Key Outcomes:
- Novartis remibrutinib (P3 REMODEL-1/2, RMS) – Met primary endpoint; superior ARR reduction vs teriflunomide, positive secondary/MRI endpoints, clean liver safety
- Clover RSV+hMPV±PIV3 vaccines (P2, older adults) – Strong neutralizing antibody responses across all targets; no immune interference; ~62% fewer reported RTIs vs pbo
- Novo Nordisk semaglutide (P3 STEP Young, pediatric obesity) – Met primary endpoint; greater BMI reduction at wk 68; 40.4% no longer obese vs 0% pbo
- Novartis del-desiran (P3 HARBOR, DM1) – Failed primary endpoint (vHOT) at wk 54; some secondary/exploratory activity seen; development path under review
- Tam-Peli/YL201 (P3 TAISHAN-302, relapsed SCLC) – Significant survival benefit vs topotecan; consistent across subgroups incl. brain/liver mets; manageable safety
- Lundbeck bexicaserin (P3 DEEp SEA, Dravet syndrome) – Completed patient randomization (>100 pts); companion DEEp OCEAN trial already fully randomized
- Scholar Rock apitegromab (FSHD) – FDA Fast Track + Orphan Drug designation granted; P2 FORGE dosing initiated (no approved therapies currently exist for FSHD)
- Ionis ZANVASTRO/zilganersen (Alexander disease) – FDA approved; first disease-modifying therapy for AxD; met primary endpoint (gait speed stabilization, wk 61); received Rare Pediatric Disease PRV
- Roche Enspryng/satralizumab (MOGAD) – FDA Priority Review granted; P3 METEOROID showed 68% relapse risk reduction vs placebo; decision expected Jan 10, 2027
- Pharming Joenja/leniolisib (APDS) – FDA approval expanded to children 4-11 yrs (≥27kg); first approved pediatric treatment; available in US from Oct 2026
- Meant Health – Expanded telehealth access to FDA-approved GLP-1s (incl. Ozempic); weight-management use remains off-label
- Tarsus & Alkeus acquisition – Completed; adds P3 gildeuretinol for Stargardt disease (topline data expected 2029)
- Schrodinger & Tectora – Co-founded new immunology/inflammation biotech; $55M Series A from NEA and RA Capital
- Tyra Biosciences – Raised $400M via share/warrant offering to advance dabogratinib across oncology (LG-UTUC, IR NMIBC) and achondroplasia
September 01, 2026
Novartis Reports Positive P3 REMODEL 1/2 Results for Remibrutinib in Relapsing Multiple Sclerosis
Novartis reported positive topline results from the P3 REMODEL-1 and REMODEL-2 studies evaluating oral remibrutinib (BTK inhibitor) vs teriflunomide in adults with relapsing multiple sclerosis (RMS). Both studies met their primary endpoint, demonstrating statistically significant superiority of remibrutinib in reducing annualized relapse rate (ARR), with superiority also observed across all key secondary endpoints, including MRI measures of inflammatory brain lesions. In a preplanned combined analysis, remibrutinib showed a clinically meaningful delay in disability progression, with a positive trend in 3-month confirmed disability progression (3mCDP) and nominally significant 6-month confirmed disability progression (6mCDP). Novartis plans to present detailed results as a late-breaker at MSToronto2026 and seek global regulatory approval for remibrutinib in RMS.
Clinical Results:
Safety Profile: Remibrutinib was well tolerated, with no liver safety signal and no cases meeting Hy’s Law criteria.
Source: Novartis PR Sep 01, 2026
September 6, 2026
Clover Reports Positive P2 Data for RSV + hMPV ± PIV3 Combination Vaccines in Older Adults
Clover Biopharmaceuticals reported positive preliminary P2 data for SCB-1022 (RSV + hMPV) and SCB-1033 (RSV + hMPV + PIV3) in adults aged 60-85, with both candidates demonstrating robust neutralizing antibody responses, including ~6-9-fold increases for RSV, 6-8-fold for hMPV and >3-fold for PIV3. No meaningful immune interference was observed with the addition of PIV3, including in participants aged 75+, while the candidates showed a favourable safety and tolerability profile. Vaccine recipients also had an approximately 62% lower rate of reported respiratory tract infections vs pbo within 28 days, although infections were not PCR-confirmed. Clover also successfully completed 2,000L commercial-scale manufacturing, further supporting late-stage development and commercialization.
