Peptide News Digest

Merck-Moderna Intismeran Phase 3 Melanoma Hit, Capricor Deramiocel PDUFA, WuXi Divests Bestchrom, BioMarin Acquires Alesta

Merck-Moderna INTerpath-001 intismeran+Keytruda Phase 3 melanoma hits RFS and DMFS. Capricor deramiocel PDUFA. WuXi Bestchrom sale. BioMarin buys Alesta.

4 stories · Covering clinical-trials, regulatory, industry

Editor's Note

Sunday's peptide-adjacent news is anchored on the landmark Phase 3 win for individualized cancer neoantigen therapy: Merck and Moderna announced Wednesday August 19 that the INTerpath-001 trial of intismeran autogene (V940 / mRNA-4157) plus pembrolizumab (Keytruda) met its primary endpoint of recurrence-free survival and the key secondary endpoint of distant metastasis-free survival in adults with high-risk (Stage IIB-IV) resected cutaneous melanoma; the readout is the first positive Phase 3 for an individualized neoantigen therapy and the first Phase 3 to demonstrate a substantial improvement over Keytruda alone in the adjuvant melanoma setting, building on the Phase 2b KEYNOTE-942 result that had shown a 49% reduction in recurrence or death risk. Capricor Therapeutics (NASDAQ: CAPR) faced its PDUFA target action date Saturday August 22 for deramiocel in Duchenne muscular dystrophy cardiomyopathy following the July 29 FDA Cellular, Tissue, and Gene Therapies Advisory Committee 9-3 vote against approval, with a Complete Response Letter the expected outcome that would mark the second CRL for the program. WuXi Biologics announced Sunday August 23 an agreement to sell its 51.1% stake in Bestchrom, a Shanghai-based supplier of chromatography resins and columns used across peptide and biologic manufacturing, to an undisclosed independent third party, part of the Chinese CDMO's continued strategic portfolio pruning. And BioMarin Pharmaceutical (NASDAQ: BMRN) announced August 21 a definitive agreement to acquire Dutch biotech Alesta Therapeutics for lead clinical-stage asset ALE1, adding to BioMarin's rare-disease portfolio alongside VOXZOGO (vosoritide, C-type natriuretic peptide analog for achondroplasia), the newly-integrated Amicus assets (GALAFOLD, POMBILITI + OPFOLDA), and the enzyme-replacement franchise.

Clinical Trials

Merck (NYSE: MRK) and Moderna (NASDAQ: MRNA) Announced Wednesday August 19, 2026 That the Phase 3 INTerpath-001 Trial of Intismeran Autogene (V940 / mRNA-4157, an Individualized Neoantigen Therapy) Plus Pembrolizumab (Keytruda) Met Its Primary Endpoint of Recurrence-Free Survival (RFS) and Key Secondary Endpoint of Distant Metastasis-Free Survival (DMFS) in Adults With High-Risk (Stage IIB-IV) Resected Cutaneous Melanoma Compared to Keytruda Alone; The Readout Is the First Positive Phase 3 for an Individualized Neoantigen Therapy and the First Phase 3 to Demonstrate Substantial Improvement Over Keytruda Alone in the Adjuvant Melanoma Setting; Phase 2b KEYNOTE-942 Five-Year Follow-Up Data Presented at ASCO 2026 Had Shown a 49% Reduction in Risk of Recurrence or Death and a 59% Reduction in Risk of Distant Metastasis or Death Versus Keytruda Alone

