September 14, 2026

Corbus CRB-913 5% Weight Loss Phase 1b Win (+12% Stock), Insmed BRINSUPRI Guidance to $1.4B, Kelun Trastuzumab Botidotin JCO

Corbus CRB-913 5% at 60 mg in Phase 1b obesity, Insmed raises BRINSUPRI FY guidance to $1.25-1.4B, Kelun trastuzumab botidotin 11.1 vs 4.4-mo PFS in JCO.

6 stories · Covering clinical-trials, industry, research, regulatory

Editor's Note

Monday morning delivered the September obesity-catalyst readout the field had been counting down to. Corbus Pharmaceuticals reported positive Phase 1b CANYON-1 topline for CRB-913 (once-daily oral peripherally-restricted CB1 inverse agonist for obesity): 5.0% mean weight loss at 60 mg, 3.3% at 40 mg, 2.8% at 20 mg versus 0.0% on placebo at 12 weeks (all p<0.0001), with no plateau observed. Discontinuation rates ran 3.1-13.1% across doses and psychiatric adverse events (depression 1.6%, anxiety 4.8%, irritability 9.7% at 60 mg) came in comparable to oral GLP-1 experience with no suicidality signal — a specific bar CB1 inverse agonists have had to clear since the 2008 Sanofi Acomplia (rimonabant) withdrawal on psychiatric grounds. Corbus shares jumped 12%, and management guided to a Phase 2 monotherapy trial initiation in H1 2027 plus early evaluation of a GLP-1 combination approach. The Morgan Stanley 24th Annual Global Healthcare Conference opened in New York (September 14-16), with Insmed raising full-year BRINSUPRI (brensocatib) revenue guidance to $1.25-1.4 billion (up from earlier launch targets) and signaling peak U.S. sales potential above $7 billion in the bronchiectasis indication alone. Alnylam trimmed its full-year TTR franchise guidance at the same conference after early Amvuttra launch demand normalized, while emphasizing pipeline strength. In publication news, Sichuan Kelun-Biotech's HER2-targeted antibody-drug conjugate trastuzumab botidotin recorded 11.1-month median progression-free survival versus 4.4 months on Roche's T-DM1 (Kadcyla) in a Phase 3 breast-cancer study just out in the Journal of Clinical Oncology. The Ultragenyx UX111 (rebisufligene etisparvovec) PDUFA action date for Sanfilippo Syndrome Type A gene therapy sits 5 days out on September 19, and the Novo Nordisk Capital Markets Day in London on September 21 is the following week's centerpiece after Friday's Morgan Stanley Underweight downgrade.

Clinical Trials

Corbus CRB-913 Phase 1b CANYON-1 Meets Primary Endpoint at All Three Doses: 5.0% Weight Loss at 60 mg vs 0.0% Placebo at 12 Weeks, No Plateau, No Suicidality; CRBP +12%

Corbus Pharmaceuticals (NASDAQ: CRBP) announced Monday September 14, 2026 positive topline results from the Phase 1b CANYON-1 trial evaluating CRB-913 (once-daily oral peripherally-restricted CB1 inverse agonist for obesity) in 254 obese non-diabetic U.S. adults across 15 sites (NCT07310901). Mean weight loss at 12 weeks: 5.0% at 60 mg (n=62), 3.3% at 40 mg (n=61), 2.8% at 20 mg (n=65) versus 0.0% on placebo (n=66) — all p<0.0001. No plateau was observed at any dose. Discontinuation rates ranged 3.1-13.1% across doses (comparable to 6.9-20.7% for oral GLP-1s in cross-trial comparison). At the 60 mg dose: nausea 22.6%, diarrhea 22.6%, constipation 4.8%, vomiting 1.6%. Psychiatric adverse events at 60 mg: depression 1.6%, anxiety 4.8%, irritability 9.7%, insomnia 0% — no serious psychiatric events or suicidality reported. CRB-913 was designed with approximately 15-fold lower brain penetration than monlunabant in preclinical models to preserve CB1-driven weight loss while limiting the psychiatric side effects that led to the 2008 withdrawal of Sanofi's Acomplia (rimonabant). CRBP shares closed up 12% on the day. Next steps: FDA clinical development plan engagement, Phase 2 monotherapy trial initiation in H1 2027, evaluation of a GLP-1 combination approach, and full data presentation at ObesityWeek 2026 (November 14-17, Washington DC).

