Peptide News Digest

uniQure BLA Huntington's, Typewriter $56M Series A, Ultragenyx Angelman Phase 3 Miss, Invivyd Names Elia CEO

uniQure files AMT-130 gene therapy BLA for Huntington's, Ultragenyx apazunersen fails Phase 3 in Angelman, Typewriter emerges from stealth with $56M.

10 stories · Covering regulatory, clinical-trials, industry

Editor's Note

Thursday's calendar was heavy on rare-disease outcomes plus a fresh in-vivo CAR-T financing that continues to press the case for non-viral gene therapy platforms. uniQure submitted the BLA for AMT-130 (ifezuntirgene inilparvovec, an AAV gene therapy for Huntington's disease) under the accelerated-approval pathway that FDA cleared earlier in 2026 — the first BLA for a Huntington's disease therapy of any kind. Ultragenyx delivered the opposite outcome the same day: apazunersen (GTX-102, an intrathecal antisense oligonucleotide) missed both the primary and key secondary endpoints in the Phase 3 Aspire study in Angelman syndrome, ending the first late-stage antisense test in this indication. Typewriter Therapeutics emerged from stealth with $56 million in Series A capital co-led by RA Capital and AN Venture Partners to advance a target-primed reverse transcription (TPRT) non-viral gene therapy platform aimed at in-vivo CAR-T and genetic liver diseases. Invivyd installed Chairman Marc Elia as CEO Monday September 1 with the VYD2311 anti-COVID monoclonal antibody Phase 3 DECLARATION and LIBERTY readouts imminent. On the peptide-industry side, Eli Lilly's Q1 2027 BLA filing plan for retatrutide continues to anchor the H2 2026 obesity-catalyst calendar, Amgen's post-August-cull MariTide monthly injectable moves toward standalone Phase 3 focus, Corbus's CANYON-1 CRB-913 topline is expected this month, and the FDA has still issued no proposed rule six weeks after the July 23-24 PCAC vote to recommend six peptides for the 503A Bulks List.

Regulatory

uniQure Submits BLA to FDA for AMT-130 (Ifezuntirgene Inilparvovec) AAV Gene Therapy for Huntington's Disease Under Accelerated Approval Pathway

uniQure N.V. (NASDAQ: QURE) announced Wednesday September 2, 2026 the submission of a Biologics License Application (BLA) to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational AAV-delivered gene therapy for the treatment of Huntington's disease. The BLA is the first ever submitted for a Huntington's disease therapy of any kind. AMT-130 is delivered by stereotactic intraparenchymal injection into the striatum and expresses a microRNA designed to silence the mutant huntingtin (mHTT) gene. The submission is supported by three-year data from the Phase 1/2 program compared to a propensity score-matched external control from the Enroll-HD natural history database. uniQure also submitted a Marketing Authorisation Application (MAA) to the UK MHRA on the same day. A four-year data readout from the ongoing Phase 1/2 study is expected before end of Q3 2026. AMT-130 previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Approval would establish the first disease-modifying therapy for Huntington's, where standard-of-care remains symptom management with tetrabenazine and deutetrabenazine plus antipsychotics.

#uniqure #qure #amt-130 #ifezuntirgene-inilparvovec #huntingtons-disease #aav-gene-therapy #bla-submission #accelerated-approval #mrna-silencing #breakthrough-therapy #rmat-designation #enroll-hd-external-control #non-peptide
Clinical Trials

Ultragenyx Apazunersen (GTX-102) Antisense Oligonucleotide Fails Phase 3 Aspire Study in Angelman Syndrome; Misses Primary Bayley-4 Cognitive Endpoint

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced Wednesday September 2, 2026 that the Phase 3 Aspire study of apazunersen (GTX-102, an intrathecally-administered antisense oligonucleotide designed to reduce production of the paternal UBE3A antisense transcript in Angelman syndrome) did not meet its primary endpoint of change from Baseline in Bayley-4 cognitive raw score, nor its key secondary endpoint of Multidomain Responder Index (MDRI) net response. The 129-patient trial enrolled ages 4 to 17 with genetically confirmed full maternal UBE3A gene deletion. Safety was consistent with the Phase 1/2 program. The result is the first Phase 3 miss for an antisense drug in Angelman syndrome and closes off the primary regulatory path for GTX-102 in the enrolled population. Ultragenyx will discuss next steps with regulators and continues the Aurora study evaluating GTX-102 in additional Angelman syndrome genotypes and age groups. RARE shares fell substantially in premarket trading. The failure joins Roche/Ionis's tominersen Huntington's disease miss (GENERATION HD1, 2022) and Sage-Biogen's zuranolone MDD approval-with-restrictions as instances where CNS antisense programs failed to translate promising Phase 1/2 signals into Phase 3 wins.