Merck (NYSE: MRK) and Moderna (NASDAQ: MRNA) announced Wednesday August 19, 2026 that the Phase 3 INTerpath-001 trial of intismeran autogene (V940 / mRNA-4157, an individualized neoantigen therapy) plus pembrolizumab (Keytruda) met its primary endpoint of recurrence-free survival (RFS) and the key secondary endpoint of distant metastasis-free survival (DMFS) in adults with high-risk (Stage IIB-IV) resected cutaneous melanoma compared to Keytruda alone. Mechanism: intismeran autogene is a personalized mRNA therapy that encodes up to 34 tumor-specific neoantigen peptides selected from the individual patient's tumor mutation signature. The mRNA is delivered as a lipid nanoparticle injection; cells at the injection site translate the mRNA into the neoantigen peptides, which are then presented to the immune system to generate a targeted T-cell response against the patient's tumor. The Phase 3 readout is the first positive Phase 3 for an individualized neoantigen therapy and the first Phase 3 to demonstrate substantial improvement over Keytruda alone in the adjuvant melanoma setting. Trial design: randomized, double-blind, placebo- and active-comparator-controlled global Phase 3 evaluating safety and efficacy of the combination versus Keytruda alone. Phase 2b KEYNOTE-942 five-year follow-up data presented at the 2026 ASCO Annual Meeting had shown a 49% reduction in risk of recurrence or death and a 59% reduction in risk of distant metastasis or death for the combination versus Keytruda alone, and the Phase 3 readout confirms and extends those benefits. The result validates the personalized neoantigen mRNA vaccine platform and opens a substantial commercial pathway for the Merck-Moderna collaboration in adjuvant oncology settings beyond melanoma including non-small-cell lung cancer, renal cell carcinoma, and cutaneous squamous cell carcinoma where INTerpath studies are ongoing.

#merck #moderna #intismeran-autogene #v940 #mrna-4157 #keytruda #pembrolizumab #interpath-001 #phase-3 #melanoma #individualized-neoantigen-therapy #cancer-vaccine #rfs-endpoint #dmfs-endpoint #keynote-942
Regulatory

Capricor Therapeutics (NASDAQ: CAPR) Faced Its Prescription Drug User Fee Act (PDUFA) Target Action Date Saturday August 22, 2026 for Deramiocel (Cardiosphere-Derived Cell Therapy, Not a Peptide) in the Treatment of Duchenne Muscular Dystrophy (DMD) Cardiomyopathy Following the July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee 9-3 Vote Against Approval; Panel Members Cited Concerns About the Stability of the Statistical Results and How Missing Data Were Handled in the Phase 3 HOPE-3 Trial Left Ventricular Ejection Fraction (LVEF) Endpoint Analysis; A Complete Response Letter (CRL) Is the Expected Outcome, and Would Mark the Second CRL for Deramiocel Following the July 2025 Initial Rejection That Cited Inadequate Substantial Evidence of Effectiveness

Capricor Therapeutics (NASDAQ: CAPR) faced its PDUFA target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived allogeneic cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, with left ventricular ejection fraction (LVEF) endpoint outcomes appearing highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the DMD population. The DMD cardiomyopathy indication remains without an FDA-approved therapy, which continues the substantial unmet need in the space. Capricor holds Rare Pediatric Disease Designation for deramiocel, which may qualify the company for a Priority Review Voucher upon eventual approval if it comes.

#capricor-therapeutics #capr #deramiocel #duchenne-muscular-dystrophy #dmd-cardiomyopathy #pdufa-action-date #hope-3 #cell-therapy #complete-response-letter #adcom-9-3-vote #lvef-endpoint #rare-pediatric-disease-designation
Industry

WuXi Biologics (HKEX: 2269) Announced Sunday August 23, 2026 an Agreement to Sell Its 51.1% Stake in Bestchrom, a Shanghai-Based Supplier of Chromatography Resins and Columns Used Across Peptide and Biologic Manufacturing, to an Undisclosed Independent Third Party; The Divestment Continues WuXi's Strategic Portfolio Pruning Following the January 2025 Sale of Its US Manufacturing Operations to Altaris Capital and the December 2024 Sale of Its Vaccine Manufacturing Business, Repositioning the Chinese CDMO Around Its Core Antibody, Antibody-Drug Conjugate, and Recombinant Protein Contract Manufacturing Services; Bestchrom's Chromatography Resins Are Used Across Peptide API Downstream Purification and Are Part of the Broader Peptide-CDMO Supply Chain

