This Week in Biotech #21
Catch up on the latest biotech breakthroughs and upcoming trends (Oct 18-22).
Welcome to the Wednesday edition of This Week in Biotech by Biotech Blueprint!
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MARKET UPDATES
🔹 Sangamo Therapeutics (SGMO) announced that the FDA has provided a clear path for accelerated approval of its gene therapy ST-920 for Fabry disease, using data from its ongoing phase 1/2 STAAR study. The FDA confirmed that one-year estimated glomerular filtration rate slope data can serve as the primary basis for approval, avoiding the need for an additional study. Sangamo expects to submit a Biologics License Application (BLA) in the second half of 2025, potentially accelerating approval by three years. Following this news, Sangamo’s stock surged 32% today.
🔹 Regeneron Pharmaceuticals (REGN) failed in its attempt to block the U.S. launch of Amgen’s biosimilar to Eylea, a drug used to treat eye diseases like macular degeneration. A U.S. appeals court denied Regeneron’s request to prevent Amgen from launching its biosimilar, Pavblu, while Regeneron appeals a West Virginia court’s earlier ruling in Amgen’s favor. Regeneron claims Amgen is infringing on its patents and will continue pursuing legal action. Following the decision, Regeneron’s stock dropped by 2% on Oct. 22. Meanwhile, Amgen plans to launch Pavblu swiftly, aiming to offer a more affordable treatment option. Eylea earned $5.89B in U.S. sales for Regeneron in 2023.
🔹 On Oct. 22, Immunic (IMUX) announced a positive outcome from the interim analysis of its phase 3 ENSURE program, which is evaluating vidofludimus calcium (IMU-838) for multiple sclerosis. An Independent Data Monitoring Committee reviewed unblinded data and confirmed that the trials are not futile, recommending they continue as planned without changes, including no need for a potential upsizing of the trial. The program remains on track for completion in 2026. Despite this positive development, Immunic’s stock dropped by 10% following the announcement. The company remains optimistic about vidofludimus calcium’s potential to transform the multiple sclerosis market and continues progressing towards regulatory approval.
🔹 Bright Minds Biosciences (DRUG) has experienced a massive stock surge, rising over 2000% in the past five days and 4500% over the month, climbing from $2.55 to $54 per share. This growth is fueled by a combination of positive developments, including a new $35M private placement and a collaboration with Firefly Neuroscience (AIFF). Together, they are working on a phase 2 clinical trial for BMB-101, a selective 5-HT2C receptor agonist aimed at treating absence epilepsy and developmental epileptic encephalopathy, conditions characterized by treatment-resistant seizures. Firefly’s AI-driven Brain Network Analytics platform will provide in-depth EEG data analysis, helping assess the drug’s efficacy. The collaboration builds on their successful previous partnership, where Firefly’s technology was used to analyze BMB-101 in earlier trials.
BIOTECH NEWS
🔹 Roche is cutting ties with UCB’s anti-tau Alzheimer’s drug bepranemab, walking away from a 2020 deal that included $120M upfront investment and potential $2B in milestones. The decision comes just before UCB is set to present phase 2a data at the 2024 Clinical Trials on Alzheimer’s Disease meeting. Despite UCB describing the data as encouraging, Roche has decided it isn’t promising enough to justify continuing. This marks the second time this year Roche has dropped a tau-targeting candidate, highlighting the high-stakes uncertainty in the Alzheimer’s drug race.
🔹 On Oct. 22, Editas Medicine announced it has achieved in vivo preclinical proof of concept for editing hematopoietic stem and progenitor cells using its proprietary lipid nanoparticle technology. This paves the way for developing in vivo therapies for genetic blood disorders like sickle cell disease and beta thalassemia. Additionally, the company revealed plans to seek partnerships or out-licensing opportunities for its reni-cel therapy, shifting focus to its expanding in vivo pipeline.
🔹 On Oct. 22, Aligos Therapeutics received FDA clearance for the Investigational New Drug (IND) application for ALG-000184, a capsid assembly modulator targeting chronic hepatitis B. This paves the way for a phase 1 study, which will evaluate ALG-000184’s interaction with other drugs. Aligos plans to file for phase 2 trials in early 2025. ALG-000184 is an oral small molecule aimed at inhibiting multiple components of the HBV lifecycle, potentially offering superior viral suppression compared to current therapies.
🔹 On Oct. 22, Alvotech and Teva Pharmaceuticals announced that the FDA approved a new intravenous formulation of Selarsdi, a biosimilar to J&J’s Stelara, for the treatment of Crohn’s disease and ulcerative colitis. This expands Selarsdi’s label to match the reference product’s indications. The FDA had previously approved Selardsi in prefilled syringes for treating plaque psoriasis and psoriatic arthritis in April 2024.
🔹 Eli Lilly has filed more lawsuits, this time against three medical spas and online vendors—Pivotal Peptides, MangoRx, and Genesis Lifestyle Medicine—for selling unauthorized products claiming to contain tirzepatide, the active ingredient in its popular weight-loss drug Zepbound. These companies allegedly sold tirzepatide without proper medical supervision or FDA approval, with some offering unapproved oral versions. Eli Lilly accuses them of false advertising, risking patient safety, and misleading consumers about efficacy and safety. The lawsuits, filed in multiple states, aim to stop the sale of these copycat products and seek monetary damages.
