December 16, 2024 - 🧬 [nGram] Today’s Scoop: Neurocrine's CRENESSITY™ FDA Approval, Larimar's Positive Data, & More

1. Neurocrine Biosciences announces FDA approval of Crenessity for congenital adrenal hyperplasia

   • Crenessity, a first-in-class treatment for classic congenital adrenal hyperplasia (CAH), has been approved by the FDA.
   • The approval is based on data from the largest-ever clinical trial program for CAH, involving both pediatric and adult patients.
   • Crenessity allows for glucocorticoid dose reduction by directly reducing excess ACTH and adrenal androgen production.
   • The treatment will be available commercially in about a week, with support programs to assist patients in accessing the medication.
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2. Larimar Therapeutics announces positive initial data from ongoing long-term open label extension study for Friedreich’s ataxia

   • Daily subcutaneous injections of 25 mg nomlabofusp in 14 participants were well tolerated for up to 260 days.
   • Tissue frataxin (FXN) levels increased from 15% to 30% in buccal cells and from 16% to 72% in skin cells by Day 90.
   • Early trends towards improvement in clinical outcomes were observed, supporting potential clinical benefits.
   • Dose escalation to 50 mg daily has begun, with a global confirmatory study planned for mid-2025.
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3. FDA approves new treatment for congenital adrenal hyperplasia

   • The FDA approved Crenessity (crinecerfont) for use with glucocorticoids to manage androgen levels in patients with classic congenital adrenal hyperplasia (CAH).
   • Approval is based on two trials: one with 182 adults and another with 103 children, showing significant reduction in glucocorticoid doses while maintaining androstenedione control.
   • Crenessity has warnings for acute adrenal insufficiency and should not be used with certain enzyme-activating drugs.
   • The FDA granted Crenessity Fast Track, Breakthrough Therapy, Orphan Drug, and Priority Review designations.
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4. Newron and EA Pharma announce license agreement for evenamide in Japan and other Asian territories

   • Newron Pharmaceuticals and EA Pharma have entered a license agreement for the development, manufacturing, and commercialization of evenamide in Japan and other Asian territories.
   • Newron will receive up to €117 million, including an upfront payment of €44 million, and tiered royalties from EA Pharma.
   • Evenamide is a novel modulator of glutamate release, showing efficacy in treatment-resistant schizophrenia (TRS) patients.
   • Newron plans to initiate a Phase III one-year study in H1 2025 outside the licensed territories.
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5. Acoramidis receives positive CHMP opinion for treatment of transthyretin amyloid cardiomyopathy

   • The CHMP has recommended EU approval for acoramidis based on the Phase 3 ATTRibute-CM study results.
   • Acoramidis, known as Attruby in the US, is a near-complete stabilizer of transthyretin (TTR) and was approved by the FDA in November 2024.
   • The ATTRibute-CM study showed a 42% reduction in composite all-cause mortality and cardiovascular-related hospitalization events at 30 months.
   • Pending European Commission approval, Bayer plans to launch acoramidis in Europe in the first half of 2025.
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6. CHMP recommends approval of Galderma’s nemolizumab for moderate-to-severe atopic dermatitis and prurigo nodularis in the European Union

   • The CHMP of the EMA has recommended marketing authorization for nemolizumab for treating moderate-to-severe atopic dermatitis and prurigo nodularis in the EU.
   • Nemolizumab is intended for subcutaneous use in patients aged 12 and older for atopic dermatitis and adults for prurigo nodularis.
   • The recommendation is based on positive results from the phase III ARCADIA and OLYMPIA clinical trials, showing significant improvements in itch and skin nodules.
   • The European Commission will now decide on the EU-wide marketing authorization, and nemolizumab is also under review by other regulatory authorities globally.
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7. BrightPath Bio and Cellistic announce collaboration for phase 1 clinical trial of iPSC-derived BCMA CAR-iNKT cell

   • BrightPath Bio and Cellistic have entered a collaboration to develop and manufacture iPSC-derived BCMA-targeting CAR-NKT cells.
   • The partnership will utilize Cellistic's 3D bioreactor-based manufacturing platform, Echo, for GMP-compliant production.
   • This collaboration aims to advance a Phase 1 clinical trial for multiple myeloma using these novel cell therapies.
   • The agreement marks a significant step in developing scalable and effective cell therapies for cancer treatment.
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