September 8, 2026

Pulmovant PHocus PVR -56.3% Phase 2 Win, Structure Aleniglipron 16.2% at 72 Weeks, Ascendis reACHin Infant Sentinel Podium

Pulmovant mosliciguat PVR -56.3% at wk 16 in PH-ILD, Structure aleniglipron 16.2% at 72 wks + ACCG-2671 amylin data, Ascendis reACHin sentinel at ESPE.

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

Editor's Note

Tuesday delivered three consequential Phase 2/1-2a readouts and one late-breaking peptide poster, all tied to congresses running this week. Pulmovant's inhaled mosliciguat produced a placebo-adjusted 56.3% reduction in pulmonary vascular resistance at Week 16 in the PHocus Phase 2 study of pulmonary hypertension associated with interstitial lung disease, with a 35.2-meter placebo-adjusted six-minute walk gain and a 53.2% NT-proBNP reduction — the parent Roivant shares jumped roughly 17% on the day and the Phase 3 PHrontier trial in ~375 patients is already enrolling. Structure Therapeutics dropped a two-program dataset: aleniglipron (oral small-molecule GLP-1) reached 16.2% mean weight loss at 72 weeks in the ACCESS open-label extension with no observed plateau, and ACCG-2671 (oral small-molecule amylin/calcitonin dual agonist) posted a ~6-day half-life supporting once-weekly dosing plus a 3.3% single-dose weight reduction at Day 24, though Structure shares fell as investors weighed the data against the incumbent injectable class. Ascendis Pharma opened the ESPE 2026 congress with the first sentinel-cohort podium for navepegritide (TransCon CNP) in infants aged 0 to under 2 years with achondroplasia. Rein Therapeutics presented the Phase 1b dose-escalation results for inhaled LTI-03, a seven-amino-acid caveolin-1 scaffolding-domain peptide, at ERS 2026 following the same-day Nature Communications publication.

Clinical Trials

Pulmovant Mosliciguat Phase 2 PHocus Trial Meets Primary Endpoint With Placebo-Adjusted 56.3% PVR Reduction at Week 16 in PH-ILD; Phase 3 PHrontier Enrolling

Pulmovant, a Roivant Sciences (NASDAQ: ROIV) company, announced Tuesday September 8, 2026 that the Phase 2 PHocus study of inhaled mosliciguat (a once-daily first-in-class inhaled soluble guanylate cyclase activator) met its primary endpoint in patients with pulmonary hypertension associated with interstitial lung disease. Placebo-adjusted pulmonary vascular resistance reduction was 56.3% at Week 16 (-51.3% mosliciguat vs +6.6% placebo, p<0.0001) — the largest PVR reduction reported in any randomized controlled pulmonary hypertension trial per Pulmovant. Secondary endpoints: placebo-adjusted six-minute walk distance +35.2 meters (p=0.0027) and NT-proBNP -357.7 pg/mL, a 53.2% reduction from baseline (p=0.0002). At Week 24 (exploratory), 6MWD was +52.7 meters placebo-adjusted and NT-proBNP was -487.1 pg/mL (-75.9%). Mosliciguat was well-tolerated with lower cough incidence (12.1%) than placebo (18.2%). The trial enrolled 135 patients across 87 sites in 20 countries. The Phase 3 PHrontier study is enrolling approximately 375 patients globally in a 1:1 randomized double-blind placebo-controlled design. Marc Humbert (Université Paris-Saclay) presented the data at the ERS Congress in Barcelona; Roivant shares closed up approximately 17% on the day. Mosliciguat activates sGC independently of heme and nitric oxide, differentiating it from sGC stimulators like riociguat.

#pulmovant #roivant-sciences #roiv #mosliciguat #phocus-trial #phase-2-primary-endpoint-met #56-3-percent-pvr-reduction #ph-ild #pulmonary-hypertension #interstitial-lung-disease #135-patients #week-16 #phrontier-phase-3 #375-patients #inhaled-sgc-activator #roiv-plus-17-percent #ers-congress-2026 #barcelona #non-peptide

Clinical Trials

Structure Therapeutics Reports 16.2% Mean Weight Loss at 72 Weeks With Oral Aleniglipron in ACCESS OLE Plus First-in-Human Data for ACCG-2671 Oral Amylin/Calcitonin Dual Agonist

