GENENTECH, INC.
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Subsidiary
- Established
- 1976-01-01
- Employees
- 11.1K
- Market Cap
- -
- Website
- http://www.gene.com
SSc-ILD Market Projected to Reach $750 Million by 2034 as Novel Therapies Enter Pipeline
• The Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD) market was valued at approximately $750 million in 2022 and is expected to grow significantly through 2034, driven by increasing disease prevalence and emerging therapies.
• aTyr Pharma continues enrollment in its Phase 2 EFZO-CONNECT study evaluating efzofitimod for SSc-ILD, with participants receiving either 270mg, 450mg, or placebo administered intravenously over six monthly doses.
• Several promising candidates in the pipeline, including PRA023, Belimumab, and Vixarelimab, are poised to reshape the treatment landscape for SSc-ILD, addressing significant unmet needs in this serious pulmonary complication.
Rivus Pharmaceuticals Strengthens Leadership Team Ahead of Critical Phase 2b MASH Trial Results
• Rivus Pharmaceuticals has appointed David Grainger, Ph.D., as Chairman of Development and Meg Fitzgerald, J.D., as Chief Legal Officer while establishing a new office in South San Francisco.
• The company is advancing its novel Controlled Metabolic Accelerators (CMAs), led by HU6, which has shown promising Phase 2a results in treating obesity-related conditions without muscle mass loss.
• Rivus is approaching a critical milestone with the imminent readout of its Phase 2b M-ACCEL study in metabolic dysfunction-associated steatohepatitis (MASH), potentially advancing HU6 to pivotal trials.
Sangamo Therapeutics Advances Neurology Pipeline and Fabry Disease Gene Therapy Amid Financial Restructuring
• Sangamo Therapeutics has secured FDA clearance for its first neurology IND targeting idiopathic small fiber neuropathy, with patient enrollment expected to begin in mid-2025 and preliminary efficacy data anticipated by Q4 2026.
• The company's Fabry disease gene therapy has demonstrated sustained benefits with a positive mean eGFR slope of 3.061 mL/min/1.73m² in 23 patients, with all 18 patients who began on enzyme replacement therapy successfully withdrawn and remaining off treatment.
• Despite reducing non-GAAP operating expenses by nearly half year-over-year and raising over $100 million in 2024, Sangamo faces financial challenges with cash runway extending only into mid-Q2 2025 as it seeks commercialization partners for key programs.
Adverum Launches Groundbreaking Phase 3 ARTEMIS Trial for One-Time Gene Therapy in Wet AMD
• Adverum Biotechnologies initiates ARTEMIS, the first-ever registrational intravitreal gene therapy trial for wet AMD, evaluating Ixo-vec against standard aflibercept treatment in 284 patients.
• The innovative trial design includes both treatment-naïve and previously treated patients, comparing a single Ixo-vec injection to regular aflibercept dosing with a primary endpoint of non-inferior vision outcomes.
• Current data shows 42% of wet AMD patients discontinue treatment after 2 years, highlighting the potential impact of Ixo-vec's one-time administration approach for long-term vision preservation.
Relay Therapeutics to Launch Phase 3 Trial for RLY-2608 in Advanced Breast Cancer Mid-2025
• Relay Therapeutics plans to initiate ReDiscover-2, a Phase 3 trial evaluating RLY-2608 plus fulvestrant in PI3Kα-mutated, HR+/HER2- advanced breast cancer patients, with enrollment target of 540 patients.
• Interim Phase 1b data showed promising results with RLY-2608 plus fulvestrant achieving 11.4-month median progression-free survival in second-line treatment and 39% confirmed overall response rate.
• The company maintains strong financial position with approximately $780 million in cash reserves as of Q4 2024, sufficient to fund operations into second half of 2027.
Novel Spinal Cord Stimulation Shows Promise in Treating Spinal Muscular Atrophy
• A groundbreaking pilot study demonstrates that spinal cord implants delivering low-level electrical stimulation helped three adults with spinal muscular atrophy regain muscle function and mobility.
• Participants showed significant improvements in walking distance, gait length, and standing ability, with one patient achieving a threefold increase in step length during the month-long trial.
• The benefits persisted temporarily even after device deactivation, suggesting potential lasting effects, though the experimental implants were removed after the short-term study.
Hinge Bio Secures $30 Million to Advance GEM-DIMER Platform for Autoimmune Therapies
• Hinge Bio has raised $30 million in a Series A financing round to support the development of its GEM-DIMER platform for autoimmune disease therapies.
• The funding will facilitate the entry of Hinge Bio's lead candidate, HB2198, into a Phase 1 clinical trial for Systemic Lupus Erythematosus in 2025.
• HB2198 targets CD19 and CD20 to deplete B cells, aiming for a reset of the immune system with a convenient, off-the-shelf antibody-based therapeutic.
• Preclinical data suggests HB2198 achieves deeper and more rapid B cell depletion compared to existing antibody-based therapies, offering potential advantages.