#corbus-pharmaceuticals #crbp #crb-913 #canyon-1 #phase-1b-primary-endpoint-met #5-percent-weight-loss-60mg #12-weeks #254-participants #no-plateau #no-suicidality #monlunabant-comparator #rimonabant-history #peripherally-restricted-cb1-inverse-agonist #crbp-up-12-percent #phase-2-h1-2027 #glp-1-combination-plan #obesityweek-2026 #non-peptide

Industry

Insmed Raises Full-Year 2026 BRINSUPRI (Brensocatib) Revenue Guidance to $1.25-1.4 Billion at Morgan Stanley Conference; Signals Peak U.S. Sales Above $7 Billion in Bronchiectasis Alone

Insmed Incorporated (NASDAQ: INSM) presented Monday September 14, 2026 at 2:35 p.m. ET at the Morgan Stanley 24th Annual Global Healthcare Conference in New York, raising full-year 2026 BRINSUPRI (brensocatib, oral dipeptidyl peptidase 1 inhibitor approved August 2025 for non-cystic fibrosis bronchiectasis in ages 12+) revenue guidance to $1.25-1.4 billion, up from earlier launch-year projections. Q2 2026 BRINSUPRI revenue was $309.2 million (49% sequential growth over Q1) driven by strong U.S. launch momentum through specialty pharmacy channels. Management flagged peak U.S. sales potential above $7 billion in the bronchiectasis indication alone, before considering additional expansion into COPD or comorbid asthma populations. The Phase 3 ASPEN trial supporting the U.S. approval enrolled 1,721 NCFB patients across 35+ countries; brensocatib reduced pulmonary exacerbations by 21.2% at the 10 mg dose (primary endpoint met). EMA and MHRA reviews are ongoing with Japanese filing planned for H2 2026. Insmed also confirmed the September 6-9 ERS Congress presentations included the ASPEN post-hoc bronchiectasis-plus-prior-NTM analysis and a COMPERA 2.0 risk-assessment analysis of treprostinil palmitil inhalation powder (TPIP) in pulmonary arterial hypertension.

#insmed #insm #brinsupri #brensocatib #dpp1-inhibitor #raised-fy2026-guidance #1-25-to-1-4-billion #7-billion-peak-us-sales #morgan-stanley-2026-conference #non-cystic-fibrosis-bronchiectasis #aspen-trial-support #ema-mhra-review #tpip-pah-context #non-peptide

Industry

Alnylam Trims Full-Year TTR Franchise Guidance at Morgan Stanley Conference Even as AMVUTTRA (Vutrisiran) ATTR-CM Launch Momentum Continues; Early Demand Mix Normalizing

Alnylam Pharmaceuticals (NASDAQ: ALNY) presented Monday September 14, 2026 at 10:00 a.m. ET at the Morgan Stanley 24th Annual Global Healthcare Conference in New York with CEO Yvonne Greenstreet and John Kennedy (SVP Global Commercialization / TTR Franchise Lead), disclosing a modest trim to full-year TTR franchise revenue guidance as early AMVUTTRA (vutrisiran, subcutaneous RNAi TTR silencer approved March 2025 for adults with ATTR amyloidosis with cardiomyopathy) launch demand normalized. Management emphasized the underlying market remains large and underdiagnosed, physician adoption is strong, and pipeline funding from AMVUTTRA commercial success is not at risk. AMVUTTRA competes in ATTR-CM against Pfizer's Vyndaqel/Vyndamax (tafamidis, oral small-molecule stabilizer) plus the recently launched BridgeBio Attruby (acoramidis, oral) — the launch mix has shifted as switch-from-tafamidis patients and treatment-naive patients balance out. HELIOS-B Phase 3 data anchored the approval with a 33% reduction in the composite of all-cause mortality plus recurrent cardiovascular events at 36 months. The trim adds nuance to the Alnylam commercial story that had run largely positive through Q2 2026 earnings; investors focused on the deeper pipeline (nucresiran next-generation TTR silencer plus the broader RNAi platform) rather than the near-term guidance move.

#alnylam-pharmaceuticals #alny #amvuttra #vutrisiran #rnai-ttr-silencer #attr-cm #full-year-guidance-trim #morgan-stanley-2026-conference #yvonne-greenstreet-ceo #vyndaqel-tafamidis-comparator #attruby-acoramidis-context #helios-b-phase-3 #33-percent-mace-reduction #nucresiran-pipeline #non-peptide

Research

Sichuan Kelun-Biotech Publishes Phase 3 HER2-Targeted Antibody-Drug Conjugate Trastuzumab Botidotin vs T-DM1 Data in Journal of Clinical Oncology: 11.1 vs 4.4 Months Median Progression-Free Survival

Sichuan Kelun-Biotech Biopharmaceutical (HKEX: 6990) announced Monday September 14, 2026 that Phase 3 registrational study results for trastuzumab botidotin — a novel HER2-targeted antibody-drug conjugate — versus T-DM1 (Roche's Kadcyla, trastuzumab emtansine) in HER2-positive unresectable or metastatic breast cancer have been published in the Journal of Clinical Oncology. Trastuzumab botidotin produced 11.1 months median progression-free survival versus 4.4 months on T-DM1, a hazard ratio of 0.39 (61% reduction in risk of progression or death). Co-senior authors: Professors Xichun Hu and Hongxia Wang of Fudan University Shanghai Cancer Center, and Dr. Junyou Ge. The NMPA approved trastuzumab botidotin in October 2025 for adults with unresectable or metastatic HER2-positive breast cancer who had received at least one prior anti-HER2 therapy. The T-DM1 comparator (approved 2013) has historically been the standard of care in this second-line setting, though Daiichi Sankyo/AstraZeneca's Enhertu (trastuzumab deruxtecan) has since taken market share on stronger DESTINY-Breast trial data. Kelun-Biotech has opened a Phase 2 study of trastuzumab botidotin in patients who previously received a topoisomerase inhibitor payload ADC (which includes Enhertu-treated patients). The Phase 3 result strengthens the case for global development beyond China.