🔹 On Oct. 21, Novo Nordisk’s recent SOUL trial results reveal that its oral diabetes drug, Rybelsus (semaglutide), reduced the risk of major cardiovascular events by 14% in type 2 diabetes patients with heart or kidney complications. The study, involving 9,650 participants, shows Rybelsus not only manages diabetes but also lowers the chances of life-threatening events like heart attacks and strokes. Novo Nordisk plans to seek regulatory approval in the U.S. and E.U. in 2025, aiming to expand the drug’s use beyond diabetes to other areas like Alzheimer’s and alcohol-related disorders, positioning it as a versatile growth driver.
🔹 On Oct. 21, Regenxbio presented positive results from a phase 2 study of ABBV-RGX-314, a gene therapy for bilateral macular degeneration, at the 2024 American Academy of Ophthalmology meeting. Highlights include a 97% reduction in treatment burden at nine months post-treatment, with 78% of patients remaining injection-free. ABBV-RGX-314 was well-tolerated, showing no cases of intraocular inflammation without the use of prophylactic steroids.
🔹 On Oct. 18, Lykos Therapeutics, led by interim CEO Michael Mullette and new Chief Medical Officer Dr. David Hough, announced a productive meeting with the FDA regarding its lead drug, midomafetamine capsules (MDMA), aimed at treating PTSD. Despite the FDA’s earlier rejection in August, the agency provided a path forward, which includes conducting an additional phase 3 trial and a potential independent third-party review of prior clinical data. Midomafetamine, based on the psychedelic drug MDMA, offers hope as the first new PTSD treatment option in nearly 25 years if ultimately approved. Lykos says it will continue working with the FDA and provide updates on their progress.
🔹 On Oct. 18, Allergan Aesthetics, an AbbVie company, announced FDA approval for botox to temporarily improve the appearance of moderate to severe platysma bands (vertical bands connecting the jaw and neck) in adults. This makes botox approved for four areas: forehead lines, frown lines, crow’s feet, and now platysma bands. The treatment reduces underlying muscle activity in these bands, enhancing jawline and neck appearance. Phase 3 clinical studies showed significant improvements, with patients reporting high satisfaction rates. This new approval expands treatment options for patients seeking non-surgical solutions for neck aesthetics.
🔹 On Oct. 18, Gilead announced its plan to voluntarily withdraw the U.S. accelerated approval for Trodelvy in treating metastatic urothelial cancer in adult patients. This decision, made in consultation with the FDA, is due to the TROPiCS-04 study not meeting the primary endpoint of overall survival. Other approved uses of Trodelvy, including in breast cancer, remain unchanged. Trodelvy remains approved in nearly 50 countries for other cancer indications and is being studied for various solid tumors.
🔹 On Oct. 18, the FDA imposed a partial clinical hold on BioNTech’s phase 3 trial of BNT316/ONC-392, an investigational lung cancer treatment, being developed in partnership with OncoC4. The trial is focused on treating metastatic non-small cell lung cancer patients who previously received PD-(L)1-inhibitor treatments. This partial hold was triggered due to varying outcomes between squamous and non-squamous NSCLC patients. The hold affects new patient enrollment, but current patients in the trial will continue treatment. Earlier in the year, BioNTech’s phase 1 trial for another treatment, BNT326/YL202, also experienced a partial hold but resumed recruitment in August after adjusting the dosage for safety.
CLINICAL TRIAL UPDATES
🔹 On Oct. 21, Nurix Therapeutics presented positive clinical trial results for its BTK degrader, NX-5948, in patients with relapsed/refractory Waldenstrom’s Macroglobulinemia, a type of cancer affecting B cells. The trial showed that 77.8% (7/9) of evaluable patients achieved objective responses, with two remaining on treatment for over a year. NX-5948 demonstrated a durable and deepening response, particularly benefiting patients with prior treatments, including BTK inhibitors and chemotherapy. The drug’s brain-penetrant properties may also offer advantages for WM cases with CNS involvement. These findings support NX-5948’s advancement into the phase 1b trial.
🔹 On Oct. 19, Gilead Sciences and Merck announced positive results from a phase 2 study of an investigational once-weekly oral combination of islatravir and lenacapavir for HIV treatment. The study showed that after 48 weeks, 94.2% of participants maintained viral suppression, with no viral load rebound, comparable to those continuing daily Biktarvy treatment. The combination was well-tolerated, with mild treatment-related adverse events, and no significant safety concerns. This novel regimen is advancing to phase 3 trials and has the potential to become the first weekly oral HIV treatment, offering a less frequent alternative to daily therapy.
🔹 Aura Biosciences announced promising early results from its ongoing phase 1 trial of bel-sar (AU-011) for bladder cancer. In the trial, which includes 13 patients, 4/5 patients with low-grade disease achieved clinical complete responses following a single low dose of bel-sar combined with light activation, showing no remaining tumor cells. Additionally, 2/3 patients with high-grade disease exhibited visual tumor shrinkage. The treatment demonstrated a favorable safety profile, with less than 10% of patients reporting mild adverse events and no serious side effects. The data also revealed evidence of immune activation, indicated by CD8+ T cell infiltration in both target and non-target tumors, suggesting a broader immune response.
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