Structure Therapeutics (NASDAQ: GPCR) reported Tuesday September 8, 2026 positive data across two lead oral small-molecule obesity programs. Aleniglipron (oral small-molecule GLP-1 receptor agonist, formerly GSBR-1290) reached mean 16.2% body weight loss at 72 weeks at the 180 mg dose in the ACCESS Phase 2b open-label extension (11.6% at 45 mg, 14.4% at 90 mg), with no observed weight-loss plateau; fewer than 5% of participants discontinued for adverse events using the improved 2.5 mg starting dose with four-week titration. The Phase 3 ACCOMPLISH program enrolls up to 3,600 adults with obesity plus comorbidity (ACCOMPLISH-1) and up to 1,100 with obesity plus type 2 diabetes (ACCOMPLISH-2), with topline expected H2 2028. Separately, ACCG-2671 (oral small-molecule dual amylin and calcitonin receptor agonist) posted first-in-human Phase 1/2a single-ascending-dose data in 31 healthy volunteers: 3.3% body weight reduction after a single 10 mg dose at Day 24, ~6-day half-life supporting once-weekly dosing, CTX-1 bone resorption biomarker reduction ~60% by Day 2, no serious adverse events, no drug-induced liver injury, no nausea or vomiting at 1-2 mg doses (dose-related GI effects at 5+ mg). A 12-week multiple-ascending-dose trial in obese participants is enrolling with topline expected H1 2027. Despite the data, GPCR shares fell in Tuesday trading on incumbent-class competitive concerns.

#structure-therapeutics #gpcr #aleniglipron #gsbr-1290 #oral-small-molecule-glp-1 #access-ole #16-2-percent-weight-loss #72-weeks #accomplish-phase-3 #accg-2671 #oral-amylin-calcitonin #phase-1-2a #6-day-half-life #once-weekly-oral-amylin #3-3-percent-day-24 #non-peptide-small-molecule

Clinical Trials

Ascendis Pharma Presents First Sentinel-Cohort Podium Data for Navepegritide (TransCon CNP) in Infants With Achondroplasia at ESPE 2026 Tuesday September 8

Ascendis Pharma A/S (NASDAQ: ASND) presented first sentinel-cohort data from the Phase 3 reACHin trial of navepegritide (TransCon CNP, a sustained-release C-type natriuretic peptide prodrug branded YUVIWEL in the U.S. for achondroplasia in children ≥2 years) in infants aged 0 to under 2 years at the ESPE 2026 congress in Marseille on Tuesday September 8, 2026 (abstract FC4.6, 3:00-4:00 p.m. CEST podium session; Genevieve Baujat MD, Necker Hospital, presenting). The reACHin study completed target enrollment and extends navepegritide's tested age range down to infancy, the population most vulnerable to achondroplasia complications from cervicomedullary compression, foramen magnum stenosis, and delayed motor milestones. Additional ESPE presentations include the HighLiGHts Phase 3 trial design for lonapegsomatropin (TransCon hGH) across Turner syndrome, SHOX deficiency, small-for-gestational-age, and idiopathic short stature; two poster presentations on adolescent hypoparathyroidism patient-reported outcomes; and a systematic literature review on pediatric growth hormone deficiency prevalence. Ascendis' TransCon franchise generated €315 million in Q2 2026 product revenue (+105% year-over-year) with YUVIWEL contributing €8 million in its first U.S. commercial quarter.

#ascendis-pharma #asnd #navepegritide #transcon-cnp #yuviwel #reachin-trial #sentinel-cohort #phase-3-sentinel #achondroplasia-infants #0-to-under-2-years #espe-2026 #marseille #podium-fc4-6 #lonapegsomatropin #transcon-hgh #highlights-trial #peptide-prodrug

Research

Rein Therapeutics Presents Inhaled LTI-03 Phase 1b Dose-Escalation Poster at ERS Congress Following Nature Communications Publication; First Synthetic Caveolin-1 Peptide for IPF Reduces IL-11 and TSLP