#sichuan-kelun-biotech #6990-hkex #trastuzumab-botidotin #her2-targeted-adc #antibody-drug-conjugate #phase-3-jco-publication #11-1-vs-4-4-months-pfs #hazard-ratio-0-39 #t-dm1-comparator #kadcyla-comparator #enhertu-context #second-line-her2-breast #nmpa-approved-october-2025 #non-peptide

Regulatory

Ultragenyx UX111 (Rebisufligene Etisparvovec) FDA PDUFA Action Date Sits 5 Days Out on September 19 for Sanfilippo Syndrome Type A Gene Therapy; If Approved, First-Ever MPS IIIA Treatment

The Ultragenyx (NASDAQ: RARE) UX111 (rebisufligene etisparvovec, AAV9-delivered gene therapy for the SGSH gene) FDA PDUFA action date for Sanfilippo Syndrome Type A (mucopolysaccharidosis type IIIA, or MPS IIIA — a rare autosomal recessive lysosomal storage disorder causing progressive irreversible neurodegeneration in young children with a median life expectancy in the mid-teens) is set for Saturday September 19, 2026 (weekend PDUFA date; the FDA typically issues weekend action-date decisions on the adjacent Friday or the following Monday), five days from the September 14 opening of Morgan Stanley conference week. The resubmitted Biologics License Application under Accelerated Approval was accepted by the FDA in April 2026 following a July 2025 complete response letter that identified chemistry, manufacturing, and controls (CMC) plus manufacturing-site inspection issues. The clinical package includes up to 8 years of follow-up in treated children, with sustained cerebrospinal fluid heparan sulfate (the accumulated substrate) reduction plus preserved developmental trajectories relative to untreated natural history. If approved, UX111 would become the first-ever therapy for MPS IIIA anywhere in the world; the drug would also confer a Rare Pediatric Disease Priority Review Voucher on Ultragenyx (historically valued $150-350 million on the secondary market). Manufacturing runs domestically at Andelyn Biosciences in Columbus, Ohio and Ultragenyx's own Bedford, Massachusetts facility.

#ultragenyx #rare #ux111 #rebisufligene-etisparvovec #aav9-gene-therapy #sanfilippo-syndrome-type-a #mps-iiia #pdufa-september-19 #5-days-out #first-ever-mps-iiia-treatment #csf-heparan-sulfate #sgsh-gene #rare-pediatric-disease-prv #crl-resolved-april-2026 #non-peptide

Industry

Novo Nordisk Capital Markets Day in London Set for Monday September 21 as Week Ahead Centerpiece After Morgan Stanley Underweight Downgrade Cut Target to DKK 250 on Semaglutide Patent Cliff

Novo Nordisk (NYSE: NVO; Copenhagen: NOVO-B) heads into the week of September 14-19, 2026 with the September 21 Capital Markets Day in London one week away, and Friday September 11 fresh in investor minds. Morgan Stanley analyst Thibault Boutherin had cut Novo Nordisk to Underweight from Equal-weight Friday with a DKK 250 price target (implying more than 10% downside) citing subdued mid-term growth outlook plus concerns about the semaglutide loss-of-exclusivity beginning 2031 (semaglutide accounts for approximately 75% of Novo's 2026 sales). HSBC on Wednesday September 9 had raised its price target to DKK 320 (Hold), and Barclays on September 8 had trimmed its target to DKK 300 (Equal Weight). Novo shares closed the prior week down 2.8% in Copenhagen at DKK 278.2. The London Capital Markets Day is expected to unveil fresh strategic ambitions to replace existing targets, plus pipeline detail on CagriSema (cagrilintide + semaglutide) after the February 2026 Redefine-1 head-to-head miss versus Zepbound (23% vs 25.5% weight loss), amycretin (GLP-1/amylin unimolecular dual agonist Phase 3 planning), and the post-semaglutide non-GLP-1 diversification story after the September 7 HERMES + ATHENA ziltivekimab heart-failure trial terminations.

#novo-nordisk #nvo #novo-b #capital-markets-day-london #september-21-2026 #morgan-stanley-underweight-downgrade #dkk-250-price-target #semaglutide-patent-cliff #2031-loss-of-exclusivity #hsbc-dkk-320-hold #barclays-dkk-300 #cagrisema-redefine-1-context #amycretin-phase-3-planning #ziltivekimab-hermes-athena-terminations #glp-1