Source: PR Newswire PR Sep 6, 2026
September 07, 2026
Novo Nordisk’s Semaglutide Meets Primary Endpoint in P3 STEP Young Study in Children with Obesity
Novo Nordisk reported positive P3 STEP Young results evaluating semaglutide (QW SC) in combination with a reduced-calorie diet and increased physical activity in children aged 6 to <12 years with obesity. The study met its primary endpoint, demonstrating a greater reduction in BMI at wk 68 vs Pbo, with 40.4% of children treated with semaglutide no longer classified as having obesity vs none treated with Pbo. Both groups received lifestyle modification throughout the study. Detailed results will be presented at Obesity Week 2026, taking place November 14-17, in Washington DC, U.S.
Efficacy Results:
Safety Profile: Semaglutide demonstrated a safety and tolerability profile consistent with previous pediatric and adult trials of semaglutide and liraglutide, with no new safety concerns or concerns related to growth or pubertal development.
Source: GlobeNewswire Sep 07, 2026
September 08, 2026
Novartis Del-desiran Fails Primary Endpoint in P3 HARBOR Study in DM1
Novartis reported that the P3 HARBOR study evaluating delpacibart etedesiran (del-desiran) vs Pbo in ~150 patients with myotonic dystrophy type 1 (DM1) did not meet its primary endpoint of video hand opening time (vHOT), with no statistically significant improvement vs Pbo at wk 54. Evidence of clinical activity was observed across secondary endpoints and exploratory analyses, while safety findings were generally consistent with previously reported data. HARBOR is a global, randomized, double-blind, pbo-controlled study evaluating del-desiran administered Q8W over 54 wks, with key secondary endpoints including hand grip strength, QMT total score, DM1-Activ and 10-meter walk/run test. Novartis will evaluate the full dataset and engage with health authorities to determine the appropriate development path for del-desiran.
Source: GlobeNewswire PR Sep 08, 2026
September 13, 2026
Tam-Peli Demonstrates Significant Survival Benefit in Relapsed Small-Cell Lung Cancer
P3 TAISHAN-302 study showed that tambotatug pelitecan (Tam-Peli/YL201), a novel anti-B7-H3 ADC, significantly improved outcomes vs. topotecan in 451 patients with relapsed SCLC after 1L therapy. The data were presented at the IASLC 2026 WCLC.
Efficacy Results:
Median follow-up (mFU): 9.4 mos (as of May 20, 2026). Survival benefit was consistent across prespecified subgroups, including patients with brain metastases, liver metastases, and chemotherapy-free interval <90 days.
Safety Results:
Overall safety profile considered manageable and consistent with the known profile of Tam-Peli, with no new safety signals identified.
Source: PR Newswire PR Sep 13, 2026
September 15, 2026
Lundbeck Completes Patient Randomization in P3 DEEP SEA Trial of Bexicaserin for Dravet Syndrome
Lundbeck reported that the last patient has been randomized the P3 DEEP SEA study evaluating bexicaserin (LP352) for seizures in children and adults aged 2-65 years with Dravet syndrome. The randomized, double-blind, pbo-controlled multicentre study enrolled >100 participants and is assessing reduction in countable motor seizure frequency; eligible participants may transition to a 52-wk OLE. DEEp SEA is one of two pivotal study in Lundbeck’s global P3 DEE program; the second study, DEEp OCEAN, evaluates bexicaserin in developmental and epileptic encephalopathies (DEEs), including Lennox-Gastaut syndrome (LGS) but excluding Dravet syndrome, and completed randomization in Jul 2026.
