Takeda Pharmaceutical Company Ltd

NextCell Pharma Strengthens Commercial Strategy with Appointment of MSC Therapy Expert Dr. Eric Strati

3 days ago

• NextCell Pharma has appointed Dr. Eric Strati, former executive at Takeda, Mesoblast, and Novartis, to its Board of Advisors to advance commercial strategy for ProTrans, its type 1 diabetes cell therapy. • Dr. Strati brings critical expertise from launching two approved MSC-based therapies (Alofisel and Ryoncil), strengthening NextCell's capabilities as it prepares for pivotal trials and partnership opportunities. • Recent analysis from the ProTrans-Young study shows promising trends in preserving insulin production in patients aged 12-21 years, with full trial results expected in the second half of 2026.

Takeda's Oveporexton Shows Promising Results in Phase 2b Trial for Narcolepsy Type 1

8 days ago

• The New England Journal of Medicine published data showing Takeda's investigational oral orexin receptor 2 agonist oveporexton demonstrated significant improvements in wakefulness and reduced cataplexy events in narcolepsy type 1 patients. • All tested doses of oveporexton helped patients achieve near-normal ranges of wakefulness while addressing the underlying orexin deficiency that causes narcolepsy type 1, with improvements sustained over the 8-week trial period. • The drug was generally well-tolerated with mostly mild to moderate side effects, and 95% of participants enrolled in the long-term extension study, with Phase 3 trial results anticipated later in 2025.

US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate

8 days ago

• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval. • Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development. • Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.

Takeda's Adzynma Becomes First UK-Approved Treatment for Rare Blood Clotting Disorder cTTP

10 days ago

• The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has approved Takeda's Adzynma (rADAMTS13) as the first treatment for congenital thrombotic thrombocytopenic purpura (cTTP) in the UK for patients of all ages. • Adzynma is an enzyme replacement therapy that provides a recombinant form of ADAMTS13, addressing the underlying enzyme deficiency that causes this ultra-rare inherited blood clotting disorder. • In clinical trials, no patients experienced acute thrombotic thrombocytopenic purpura events while receiving Adzynma as preventative treatment, demonstrating significant efficacy compared to plasma-based therapies.

Global Thrombocytopenia Clinical Trials Market Sees Significant Growth in 2025

10 days ago

• The global thrombocytopenia clinical trials landscape is expanding rapidly with over 25 pharmaceutical companies developing 25+ treatment therapies, according to recent market analysis. • Key industry players including GSK, Novartis, Amgen, and Sanofi are leading clinical development efforts, with several promising candidates in late-stage trials showing efficacy in reducing thrombocytopenia events. • Recent advances include Takeda's TAK-755 demonstrating 60% reduction in thrombocytopenia events compared to standard of care, and HUTCHMED completing enrollment for its pivotal Phase III ESLIM-01 trial of sovleplenib for immune thrombocytopenia.

FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval

a month ago

• The FDA has asked Novavax to conduct an additional randomized, controlled study of its COVID-19 vaccine NVX-CoV2601 before considering full approval, despite the vaccine having emergency use authorization since 2022. • Interim results from a phase 2/3 study showed Novavax's updated XBB.1.5 variant vaccine generated 5.8 times higher neutralizing antibody levels compared to the original formulation, with a favorable safety profile. • The FDA's request represents a significant setback for Novavax, which had expected approval based on prior conversations with regulators and had already passed its April 1 PDUFA date.

Novartis Announces $23 Billion Investment to Expand US Manufacturing and R&D Footprint

a month ago

• Novartis plans to invest $23 billion over five years to establish seven new facilities and expand three existing ones across the United States, enabling 100% end-to-end US production of key medicines. • The expansion includes a $1.1 billion research hub in San Diego and new radioligand therapy manufacturing facilities in Florida and Texas, creating approximately 5,000 jobs directly and indirectly. • This strategic investment reverses Novartis' recent US pullback and comes amid trade policy uncertainties, positioning the company to maintain its projected sales growth of +5% CAGR through 2029.

XOMA Royalty Expands Portfolio to Over 120 Assets, Reports Financial Results for 2024

2 months ago

• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.

AstraZeneca's Eneboparatide Meets Primary Endpoint in Phase III Trial for Hypoparathyroidism

2 months ago

• Eneboparatide, an investigational parathyroid hormone receptor agonist, successfully normalized serum calcium levels and eliminated the need for standard supplementation in adults with chronic hypoparathyroidism at 24 weeks. • The CALYPSO Phase III trial enrolled 202 patients and will continue to 52 weeks to further characterize the drug's long-term safety and efficacy profile, with full data to be shared with global health authorities. • Hypoparathyroidism affects over 200,000 people in the US and EU, with approximately 80% being women, representing a significant unmet medical need despite existing treatments.

GSK's Blenrep Secures UK Approval for Multiple Myeloma Treatment in Combination Therapy

2 months ago

• The UK's medicines regulatory body has approved GSK's Blenrep (belantamab mafodotin) in combination with other drugs for multiple myeloma patients whose first treatment failed or caused severe side effects. • This approval marks a significant comeback for Blenrep, which was withdrawn from markets in 2022 after failing to outperform existing treatments when used as monotherapy. • Clinical trials demonstrated Blenrep's combination therapy extended progression-free survival and overall survival compared to standard care regimens, including those based on Darzalex (daratumumab).