#ultragenyx #rare #apazunersen #gtx-102 #antisense-oligonucleotide #angelman-syndrome #ube3a #aspire-phase-3 #bayley-4-cognitive-endpoint #129-participants #cns-aso #aurora-continuing-study #non-peptide
Industry

Typewriter Therapeutics Emerges From Stealth With $56 Million Series A to Advance TPRT Non-Viral Gene Therapy Platform for In-Vivo CAR-T and Genetic Liver Diseases

Typewriter Therapeutics announced Thursday September 3, 2026 its emergence from stealth with $56 million in Series A financing co-led by RA Capital Management and AN Venture Partners, with participation from ANRI, Gemseki, and SBI US Gateway Fund. The proceeds will advance the company's target-primed reverse transcription (TPRT) non-viral gene therapy platform toward clinical development, with initial focus on two high-value indications: in-vivo CAR-T and genetic liver diseases. TPRT uses jumping-gene-inspired biology to insert therapeutic DNA at specific target sites without the packaging-size constraints of AAV vectors or the payload delivery constraints of lipid nanoparticles. Typewriter plans to initiate non-human primate studies in late 2026 and establish its first in-vivo CAR-T development candidate over the next 12 months. The financing continues the H2 2026 in-vivo CAR-T financing wave that also delivered Umoja Biopharma's $100 million+ round and Capstan Therapeutics' Series C earlier in the summer.

#typewriter-therapeutics #56-million-series-a #ra-capital-management #an-venture-partners #tprt-platform #target-primed-reverse-transcription #non-viral-gene-therapy #in-vivo-car-t #genetic-liver-diseases #jumping-gene-biology #non-peptide
Industry

Invivyd Appoints Chairman Marc W. Elia as CEO Ahead of VYD2311 Phase 3 DECLARATION and LIBERTY COVID Antibody Readouts

Invivyd (NASDAQ: IVVD, Waltham, Massachusetts biopharma focused on serious viral infectious diseases) announced Tuesday September 1, 2026 that the Board of Directors appointed Chairman Marc W. Elia as Chief Executive Officer effective August 30, 2026. Elia has led Invivyd's corporate and scientific strategy since joining the board in 2022 and will serve as Chairman and CEO going forward at an annual base salary of $750,000. The board also named Ajay Royan Lead Independent Director and added Ian Sheffield as an independent director. The leadership transition is timed to Phase 3 readouts for VYD2311, an investigational anti-SARS-CoV-2 monoclonal antibody, in the DECLARATION and LIBERTY studies — both expected in the coming weeks. Invivyd's Pemgarda (pemivibart, the previous-generation SARS-CoV-2 antibody for pre-exposure prophylaxis in immunocompromised adults) received Emergency Use Authorization in March 2024 and remains the company's primary commercial product. A DECLARATION/LIBERTY win would extend the pre-exposure prophylaxis franchise and provide the commercial anchor for a broader antibody-plus-vaccine COVID pipeline.

#invivyd #ivvd #marc-elia #ceo-appointment #vyd2311 #declaration-trial #liberty-trial #sars-cov-2-antibody #pre-exposure-prophylaxis #immunocompromised #pemgarda-pemivibart #non-peptide
Industry

Eli Lilly Retatrutide Q1 2027 BLA Filing Plan Follow-Through After TRIUMPH-2 and TRIUMPH-3 Wins; Early-Access Program Rolls Out for Defined Patient Groups

Eli Lilly (NYSE: LLY) continued rollout through September 2026 of the early-access program for retatrutide (a once-weekly subcutaneous injectable GIP/GLP-1/glucagon triple agonist peptide for obesity) ahead of the Q1 2027 BLA submission plan confirmed after the July 23, 2026 TRIUMPH-2 and TRIUMPH-3 Phase 3 wins in obesity plus type 2 diabetes and obesity plus established cardiovascular disease. The 12 mg retatrutide dose produced 28.3% mean weight loss at 80 weeks in the TRIUMPH-1 Phase 3 obesity monotherapy trial reported in May 2026, the highest weight loss reported for any obesity therapy in a Phase 3 setting. TRIUMPH-4 (knee osteoarthritis, 68 weeks) added 28.7% weight loss plus improvements in pain and physical function. Seven Phase 3 readouts total are expected across the retatrutide program in 2026. Lilly management has framed retatrutide as the successor commercial franchise to Zepbound and Mounjaro, with launch anticipated 2027-2028 under the Most Favored Nation pricing framework that would apply from initial approval per the November 2025 pricing agreement.

