myeloma is the third most common blood cancer globally and is generally considered treatable but not curable. There are approximately 180K new cases of multiple myeloma diagnosed globally each year. The reality for most patients is a relentless cycle of remission and relapse as their disease becomes refractory to treatments. BLENREP is available through a new, streamlined Risk Evaluation and Mitigation Strategy that supports appropriate use and patient safety while reducing administrative burden. GSK is advancing the DREAMM clinical programme with interim efficacy and safety data for BLENREP as a first-line treatment expected in early 2028.
Novartis to Acquire Avidity for $12 Billion
Novartis announced that it has agreed to acquire Avidity Biosciences, a San Diego-based biopharmaceutical company focused on a new class of therapeutics enabling RNA delivery to muscle, for $12 billion in cash. The acquisition strengthens Novartis’s neuroscience franchise with three late-stage programs addressing genetic neuromuscular diseases and advances the Novartis xRNA strategy by adding a scientifically robust, muscle-directed Antibody Oligonucleotide Conjugates platform.
“Avidity’s pioneering AOC platform for RNA therapeutics and its late-stage assets bolster our commitment to delivering innovative, targeted and potentially first-in-class medicines to treat devastating, progressive neuromuscular diseases,” said Vas Narasimhan, CEO of Novartis. “The Avidity team has built robust programs with industry-leading delivery of RNA therapeutics to muscle tissue. We look forward to developing these programs to meaningfully change the trajectory of diseases for patients.”
The proposed acquisition brings three late-stage disease-modifying therapies into the Novartis pipeline: programs for myotonic dystrophy type 1, a rare progressive neuromuscular disorder with a poor prognosis and no disease-modifying therapies; facioscapulohumeral muscular dystrophy, a rare hereditary disorder causing relentless loss of muscle function and progressive disability; and Duchenne muscular dystrophy, a severe, early-onset disease marked by progressive muscle damage and reduced life expectancy.
Under the terms of the transaction, holders of Avidity common stock will receive $72.00 per share in cash at closing, representing a 46% premium to the closing share price on October 24, 2025. Before closing, Avidity will transfer its early-stage precision cardiology programs and collaborations to SpinCo, a newly formed company. Holders of Avidity common stock will receive a distribution of one share of SpinCo for every ten shares of Avidity they hold or a pro rata cash distribution if certain SpinCo assets are sold to a third party.
The acquisition is expected to create an industry-leading pipeline, building on the Novartis expertise in spinal muscular atrophy and commercialization capabilities in genetic neuromuscular diseases. The companies expect the merger to close in the first half of 2026, subject to completion of the SpinCo separation and other customary closing conditions.
BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study
BridgeBio Pharma announced positive topline results from its Phase 3 FORTIFY study evaluating BBP-418 in individuals with limb-girdle muscular dystrophy type 2I/R9, a progressive muscular dystrophy. The study met all primary and secondary interim analysis endpoints, with BBP-418 demonstrating a well-tolerated safety profile consistent with prior studies.
The primary interim analysis endpoint, glycosylated alpha-dystroglycan, increased by 1.8 times from baseline at three months, with improvements sustained through 12 months versus placebo (p<0.0001). Treatment with BBP-418 also resulted in an average 82% reduction from baseline in serum creatine kinase, a marker of muscle damage, showing a statistically significant difference versus placebo at 12 months.
Participants treated with BBP-418 also showed statistically significant and clinically meaningful improvements in key functional measures at 12 months. Ambulatory function, measured by the 100-meter timed test, improved by 0.14 meters per second from baseline and 0.27 meters per second versus placebo. Pulmonary function, assessed by forced vital capacity, rose approximately 3% from baseline and 5% relative to placebo.
“For a condition with no targeted treatment options and an inexorable march toward loss of ambulation, breathing, and independence, the prospect of BBP-418 offers a compelling new hope,” noted market analysts reviewing the data. The treatment’s effectiveness held up across different genetic backgrounds, benefiting both the common L276I genotype and other FKRP mutations.
LGMD2I/R9 is caused by bi-allelic partial loss-of-function of the fukutin-related protein gene, resulting in hypoglycosylation of alpha-dystroglycan and progressive muscle damage. BBP-418 is an oral substrate supplementation intended to saturate the partially functional FKRP enzyme, driving increased glycosylation of alpha-dystroglycan and potentially stabilizing or improving muscle integrity. The compound already holds Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA, which could lead to a priority review voucher if approval follows. BridgeBio intends to file a New Drug Application with the FDA in the first half of 2026.
Eli Lilly to Acquire Adverum Biotechnologies
Eli Lilly and Company announced that it has agreed to acquire Adverum Biotechnologies, a clinical-stage company pioneering intravitreal gene therapy for eye diseases. Under the terms of the agreement, Lilly will offer Adverum $3.56 per share in cash, including an additional $8.91 in milestone payments contingent on US approval of ixo-vec within seven years and achieving more than $1 billion in annual global sales within ten years. This brings the total consideration to $12.47 per share, valuing the deal at a possible $261.7 million.
“We are excited about the potential to join Lilly, with a proven track record in the discovery, development, and commercialization of innovative medicines for chronic and age-related conditions,” said Laurent Fischer, MD, CEO of Adverum. “We share Lilly’s commitment to healthy aging and genetic medicines innovation. Their scientific depth and global reach offer the opportunity to accelerate our vision to deliver a transformative one-and-done therapy that can potentially restore and preserve vision for millions of patients living with wAMD.”
As part of the deal, Lilly will acquire Adverum’s lead candidate ixo-vec (ixoberogene soroparvovec). This intravitreal gene therapy advanced into a Phase III trial for the treatment of wet age-related macular degeneration in March 2025. Ixo-vec is designed as a one-time intravitreal injection that delivers continuous aflibercept expression, aiming to reduce the treatment burden associated with frequent anti-VEGF injections. The therapy has been granted fast-track and regenerative medicine advanced therapy designations by the FDA, as well as PRIME and innovation passport designations from European and UK regulators.
For Adverum, the potential buyout from Lilly provides critical financial respite. The biotech has been struggling for cash in recent times, holding $44.4 million in July 2025. Lilly will provide Adverum with up to $65 million in bridge financing to support ongoing development ahead of the expected fourth-quarter 2025 closing. The acquisition aligns with Lilly’s strategy to expand in genetic medicines and ophthalmology, complementing its gene therapy portfolio, which includes programs for cardiovascular diseases, RNA-based therapies, metabolic diseases, and genetic hearing loss.
