NOVARTIS

Scholar Rock's apitegromab, a muscle-preserving therapy for spinal muscular atrophy, succeeded in a Phase 3 trial, showing significant motor function improvement when combined with standard SMA therapies. The company plans to seek approvals in the U.S. and Europe early next year. The drug's success has implications for ongoing research into muscle preservation and potential treatments for obesity.

AstraZeneca entered an exclusive licensing deal with CSPC Pharmaceutical Group to develop a preclinical lipid-lowering drug candidate, YS2302018, aiming to treat dyslipidemia and related cardiometabolic diseases. The deal includes $100 million upfront and up to $1.92 billion in milestones, with CSPC eligible for royalties. YS2302018 is an oral small molecule disruptor of Lp(a), a type of LDL cholesterol linked to higher heart attack and stroke risks. AstraZeneca joins Eli Lilly and others in the Lp(a) drug development arena.

The report on the Personalized Medicine Market by Vantage Market Research highlights market size, forecast, opportunities, key drivers, regulatory scenario, industry trends, new product approvals, competitive landscape, and regional analysis. Key players include Abbott Laboratories, Merck & Co., AstraZeneca, Pfizer, GlaxoSmithKline, Novartis, Amgen, Bayer, Eli Lilly, Illumina, Danaher, QIAGEN, 23andMe, Roche, Thermo Fisher Scientific, GE Healthcare, and Precision Biologics. Market types cover diagnostics, therapeutics, medical care, and nutrition, while applications include oncology, neurology, cardiology, antiviral, psychiatry, immunology, and others. The report also examines COVID-19's impact on the market.

Myricx Bio announces Paolo Paoletti joining its Board as an independent Non Executive Director, following a £90m series A financing. Paoletti, with extensive oncology experience, previously led GSK Oncology and GammaDelta Therapeutics, and is currently involved in several life science boards and committees.

Health Canada approves Illuccix for selecting patients with progressive metastatic castration-resistant prostate cancer (mCRPC) for PSMA-targeted radionuclide therapy, expanding its clinical utility in Canada.

The PQAT-RW, a generic instrument, assesses patient-perceived benefits and risks/harms of treatment within the same instrument at the individual level, providing a holistic picture of patients’ evaluation of treatment. It includes both free-text response options and predefined response scales, allowing patients to provide information in their own words and identify previously unknown aspects of a treatment. The PQAT-RW was developed by adapting the PQATv2 for real-world settings and was content validated in a patient population with diverse chronic conditions. Findings demonstrate its value in gathering individualized data about patients’ perceived benefits and disadvantages of a specific treatment, evaluating patients’ perception of the balance between benefits and disadvantages, and its impact on treatment continuation. The PQAT-RW is suitable for various real-world settings, facilitating incorporation of patient perspectives into regulatory and payer decisions, improving communication between patients and clinicians, and identifying factors influencing treatment adherence.

Pharmaceutical companies are increasingly collaborating with academic centers for early-stage drug discovery to mitigate rising R&D costs and risk exposure, as blockbuster drugs face generic competition and drug development becomes more complex.

Pelabresib combined with ruxolitinib significantly reduced splenomegaly and improved anemia in JAKi-naïve myelofibrosis patients, according to the phase 3 MANIFEST-2 trial. The combination therapy showed substantial spleen volume reduction, symptom relief, and anemia management, with manageable adverse effects.

Real-world outcomes of relapsed/refractory multiple myeloma patients treated with cilta-cel showed 89% overall response rate and 70% complete response, despite many being ineligible for the trial leading to its approval. Median 12-month progression-free survival was 68% and overall survival 82%, with similar serious side effects reported.

E. Heath reports advisory/consulting, steering committee, honoraria/paid travel, speaker’s bureau, and research support from various companies. J.R. Ribeiro, K. Poorman, and J. Xiu report personal fees and other support from Caris Life Sciences. H. Mamdani reports other support and grants from Daiichi Sankyo, AstraZeneca, and Genentech. A.F. Shields reports personal fees from Caris Life Sciences. G.L. Lopes reports stock ownership, honoraria, consulting, research funding, travel, and other relationships with multiple companies. S.A. Kareff reports personal fees and travel grants from various organizations. M. Radovich and G.W. Sledge report other support from Caris Life Sciences. G.A. Vidal reports relationships with Guardant360, Gilead, BillionToOne, Genentech/Roche, GSK, and AstraZeneca. J.L. Marshall reports personal fees from Caris. No other disclosures were reported by the other authors.