#eli-lilly #lly #retatrutide #triumph-1 #triumph-2 #triumph-3 #triumph-4 #triple-agonist-peptide #gip-glp-1-glucagon #bla-q1-2027 #early-access-program #28-3-percent-weight-loss #mfn-pricing-framework
Industry

Amgen MariTide Consolidates as Sole Obesity Focus After AMG 513 Discontinuation; Late-Stage GLP-1-to-MariTide Switch Trial Underway

Amgen (NASDAQ: AMGN) continued through September 2026 the consolidation of its obesity portfolio around MariTide (maridebart cafraglutide, a monthly subcutaneous injectable antibody-peptide conjugate combining GLP-1 receptor agonist activity via the peptide component with GIP receptor antagonism via the antibody component) following the July 29, 2026 Q2 disclosure that Phase 1 AMG 513 development was halted. Amgen has launched a late-stage program specifically to test whether patients currently on weekly GLP-1 injections can be switched to monthly MariTide with equivalent efficacy and comparable tolerability — a differentiation strategy that uses the monthly dosing convenience as a switch driver rather than a first-line efficacy claim. Amgen expects to file for MariTide regulatory approval in late 2026 to early 2027. Six Phase 3 MariTide trials are enrolling across obesity, type 2 diabetes, heart failure, and obstructive sleep apnea. Cantor Fitzgerald analyst commentary earlier in 2026 had flagged a 4% bone mineral density decline signal from Phase 1 as a concern; Phase 3 data on bone health will factor into commercial positioning.

#amgen #amgn #maritide #maridebart-cafraglutide #monthly-injectable #antibody-peptide-conjugate #glp-1-agonist-gip-antagonist #amg-513-discontinued #glp-1-switch-trial #six-phase-3-trials #bone-mineral-density-signal #late-2026-early-2027-filing
Clinical Trials

Corbus CANYON-1 Phase 1b Topline for CRB-913 Peripherally Restricted CB1 Inverse Agonist in Obesity Expected This Month

Corbus Pharmaceuticals (NASDAQ: CRBP) continued through Thursday September 3, 2026 the countdown to CANYON-1 Phase 1b topline data for CRB-913 (a once-daily orally-administered peripherally-restricted CB1 inverse agonist for obesity). Last patient last visit was announced August 4, 2026, and topline is expected in September 2026. The 16-week double-blind placebo-controlled dose-ranging study enrolled 240 obese non-diabetic U.S. adults at once-daily doses of 20 mg, 40 mg, and 60 mg with 4-week safety follow-up. CRB-913 is engineered to remain peripheral (approximately 15-fold lower brain penetration than monlunabant in preclinical models) to preserve efficacy while limiting the psychiatric side effects that led to withdrawal of Sanofi's Acomplia (rimonabant) in 2008. The readout is one of the most-watched non-incretin obesity data points on the September calendar. A clean tolerability profile plus dose-responsive weight loss would enable Phase 2 initiation and set up CRB-913 as a differentiated add-on or alternative to the incretin class.

#corbus-pharmaceuticals #crbp #crb-913 #canyon-1 #peripherally-restricted-cb1-inverse-agonist #obesity #phase-1b-topline #240-participants #monlunabant-comparator #rimonabant-history #september-2026-readout #non-peptide
Clinical Trials

Enveda ENV-308 Lac-Phe-Mimetic Pill Phase 1 Muscle Preservation Data Continues to Drive Post-GLP-1 Discontinuation Discussion