Genprex's Reqorsa Advances in Phase 2 Trial for Small Cell Lung Cancer
• Genprex's Acclaim-3 trial, evaluating Reqorsa with Tecentriq for extensive stage small cell lung cancer (ES-SCLC), has advanced to Phase 2.
• The Phase 1 dose escalation showed a favorable safety profile, leading to the selection of 0.12 mg/kg as the Recommended Phase 2 Dose (RP2D).
• The Phase 2 expansion will enroll approximately 50 patients to determine the 18-week progression-free survival rate.
• Reqorsa, a gene therapy expressing the TUSC2 protein, has received Fast Track and Orphan Drug Designations from the FDA for SCLC treatment.
Health Canada Approves Celltrion's Omlyclo, First Omalizumab Biosimilar
• Health Canada has approved Omlyclo, Celltrion's biosimilar of omalizumab, for allergic asthma, chronic rhinosinusitis with nasal polyps, and chronic idiopathic urticaria.
• The approval was based on a Phase III trial demonstrating bioequivalence to Xolair in efficacy, safety, pharmacokinetics, and immunogenicity in CSU patients.
• Celltrion also received positive CHMP opinions for biosimilars of aflibercept, denosumab, and tocilizumab, expanding its biosimilar portfolio in Europe.
• Omlyclo is under review with the FDA, potentially offering a more accessible treatment option for various allergic conditions in the US market.
FDA Approvals of Ribociclib and Inavolisib Reshape HR+ Breast Cancer Treatment
• Ribociclib's approval for adjuvant use in high-risk HR+/HER2- early breast cancer marks a significant advancement, potentially benefiting a broader patient population.
• Inavolisib, combined with palbociclib and fulvestrant, gains FDA approval for endocrine-resistant, PIK3CA-mutated HR+/HER2- advanced breast cancer, improving progression-free survival.
• The FDA emphasizes the need for increased diversity in clinical trials and post-marketing studies to ensure the safety and efficacy of new drugs across all patient populations.
• Dose optimization strategies, like those promoted by Project Optimus, are crucial for balancing efficacy and toxicity in novel targeted therapies for breast cancer.
Sozinibercept Shows Promise in Improving Visual Outcomes for nAMD Patients
• Sozinibercept, a novel VEGF-C/D inhibitor, is under investigation for neovascular age-related macular degeneration (nAMD) to improve visual acuity beyond current VEGF-A therapies.
• Phase 2b trials showed that sozinibercept combined with ranibizumab led to a statistically significant additional gain of 3.4 letters in BCVA compared to ranibizumab alone at 24 weeks.
• Ongoing Phase 3 trials (ShORe and COAST) are evaluating sozinibercept in combination with ranibizumab or aflibercept, with potential regulatory filings planned after 12-month efficacy analysis.
• Next-generation nAMD therapies like sozinibercept aim to target multiple pathways beyond VEGF-A to enhance vision and quality of life for patients, addressing the limitations of current treatments.
FDA Approves Inavolisib Plus Palbociclib and Fulvestrant for PIK3CA-Mutated, HR+/HER2- Advanced Breast Cancer
• The FDA approved inavolisib in combination with palbociclib and fulvestrant for adults with endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative advanced or metastatic breast cancer.
• The approval was based on the INAVO120 trial, which demonstrated a significant improvement in progression-free survival compared to placebo plus palbociclib and fulvestrant.
• Patients receiving the inavolisib regimen achieved an objective response rate of 58% compared to 25% in the placebo arm, with a median duration of response of 18.4 months vs 9.6 months, respectively.
• Inavolisib's manageable toxicity profile, particularly in patients without diabetes or glucose intolerance, makes it a valuable option for those with poor prognosis, hormone receptor-positive breast cancer.
SMA Clinical Trials Update: Investigational Therapies and Expanded Risdiplam Studies
• Several companies are advancing novel therapies for spinal muscular atrophy (SMA), with over 20 treatments currently in clinical development.
• Roche is conducting a Phase IV open-label study to assess the effectiveness and safety of risdiplam in pediatric SMA patients following gene therapy.
• Biogen is evaluating higher doses of intrathecally administered nusinersen to improve clinical efficacy in SMA patients.
• Novartis is studying the safety and efficacy of intrathecal OAV101 in SMA patients who have discontinued nusinersen or risdiplam treatment.
Experts Highlight Key Trends and Challenges in Rare Disease Drug Development for 2024
• The rare disease therapeutics landscape shows promising momentum in 2024, with three orphan drug approvals already achieved and approximately 50% of approved drugs targeting rare conditions.
• The historic FDA approval of CRISPR gene therapy in late 2023 marks a significant milestone, with in-vivo CRISPR therapies and dual AAV gene therapy approaches showing promise for future treatments.
• Clinical trial recruitment remains a major challenge for rare disease research due to small patient populations, geographic dispersion, and new FDA diversity requirements, though patient advocacy groups are helping bridge these gaps.
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