Beam Therapeutics Secures FDA Orphan Drug Designation for BEAM-302 in AATD

Beam Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its investigational therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeting lipid nanoparticle (LNP) formulation containing a guide RNA and an mRNA encoding a base editor designed to correct the underlying genetic mutation responsible for AATD. The designation provides Beam with potential development incentives, including tax credits, user fee exemptions, and up to seven years of market exclusivity upon approval.

“Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “This recognition by the FDA, following the receipt of RMAT designation from the FDA just weeks ago, highlights the urgency of addressing this serious genetic disease and the potential of BEAM-302 to directly correct the DNA mutation, the underlying root cause of this illness. We are encouraged by the FDA’s continued support of this program and are committed to its advancement to deliver a one-time, potentially curative treatment to patients as quickly and safely as possible.”

BEAM-302 is currently being evaluated in a Phase I/II clinical trial, with positive early data reported in March 2025. The initial results from the first three single-ascending dose cohorts demonstrated that the therapy was well tolerated and showed dose-dependent correction of the disease-causing mutation. Notably, the 60 mg dose cohort achieved total AAT protein levels above the therapeutic threshold. Dosing in the fourth cohort (75 mg) has begun, with updated data expected in the second half of 2025.

Beam also plans to initiate Part B of the study, which will enroll AATD patients with mild to moderate liver disease, later this year. The company previously secured FDA clearance for its Investigational New Drug (IND) application for BEAM-302 in March 2025 and received Regenerative Medicine Advanced Therapy (RMAT) designation in May 2025.

Incannex Healthcare Advances IHL-42X to Phase III After FDA Protocol Clearance

Incannex Healthcare Inc. announced that its Phase III RePOSA trial for IHL-42X in obstructive sleep apnea (OSA) will proceed, following FDA protocol authorization under the company’s IND. IHL-42X is Incannex’s lead asset, and the trial marks a key advancement toward addressing a condition affecting nearly one billion people globally, with no currently approved oral drug therapies. The trial design will leverage a U.S.-only strategy and existing Phase II infrastructure to expedite execution.

“The progression of RePOSA into Phase III represents a major milestone for Incannex and our shareholders,” said Joel Latham, President and CEO of Incannex. “IHL-42X is a high-value asset targeting one of the most prevalent and under-treated conditions globally, OSA. With FDA clearance, a streamlined study design, and the drug product already manufactured, we are uniquely positioned to execute efficiently and deliver meaningful value. IHL-42X has the potential to become the first FDA-approved oral therapy for obstructive sleep apnea, and we believe it could transform the treatment paradigm for millions of patients across the globe.”

The Phase III study will be a randomized, placebo-controlled trial over 12 months, with a 3-month head-to-head comparison against monotherapies, dronabinol, and acetazolamide. It will measure the change in Apnea-Hypopnea Index (AHI) as the primary endpoint, alongside secondary endpoints like sleep quality and patient-reported outcomes. Approximately 30 U.S. sites will participate, including 20 from Phase II.

“We’re building on strong foundations. The ability to carry forward much of the operational infrastructure from Phase II gives us a significant edge in both speed and cost,” added Latham. “As we prepare to report Phase II topline results in the coming weeks, we’re excited by the growing momentum around IHL-42X and the commercial conversations already underway. This is a high-impact opportunity, and we remain focused on delivering the strongest outcome possible for our shareholders.”

Kura Oncology and Kyowa Kirin Gain FDA Priority Review for Ziftomenib NDA in NPM1-Mutant AML

Kura Oncology, Inc. and Kyowa Kirin Co., Ltd. announced that the FDA has accepted Kura’s New Drug Application (NDA) for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an NPM1 mutation. The NDA has been granted Priority Review, with a PDUFA target action date of November 30, 2025. If approved, ziftomenib would become the first menin inhibitor authorized for this genetically defined AML subgroup.

The NDA submission is based on positive results from the Phase II KOMET-001 registrational trial, which achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh). Ziftomenib demonstrated a favorable safety profile with limited myelosuppression and only 3% treatment-related discontinuations. The investigational therapy has previously received Breakthrough Therapy, Fast Track, and Orphan Drug Designations from the FDA.

“The FDA’s acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin, we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.”

“Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease.”

GSK’s NDA for Linerixibat Accepted by FDA for PBC-Related Cholestatic Pruritus

GSK plc announced that the FDA has accepted its New Drug Application (NDA) for linerixibat, a targeted ileal bile acid transporter (IBAT) inhibitor, for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 24, 2026.

The NDA is supported by positive results from the Phase III GLISTEN trial, which met its primary and key secondary endpoints. The study demonstrated a rapid, significant, and sustained reduction in cholestatic pruritus and itch-related sleep interference compared to placebo. The safety profile of linerixibat remained consistent with earlier findings and its IBAT inhibition mechanism.

“The FDA’s acceptance of this file is an important milestone in the development of linerixibat. We believe that linerixibat has the potential to make a difference in the lives of patients living with relentless itch associated with PBC and its related sleep interference. These are debilitating symptoms which currently have very limited treatment options,” said Kaivan Khavandi, SVP, Global Head, Respiratory, Immunology & Inflammation R&D, GSK.

Linerixibat has not yet been approved for use in any country. If approved, it would offer a novel treatment option for a rare, burdensome condition with high unmet medical need.

Ascendis Pharma’s TransCon CNP Receives FDA Priority Review for Achondroplasia

Ascendis Pharma A/S announced that the FDA has accepted its New Drug Application (NDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia, a rare genetic bone growth disorder. The application has been granted Priority Review, and the FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025. Notably, the FDA does not currently plan to convene an advisory committee meeting to discuss the application.

TransCon CNP is an investigational once-weekly prodrug of C-type natriuretic peptide (CNP) designed to deliver sustained exposure to active CNP, enabling continuous inhibition of the overactive FGFR3 pathway, the underlying cause of achondroplasia. In Phase II clinical trials, TransCon CNP showed a statistically significant improvement in annualized growth velocity, meeting its primary endpoint. Additionally, the treatment demonstrated multiple improvements beyond linear growth, including better lower limb alignment, spinal canal dimensions, and muscle strength, with a safety and tolerability profile similar to placebo.

“Too many profound medical needs endure for people living with achondroplasia,” said Chandler Crews, Founder of The Chandler Project. “Therapies that could address some of the underlying, serious complications of achondroplasia offer welcome potential to improve health outcomes beyond what currently approved therapies and interventions offer.”

“People living with achondroplasia and their physicians have expressed an urgent need for a meaningful treatment option to address the complications of achondroplasia,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “We look forward to working with the FDA during its review to make TransCon CNP available as quickly as possible.”

If approved, TransCon CNP could become a new standard of care for children with achondroplasia by targeting not just growth but also the broader skeletal and functional complications of the condition.