Enveda Biosciences (Boulder, Colorado; AI-enabled natural product discovery platform) continues to draw attention through September 2026 for ENV-308, the first-in-class oral small-molecule mimetic of Lac-Phe (N-lactoyl-phenylalanine, an exercise-produced signaling molecule) whose Phase 1 safety and tolerability data was reported August 18, 2026. The Phase 1 trial in 88 healthy volunteers showed ENV-308 was well-tolerated with low GI side effects and reduced circulating leptin as a biomarker signal that the drug reaches metabolically-active concentrations in humans. In preclinical animal studies, ENV-308 preserved lean muscle during weight loss and prevented weight regain after weight-loss therapy was stopped. Enveda cites the statistic that roughly 1 in 8 U.S. adults have used a GLP-1 medication and most stop within a year for cost, side-effect, or plateau reasons. Phase 2 will test whether ENV-308 helps people maintain weight after stopping GLP-1s. Timeline: realistic earliest approval 2028-2029. ENV-308 joins bimagrumab (Eli Lilly, anti-activin receptor Phase 3), HM17321 (Hanmi-Genentech UCN2 analog, Phase 1), and ARO-INHBE (Arrowhead RNAi, Phase 1/2a) in the muscle-preservation pipeline.

#enveda-biosciences #env-308 #lac-phe #exercise-mimetic #post-glp-1-weight-maintenance #lean-muscle-preservation #phase-1 #88-volunteers #leptin-reduction #bimagrumab-comparator #hm17321-comparator #aro-inhbe-comparator #non-peptide
Regulatory

FDA Peptide Compounding Regulatory Limbo Enters Month Six Since July 23-24 PCAC Vote; No Proposed Rule, No Federal Register Notice, No Interim Enforcement Policy

As of Thursday September 3, 2026, the FDA has issued no proposed rule, no Federal Register notice, and no interim enforcement policy following the July 23-24, 2026 Pharmacy Compounding Advisory Committee (PCAC) vote to recommend six peptides — BPC-157, KPV, TB-500, MOTS-c, Semax, and Epitalon — for inclusion on the Section 503A Bulk Drug Substances List. The regulatory silence extends the gray zone that began with the April 16, 2026 removal of 12 peptides from Category 2 and continues to leave compounding pharmacies operating under enforcement risk. CDER warning-letter activity to compounding pharmacies remained elevated through Q3 2026 versus prior years. The February 2027 PCAC meeting to consider cathelicidin (LL-37), GHK-Cu, dihexa acetate, melanotan II, and pegylated mechano growth factor (PEG-MGF) remains scheduled; formal FDA rulemaking to translate PCAC recommendations into a proposed rule typically runs 6 to 18 months. The FDA Commissioner nomination of Dr. Heidi Overton continues to await Senate confirmation, with acting Commissioner Kyle Diamantas leading the agency through the interim.

#fda #pcac #503a-bulks-list #bpc-157 #kpv #tb-500 #mots-c #semax #epitalon #compounding-pharmacies #warning-letters #february-2027-pcac #overton-nomination-pending #month-six-limbo
Clinical Trials

Boehringer Ingelheim BI 3034701 First-in-Class GLP-1/GIP/NPY2 Triple Receptor Agonist Continues Phase 2 Enrollment in Obesity

Boehringer Ingelheim continues enrollment through September 2026 in the Phase 2 clinical trial of BI 3034701 (a potential first-in-class GLP-1/GIP/NPY2 triple receptor agonist), which the company initiated in July 2026 as part of the expanded obesity pipeline anchored on survodutide (dual GLP-1/glucagon agonist, in Phase 3 MASH with Zealand Pharma partnership). BI 3034701 was discovered via the Boehringer-Gubra Biopharmaceutical peptide discovery collaboration and is designed to engage the NPY2 receptor alongside GLP-1 and GIP receptors — a differentiated triple-target mechanism versus Eli Lilly's retatrutide (GIP/GLP-1/glucagon) and Hengrui's ribupatide (GIP/GLP-1/glucagon). The NPY2 receptor pathway is involved in central appetite regulation and has been targeted historically by hormones including PYY(3-36); Boehringer positions BI 3034701 as engaging appetite regulation through a complementary mechanism to the incretin axis. Phase 2 readout timing has not been publicly disclosed. Boehringer Ingelheim's overall obesity pipeline positions the company as a distant third-place challenger to Eli Lilly and Novo Nordisk with survodutide, BI 3034701, and the emerging GLP-1/glucagon dual agonist program.

#boehringer-ingelheim #bi-3034701 #glp-1-gip-npy2 #triple-receptor-agonist #obesity-phase-2 #gubra-peptide-discovery-collaboration #survodutide-context #npy2-appetite-regulation #retatrutide-comparator #ribupatide-comparator