WuXi Biologics (HKEX: 2269) announced Sunday August 23, 2026 an agreement to sell its 51.1% stake in Bestchrom, a Shanghai-based supplier of chromatography resins and columns used across peptide and biologic manufacturing, to an undisclosed independent third party. Financial terms of the transaction were not publicly disclosed. Bestchrom's chromatography resins and columns are used across peptide active pharmaceutical ingredient (API) downstream purification (removing impurities from crude peptide product after synthesis) and are part of the broader peptide-CDMO supply chain that supports the GLP-1 and rare-disease peptide manufacturing scale-up. The divestment continues WuXi's strategic portfolio pruning: the company sold its US manufacturing operations to Altaris Capital in January 2025 following the BIOSECURE Act pressures on Chinese biotech companies serving US customers, and sold its vaccine manufacturing business in December 2024. The Bestchrom divestment repositions WuXi around its core antibody, antibody-drug conjugate, and recombinant protein contract manufacturing services. For the broader peptide manufacturing landscape, the Bestchrom transaction adds another data point to the CDMO consolidation and specialization trend that also includes Samsung Biologics' pending $1.8 billion acquisition of PolyPeptide Group (prospectus expected end of August), the Gland Pharma / Neuland Laboratories sterile API partnership in Visakhapatnam, and ongoing capacity build-outs at Bachem, CordenPharma, and AmbioPharm.

#wuxi-biologics #bestchrom #chromatography-resins #peptide-manufacturing #cdmo-consolidation #shanghai-based #biosecure-act #downstream-purification #peptide-api #51-1-percent-stake
Industry

BioMarin Pharmaceutical (NASDAQ: BMRN) Announced Friday August 21, 2026 a Definitive Agreement to Acquire Dutch Biotech Alesta Therapeutics to Gain Alesta's Lead Clinical-Stage Asset ALE1, Adding to BioMarin's Rare-Disease Portfolio Alongside VOXZOGO (Vosoritide, C-Type Natriuretic Peptide Analog Administered as Daily Subcutaneous Injection for Achondroplasia and BioMarin's First $1+ Billion Blockbuster Franchise), the Newly-Integrated Amicus Therapeutics Assets (GALAFOLD Migalastat for Fabry Disease, POMBILITI + OPFOLDA Cipaglucosidase Alfa + Miglustat for Late-Onset Pompe Disease), and the Broader Enzyme-Replacement Franchise; The Alesta Acquisition Continues BioMarin's Pattern of Rare-Disease Portfolio Expansion Following the Amicus Integration Completed Earlier in 2026

BioMarin Pharmaceutical (NASDAQ: BMRN) announced Friday August 21, 2026 a definitive agreement to acquire Dutch biotech Alesta Therapeutics to gain Alesta's lead clinical-stage asset ALE1. Financial terms of the transaction were not publicly disclosed. Alesta's ALE1 program is in clinical development; the specific mechanism and indication have not been fully disclosed in initial press coverage, and additional details are expected in the deal filing documents. The acquisition adds to BioMarin's rare-disease portfolio alongside VOXZOGO (vosoritide, C-type natriuretic peptide analog administered as daily subcutaneous injection for achondroplasia in pediatric patients; delivered Q2 2026 revenue of $253 million +14% YoY and is on track for $1+ billion annual sales as BioMarin's first blockbuster franchise), the newly-integrated Amicus Therapeutics assets (GALAFOLD migalastat oral chaperone for Fabry disease, POMBILITI + OPFOLDA cipaglucosidase alfa plus miglustat for late-onset Pompe disease), and the broader enzyme-replacement franchise (VIMIZIM, Naglazyme, Aldurazyme, Palynziq). The Alesta acquisition continues BioMarin's pattern of rare-disease portfolio expansion following the Amicus integration completed earlier in 2026 that added roughly $220 million in expected non-GAAP cost synergies by 2028 and non-GAAP diluted EPS accretion beginning 2027. BioMarin's strategy of building a diversified rare-disease commercial franchise across peptide analogs (Voxzogo), oral small-molecule chaperones (GALAFOLD), enzyme replacements (Palynziq, VIMIZIM, Naglazyme, Aldurazyme), and now Alesta's ALE1 program continues to differentiate the company from single-franchise rare-disease competitors.

#biomarin-pharmaceutical #bmrn #alesta-therapeutics #ale1 #acquisition #voxzogo #vosoritide #rare-disease-portfolio #amicus-therapeutics #galafold #pombiliti #opfolda #dutch-biotech