Rein Therapeutics (NASDAQ: RNTX) presented Tuesday September 8, 2026 (poster PA6217, 12:30-2:00 p.m. CEST) at the European Respiratory Society Congress in Barcelona the Phase 1b randomized double-blind placebo-controlled dose-escalation study of inhaled LTI-03 in patients with idiopathic pulmonary fibrosis. LTI-03 is a first-in-class synthetic seven-amino-acid peptide derived from the caveolin-1 scaffolding domain (CSD), designed with a dual mechanism targeting alveolar epithelial cell survival and inhibition of profibrotic signaling. The trial randomized 24 IPF participants 3:1 to LTI-03 5 mg/day (N=9), LTI-03 10 mg/day (N=9), or placebo (N=6) for 14 days. LTI-03 was well-tolerated with no treatment-related discontinuations, no severe TEAEs, and no spirometry-based airway obstruction; both doses significantly reduced interleukin-11 (p=0.0406 at 5 mg/day; p=0.044 at 10 mg/day) and thymic stromal lymphopoietin. The results were published the same day in Nature Communications (Philip Molyneaux MD, Imperial College London, presenting). LTI-03 has FDA Orphan Drug and Fast Track designations (Fast Track granted August 2026); the Phase 2 RENEW trial is enrolling across five countries with interim data anticipated H2 2026.

#rein-therapeutics #rntx #lti-03 #inhaled-peptide #caveolin-1-scaffolding-domain #csd-peptide #seven-amino-acid-peptide #idiopathic-pulmonary-fibrosis #ipf #phase-1b-dose-escalation #nature-communications #il-11-reduction #tslp-reduction #24-participants #renew-phase-2 #fda-fast-track #fda-orphan-drug #ers-congress-2026

Industry

Novo Nordisk Stock Under Pressure Following Split Monday Announcements: STEP Young Pediatric Win Offset by Ziltivekimab HERMES + ATHENA HF Terminations; Barclays Cuts Price Target to DKK 300

Novo Nordisk (NYSE: NVO; Copenhagen: NOVO-B) shares closed at DKK 298.95 in Copenhagen on Monday September 7, 2026 (roughly 1.9% below Friday) after the company's simultaneous release of STEP Young Phase 3 pediatric semaglutide data (40.4% of children aged 6 to under 12 achieved BMI below the obesity threshold at week 68 versus 0% placebo) and confirmation that the HERMES and ATHENA Phase 3 ziltivekimab (anti-IL-6 monoclonal antibody) heart-failure trials had been terminated early after a data monitoring committee ruled the studies unlikely to succeed. Barclays' James Gordon cut the Novo Nordisk price target from DKK 310 to DKK 300 while maintaining an Equal Weight rating. Analyst commentary broadly framed the day as a scientific-engine-versus-diversification tension: the semaglutide franchise continues to add indication paths (pediatric obesity below 12 years is one of the last major label extensions available), while the non-GLP-1 diversification story tied to ziltivekimab now depends primarily on the post-acute heart-failure study that remains ongoing. Detailed STEP Young results will be presented at ObesityWeek 2026 in Washington DC November 14-17.

#novo-nordisk #nvo #novo-b #step-young #ziltivekimab #hermes-athena-terminations #barclays-cut-target #dkk-300-price-target #equal-weight-rating #semaglutide-pediatric #non-glp-1-diversification #post-acute-hf-study #obesityweek-2026 #glp-1

Regulatory

FDA Peptide Compounding Regulatory Status Continues Unchanged 46 Days After July PCAC Vote; No Proposed Rule, No Federal Register Notice

As of Tuesday September 8, 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) 5-3 vote recommending six of seven candidate peptides — BPC-157, KPV, TB-500, MOTS-c, Semax, and Epitalon — for inclusion on the Section 503A Bulk Drug Substances List; emideltide (DSIP) was rejected. Forty-six days have passed since the meeting ended on July 24. Center for Drug Evaluation and Research warning-letter activity to compounding pharmacies has remained elevated through Q3 2026, and no acting-FDA-commissioner statement has updated the industry timeline. The February 2027 PCAC meeting to consider cathelicidin (LL-37), GHK-Cu, dihexa acetate, melanotan II, and PEG-MGF remains scheduled. Nominated FDA Commissioner Dr. Heidi Overton (August 19, 2026) continues to await Senate confirmation with Kyle Diamantas leading the agency in the interim. Formal FDA rulemaking to translate the PCAC recommendation into a proposed rule and then a final rule typically runs 12 to 24 months from PCAC vote; the interval of 46 days is within the normal window but longer than industry advocates had modeled at the meeting.

#fda #pcac #503a-bulks-list #bpc-157 #kpv #tb-500 #mots-c #semax #epitalon #emideltide-rejected #compounding-pharmacies #46-days-since-vote #february-2027-pcac #overton-nomination-pending #diamantas-acting