FDA Approval and Launch Readiness of Paltusotine (PALSONIFY)
Crinetics' NDA for paltusotine (PALSONIFY) remains on track with an anticipated FDA approval in September 2025.
The company has assembled a highly experienced launch team and is preparing for a transformational market entry.
Engagement with patient communities and advocacy groups has reinforced confidence that PALSONIFY addresses a critical unmet need in acromegaly treatment.
The launch is expected to be gradual, with formulary placement taking 6-9 months post-approval and patient adoption aligning with infrequent endocrinologist visits (2-4 times/year).
Commercial preparations include a dedicated sales force of approximately 30 representatives and ongoing payer discussions emphasizing PALSONIFY's value proposition of rapid disease control and convenience.
Crinetics is actively working on international expansion, targeting a European launch in 2026.
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.
ACADIA's Pipeline Milestones and Upcoming Data Readouts
ACADIA showcased nine disclosed pipeline programs during its R&D Day, with five Phase II or III data readouts expected through 2027.
Key upcoming milestones include initiating a Phase II study of ACP-204 in Lewy body dementia psychosis in Q3 2025 and top-line results from the COMPASS PWS Phase III study in Q4 2025.
The company plans to start a Phase III study of trofinetide in Rett syndrome in Japan in Q3 2025 and a first-in-human study of ACP-271 before year-end.
Progress in the pipeline underscores ACADIA's strategic focus on expanding indications and leveraging novel biology, with multiple data readouts potentially transforming its market position.
Veracyte delivered approximately 25,500 Decipher tests in Q2, on track to exceed 100,000 tests annually, marking a significant milestone.
Decipher volume grew 28% year-over-year, with 13 consecutive quarters of over 25% growth, driven by strong execution and expanding clinical utility.
The metastatic prostate cancer test launch in June has generated increased physician interest, especially in high-risk NCCN patients, contributing to halo effects and future growth potential.
The company expects Decipher to maintain double-digit growth driven by market share gains, incidence growth, and expanding clinical evidence, including ongoing prospective trials and guideline updates.
Sephience received EU approval in late June and FDA approval in the US for broad labeling for patients aged 1 month and above.
The company expects Sephience to become the standard of care for PKU, with a revenue opportunity exceeding $1 billion in the US.
European launch began in Germany in mid-July, leveraging early access mechanisms in other countries while pricing and reimbursement are being finalized.
Initial US launch is planned within two weeks, targeting 104 PKU centers that treat over 80% of US PKU patients.
The company is engaging payers with positive feedback, minimal restrictions, and plans to expand access in Japan and Brazil before year-end.
Successful U.S. Launch of AMVUTTRA for ATTR-CM and Early Market Penetration
Alnylam reported a rapid and broad uptake of AMVUTTRA in the U.S. within just the first full quarter post-launch, with approximately 1,400 patients treated by June 30, 2025.
The launch has exceeded initial expectations, with coverage and access achieved faster than anticipated across major health systems and payers.
Nearly all of the 170 priority health systems now have formulary access to AMVUTTRA, enabling treatment initiation in a majority of ATTR-CM patients.
Early utilization data shows a balanced mix of first-line and stabilizer-progressing patients, with prescriber base tripling quarter-over-quarter, indicating strong physician confidence.
Impact of FDA Inspection Delay on EYLEA HD and BLA for Odronextamab
EYLEA HD regulatory application delays caused by FDA site inspection at Catalent Indiana LLC, which was not specific to EYLEA HD.
Novo Nordisk expects to file a comprehensive response next week, anticipating an expeditious resolution.
BLA for odronextamab, a bispecific antibody for follicular lymphoma, was impacted by the same site inspection, resulting in a Complete Response Letter (CRL) issued earlier this week.
Management remains optimistic about resolving these issues quickly based on Novo's communication and progress with third-party fillers.
Strategic Focus on Phase III Readouts and Clinical Progress
The company is actively progressing towards multiple pivotal Phase III trials, including in myelofibrosis and endometrial cancer, with top-line data expected in 2026.
There is a strong emphasis on the potential of selinexor in combination with ruxolitinib to redefine the standard of care for myelofibrosis, with an estimated peak revenue potential of up to $1 billion annually in the U.S.
Management highlighted the significance of recent enrollment milestones, such as closing new patient screening for the SENTRY trial in myelofibrosis, which is a key step in their clinical development strategy.
The company is leveraging its clinical data to support regulatory and commercial ambitions, emphasizing the potential for selinexor to address unmet needs in diseases with limited treatment options.
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