Roche Expands Molecular Glue Portfolio with $2 Billion Monte Rosa Partnership

2 months ago

• Roche has signed a $2 billion partnership with Monte Rosa Therapeutics, including a $50 million upfront payment, to develop molecular glue therapies targeting previously "undruggable" proteins in cancer and neurological diseases. • This marks Roche's second major molecular glue deal in a month, following a similar $2 billion alliance with Orionis Biosciences, demonstrating the company's strategic push to establish leadership in this emerging therapeutic category. • Monte Rosa will lead discovery and preclinical activities using its QuEEN platform, with Roche taking over late-stage development of candidates that can potentially address the 80% of human proteins currently inaccessible to traditional drug development approaches.

Robust Pipeline for Nasopharyngeal Cancer Treatment Emerges with 25+ Pharmaceutical Companies Developing Novel Therapies

3 months ago

• A comprehensive pipeline analysis reveals over 25 pharmaceutical companies actively developing approximately 30 drug candidates for nasopharyngeal cancer, signaling significant industry investment in this therapeutic area. • BL-B01D1, a first-in-class bispecific antibody-drug conjugate targeting both EGFR and HER3, has advanced to Phase III clinical trials, showing promise for patients with recurrent or metastatic nasopharyngeal carcinoma. • Novel approaches in development include Takeda's TAK-500, a STING agonist immunostimulatory antibody drug conjugate, and Ascentage Pharma's APG-5918, highlighting diverse therapeutic strategies targeting this challenging cancer.

Phase 4 KINECT-PRO Trial Shows Ingrezza Significantly Improves Quality of Life in Tardive Dyskinesia Patients

3 months ago

• Neurocrine Biosciences' phase 4 KINECT-PRO study demonstrates sustained improvements in physical, social, and emotional impacts of tardive dyskinesia with Ingrezza treatment across all severity levels. • The 24-week trial showed significant patient-reported benefits starting as early as 4 weeks at the lowest dose (40mg), with 52 out of 59 enrolled patients completing the study. • Results revealed consistent efficacy regardless of underlying psychiatric conditions, with improvements measured across multiple validated clinical scales including AIMS and TD Impact Scale.

FDA Clears Northwestern University's IND Application for Calidi's Novel Glioma Treatment CLD-101

4 months ago

• The FDA has cleared Northwestern University's IND application for Calidi Biotherapeutics' CLD-101, a stem cell-based platform delivering oncolytic viruses for treating high-grade glioma, with Phase 1b/2 trials starting late 2024. • Previous Phase 1 trials demonstrated promising results with median progression-free survival of 9.05 months and overall survival of 18.4 months, showing no dose-limiting toxicity. • The treatment addresses a critical unmet need in high-grade glioma patients, who currently face a challenging five-year survival rate of only 5-10% with conventional therapies.

FDA Approves Lilly's Omvoh (mirikizumab) for Crohn's Disease

4 months ago

• The FDA has approved Eli Lilly's Omvoh (mirikizumab-mrkz) for treating moderately to severely active Crohn's disease in adults, expanding its use for inflammatory bowel disease. • The approval was based on the VIVID-1 trial, which showed significant clinical remission and endoscopic response rates compared to placebo after one year of treatment. • Omvoh is the first biologic in over 15 years with Phase 3 two-year efficacy data at launch, demonstrating long-term disease control and visible healing of the intestinal lining. • Lilly has submitted marketing applications globally, aiming to provide a new treatment option for Crohn's patients and is working with insurers to enable patient access.

Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments

4 months ago

• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions. • Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways. • These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets. • The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.

FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric Steroid-Refractory Acute Graft-versus-Host Disease

5 months ago

• The FDA has approved Ryoncil (remestemcel-L-rknd) as the first mesenchymal stromal cell (MSC) therapy for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients. • Ryoncil's approval was based on a Phase III trial demonstrating a 70% overall response rate in children with SR-aGVHD after 28 days of treatment. • This allogeneic, bone marrow-derived MSC therapy offers a new treatment option for children with SR-aGVHD who do not respond to steroid treatment. • Ryoncil is administered intravenously and should be monitored for infusion reactions; common side effects include infections, fever, hemorrhage, and abdominal pain.

GSK's Blenrep Demonstrates Significant Survival Benefit in Multiple Myeloma Trial, Paving Way for Potential Comeback

6 months ago

• GSK's Blenrep, combined with chemotherapy and a steroid, reduced the risk of death by 42% compared to a standard-of-care treatment in relapsed or refractory multiple myeloma. • The DREAMM-7 trial data showed a projected median overall survival of 84 months for the Blenrep combination versus 51 months for the comparator arm. • Blenrep, an antibody-drug conjugate targeting BCMA, is under regulatory review in multiple regions, potentially offering a new treatment option for myeloma patients. • Despite previous market withdrawal due to a failed trial, Blenrep's new data suggests a possible paradigm shift in treating relapsed or refractory multiple myeloma.

Eli Lilly's Kisunla (Donanemab) Receives Approval in Japan for Early Alzheimer's Disease

8 months ago

• Eli Lilly's Kisunla (donanemab-azbt) has been approved in Japan for treating early symptomatic Alzheimer's disease, including mild cognitive impairment. • The approval is based on the Phase III TRAILBLAZER-ALZ 2 study, which demonstrated a significant reduction in clinical decline in early-stage patients. • Kisunla aims to remove amyloid plaques, potentially allowing patients to discontinue treatment once plaque clearance is achieved, reducing infusion burden. • Japan is the second major market to approve Kisunla, offering a new treatment option for the country's rapidly aging population with rising dementia cases.