Source: PR Newswire, Sep 15, 2026
September 02, 2026
Scholar Rock Receives US FDA Fast Track and Orphan Drug Designations for Apitegromab in FSHD
Scholar Rock stated that the US FDA granted Fast Track and Orphan Drug designations to apitegromab for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD), while participant dosing has commenced in the P2 FORGE study. FORGE is evaluating apitegromab, as monotherapy in adults with genetically confirmed FSHD. The P2 study will enroll ~60 participants randomized 1:1 to receive apitegromab 10 mg/kg or placebo IV Q4W for 52 wks. The primary endpoint is percent change in total lean muscle volume (LMV) measured by MRI at Wk 52, while secondary endpoints include percent change in LMV at Wk 24, changes in muscle parameters such as muscle fat fraction at Wks 24 and 52, and safety & tolerability. Additional exploratory endpoints will also be assessed. The FSHD program is supported by translational preclinical data demonstrating that a murine form of apitegromab increased muscle mass, strength and endurance in the gold-standard FLExDUX4 FSHD mouse model. There are currently no approved therapies for FSHD.
Source: Business Wire PR Sep 02, 2026
September 03, 2026
Ionis Receives US FDA Approval for ZANVASTRO as First Disease-Modifying Treatment for Alexander Disease
Ionis Pharmaceuticals received US FDA approval for ZANVASTRO (zilganersen) for the treatment of pediatric and adult patients with Alexander disease (AxD), making it the first and only disease modifying treatment for this ultra rare, progressive neurological disorder. Zanvastro is designed to reduce the production of excess glial fibrillary acidic protein (GFAP) and is administered as a 50 mg intrathecal injection Q3M. The approval was based on positive results from the pivotal P1/3 study, which met its primary endpoint in patients ≥5 years, with ZANVASTRO 50 mg demonstrating statistically significant and clinically meaningful stabilization of gait speed vs control at wk 61, with a 33.3% LS (Least-square) mean difference (p=0.041). In patients aged 2-4 years, ZANVASTRO also improved gross motor function as measured by GMFM-88 vs control at wk 61, while patient, caregiver and clinician reported secondary and exploratory endpoint outcomes consistently favoured ZANVASTRO. The therapy demonstrated a favourable safety and tolerability profile, with most AEs being mild or moderate and fewer serious TEAEs vs control. In conjunction with the approval, Ionis received a Rare Pediatric Disease PRV. In Jun 2026, Ionis entered into a license agreement with Recordati, granting exclusive rights to develop and commercialize zilganersen outside the US, with regulatory submissions in Europe and Japan expected in 2027.
Efficacy Results:
Source: Business Wire PR Sep 03, 2026
September 10, 2026
Roche Receives US FDA Priority Review for Enspryng in MOGAD
Roche received US FDA Priority Review for the sBLA for Enspryng (satralizumab) for the treatment of myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), a rare autoimmune CNS disorder with no approved treatments. The filing was supported by positive P3 METEOROID results, in which Enspryng significantly reduced the risk of MOGAD relapse by 68% vs Pbo (p=0.0025), with 87% of patients remaining relapse-free at 48 wks vs 67% with Pbo. Enspryng also demonstrated significant improvements in key secondary endpoints, including annualised relapse rate, MRI lesion activity and rescue therapy use, with a safety profile consistent with its established experience in NMOSD. The US FDA is expected to make an approval decision by Jan 10, 2027, while the EMA has validated the MOGAD application, with a European Commission decision expected in Q3’27.
Efficacy Results:
Source: GlobeNewswire PR Sep 10, 2026
September 11, 2026
US FDA Expands Joenja Approval to Children Aged 4-11 Years with APDS
Pharming received US FDA approval for Joenja (leniolisib), an oral selective PI3Kδ inhibitor, for the treatment of children aged 4-11 years with activated phosphoinositide 3-kinase delta syndrome (APDS) who weigh ≥27 kg, making it the first US FDA approved treatment for this pediatric population. The approval is supported by a multinational, open-label, single-arm P3 study in children aged 4-11 years, which demonstrated improvements over 12 wks in reduced lymphadenopathy and increased naive B cells, indicating improvement in the underlying immune defect. The safety profile was consistent with prior Joenja experience, with all TEAEs mild to moderate and no drug-related SAEs observed. The newly approved 40 mg and 50 mg BID doses are expected to become available in the US in Oct 2026. Pharming submitted a separate sNDA on Jul 30, 2026, seeking approval of lower doses for children weighing 13 to <27 kg.
