FDA Priority Review and Regulatory Progress for Ziftomenib
The FDA has accepted Kura's NDA for ziftomenib in relapsed/refractory NPM1-mutant AML, granting a priority review with a PDUFA date of November 30, 2025.
Management reports ongoing collaborative and constructive interactions with the FDA, emphasizing alignment with the review timeline.
Regulatory milestones include addressing information requests and preparing for pre-approval inspections, with a focus on ensuring a successful review outcome.
Myriad's Strategic Shift to Cancer Care Continuum Focus
Myriad is updating its long-range strategy to focus on the Cancer Care Continuum (CCC).
The new strategy aims to drive accelerated growth and profitability through three pillars: expanding in cancer diagnostics, growing prenatal and mental health markets, and maintaining financial discipline.
The company plans to leverage its leadership in hereditary cancer testing and expand into therapy selection, genomic profiling, immuno-oncology, and MRD testing.
Partnerships, such as with PATHOMIQ for AI-enabled prostate cancer testing, will play a key role in this expansion.
Management emphasizes increased urgency, disciplined execution, and strategic partnerships to achieve growth targets.
U.S. Launch Growth and Real-World Data for Amtagvi
Iovance has surpassed 100 patients treated in a single quarter for the first time with Amtagvi, indicating strong adoption.
Real-world data shows a nearly 49% response rate overall and approximately 61% in patients treated in earlier lines, reinforcing the therapy's durability.
The company plans to onboard large community practices in Q4, targeting earlier treatment settings and potentially higher response rates.
Demand is driven by increased field activities, new centers, and strategic distribution channels like specialty pharmacies, expanding access in community settings.
Management emphasizes the potential for peak U.S. sales of $1 billion or more, with international markets offering additional growth opportunities.
The focus on earlier treatment settings and community oncology networks aims to position Amtagvi as a preferred option for appropriate patients.
FDA Label Expansion for EMPAVELI in C3G and Primary IC-MPGN
FDA approved a label expansion for EMPAVELI for patients 12 years and older with C3 myelopathy and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
This is the company's third FDA approval in 4 years, highlighting scientific strength and regulatory success.
The approval enables treatment with a C3-targeting therapy that achieves outcomes across proteinuria reduction, eGFR stabilization, and C3 deposit clearance.
Launch is underway, expanding into rare nephrology and underserved populations, including pediatric patients and post-transplant recurrence.
Strategic Merger of Pelthos and Channel Therapeutics
The merger involved a reverse merger process, creating a new public entity, Pelthos Therapeutics, which launched Zelsuvmi, a treatment for Molluscum contagiosum.
Pelthos's market forecast suggests capturing fewer than 100,000 patients in a 16.7 million patient market, with potential royalties of around $23 million annually in the U.S.
Ligand's strategic ownership of the nitric oxide platform and pipeline of late-stage programs offers multiple future royalty streams, emphasizing its focus on high-value assets.
FIRDAPSE generated $84.8 million in net product revenue in Q2 2025, up $7.5 million from Q2 2024, with a reaffirmed full-year guidance of $355-$360 million.
The growth was impacted in 2024 by the Change Healthcare cybersecurity breach, which shifted volume from Q1 to Q2, but the impact was fully resolved by June 2024.
Year-to-date, FIRDAPSE revenue increased 16.9% over the first half of 2024, indicating strong underlying demand and market durability.
Management emphasizes high prescription approval rates above 90% and low discontinuation rates below 20%, supporting sustained performance.
The company is actively expanding education efforts supported by updated NCCN guidelines, targeting undiagnosed cancer-associated LEMS patients, with an opportunity to reach a high-potential underserved population.
Global Expansion of VYJUVEK with Regulatory Approvals in Japan and Europe
Japan's Ministry of Health, Labour and Welfare approved VYJUVEK with a broad label similar to Europe, including all DEB patients from birth with home administration options.
Japan launch expected before year-end, leveraging recent positive open-label extension data and initial clinician experience.
European launch in Germany and France scheduled for the second half of 2025, with over 500 identified patients in each country supported by key centers.
European launch will include comprehensive patient support programs for home and caregiver administration, supported by dedicated commercial teams.
European reimbursement process is ongoing, with early engagement indicating recognition of the unmet medical need and potential for steady multi-year growth.
Dafinchi