Source: GlobeNewswire PR Sep 11, 2026
September 12, 2026
Meant Health Expands Telehealth Access to FDA-Approved GLP-1 Medications Including Ozempic
Meant Health expanded telehealth access to FDA-approved GLP-1 medications, including Ozempic (semaglutide), connecting patients with U.S.-licensed clinicians for eligibility assessment, prescribing and ongoing care coaching. Patients who qualify may receive FDA-approved GLP-1 prescriptions through the platform, while Meant’s clinical partners handle evaluation and prescribing and Care Coaches provide follow-up support. Ozempic is FDA-approved for type 2 diabetes, while its use for weight management is off-label and remains at the clinician’s discretion.
Source: PR Newswire, Sep 12, 2026
September 04, 2026
Tarsus Pharmaceuticals Completes Alkeus Acquisition, Adds P3 Gildeuretinol for Stargardt Disease
Tarsus Pharmaceuticals completed the acquisition of Alkeus Pharmaceuticals, adding worldwide rights to ALK-001 (gildeuretinol), an investigational oral late-stage molecular entity designed to reduce the accumulation of toxic vitamin A dimers while preserving the normal visual cycle for the treatment of Stargardt disease. ALK-001 has received U.S. FDA BTD, ODD, FTD, RPDD and is currently being evaluated in the global P3 NORTHSTAR study, with topline data expected in 2029. The acquisition, initially agreed on Aug 6, 2026, closed following satisfaction of customary closing conditions.
Source: GlobeNewswire PR Sep 04, 2026
September 09, 2026
Schrodinger Co-founds Tectora Therapeutics with $55M Series A to Advance Immunology and Inflammation Programs
Schrodinger co-founded Tectora Therapeutics with New Enterprise Associates (NEA) and RA Capital Management to develop oral therapies for immune-mediated diseases, with Tectora securing a $55M Series A financing from NEA and RA Capital. Schrodinger contributed two early-stage small-molecule programs, SDGR-4594 and SDGR-8139, in exchange for an equity stake in Tectora and eligibility for future milestones and royalties. Tectora will collaborate with Schrodinger’s drug discovery team and leverage its computational platform to advance the programs toward clinical candidates, with the initial focus on immunology and inflammation targets. The company plans to progress the programs through preclinical development and into clinical studies.
Source: Business Wire PR Sep 09, 2026
September 14, 2026
Tyra Raises $400M to Advance Dabogratinib Development Across Oncology and Achondroplasia
Tyra Biosciences priced a $400M underwritten offering of 9.079M common shares at $22.03/share and pre-funded warrants to purchase 9.079M shares at $22.029/warrant, with gross proceeds expected to be approximately $400M before underwriting discounts and expenses. The financing is expected to close on September 15, 2026, subject to customary conditions. Tyra plans to use the proceeds, together with existing cash, cash equivalents and marketable securities, to advance its dabogratinib 3×3 development strategy across low-grade upper tract urothelial carcinoma (LG-UTUC), intermediate-risk non-muscle invasive bladder cancer (IR NMIBC) and achondroplasia (ACH), as well as support preclinical and drug discovery programs and general corporate purposes. The offering was led by RA Capital Management, with participation from new and existing institutional investors.
Source: PR Newswire PR Sep 14, 2026
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Babli Singh is a Senior Analyst with strong analytical skills and a deep understanding of the biopharmaceutical and healthcare industries. She specializes in monitoring emerging trends and distilling complex information into concise, insightful summaries that deliver clear value and actionable intelligence for a global professional audience.
