The week witnessed significant developments in the pharmaceutical sector, encompassing regulatory actions, setbacks, and promising outcomes. The FDA issued a reprimand against a major pharmaceutical company for disseminating false and misleading claims about its non-small cell lung cancer drug. Concurrently, a developer of MDMA therapy for PTSD experienced a considerable obstacle. In the drug development domain, the acute myeloid leukemia candidate, Iomab-B, suffered a critical setback, whereas a drug candidate for Duchenne Muscular Dystrophy showed promising results in clinical trials. The healthcare sector also saw a rise in acquisitions and licensing deals, yet faced hurdles as multiple companies announced layoffs as part of their restructuring initiatives.### Layoffs and Impact on R&DPrecigen Inc. (PGEN), a biopharmaceutical entity focusing on gene and cell therapies, announced a 20% reduction in its workforce on Wednesday. This decision forms part of a strategic shift aimed at prioritizing its clinical portfolio and optimizing resources. The company is now redirecting its efforts towards the potential commercialization of PRGN-2012, an AdenoVerse gene therapy for treating recurrent respiratory papillomatosis. This therapy is on course for a rolling Biologics License Application (BLA) submission under an accelerated approval pathway.PGEN shares closed at $1.10 on Friday, marking a 13.39% drop.Entero Therapeutics Inc. (formerly First Wave BioPharma, Inc.) (ENTO) is taking drastic measures by terminating all non-essential employees and vacating its Boca Raton office. Alongside these steps, the company has halted all non-essential research and development activities. CEO James Sapirstein and Board members Charles Casamento and Terry Coelho are also departing. These actions are in response to a Default notice from Mattress Liquidators Inc., which demands immediate repayment of all outstanding obligations.ENTO shares closed at $0.23 on Friday, reflecting a 3.80% increase.AN2 Therapeutics Inc. (ANTX) plans to cut approximately 50% of its workforce as part of a strategic restructuring to extend its cash runway through 2027. The company will discontinue the EBO-301 study, a phase II/III trial evaluating Epetraborole for treatment-refractory mycobacterium avium complex (MAC) lung disease due to underwhelming results.ANTX shares closed at $1.03 on Friday, representing a nearly 61% decline.### FDA Decisions and WarningsARS Pharmaceuticals Inc. (SPRY) has received FDA approval for neffy, the first and only needle-free treatment for type I allergic reactions, including anaphylaxis, in adults and children weighing at least 30 kg. Traditionally, epinephrine administered intramuscularly via an auto-injector has been the standard treatment for anaphylaxis. The approval of neffy, an intranasal epinephrine product, marks the first significant advancement in epinephrine delivery in over three decades. The product is expected to be available in the U.S. within eight weeks.SPRY shares closed at $10.88 on Friday, up by 10.01%.The FDA has declined to approve Lykos Therapeutics’ Midomafetamine capsules for treating post-traumatic stress disorder in adults, requesting another phase III trial to gather more safety and efficacy data. The company plans to address the FDA’s concerns in the coming months and will update on the resubmission of the drug candidate. Midomafetamine, commonly known as MDMA or Ecstasy, remains a Schedule I drug under the Controlled Substances Act, prohibiting its recreational or medical use.Lykos Therapeutics is a public benefit corporation established by the Multidisciplinary Association for Psychedelic Studies.Bristol Myers Squibb Co. (BMY) received a letter from the FDA’s Office of Prescription Drug Promotion for making false or misleading claims regarding Krazati. Initially granted accelerated approval for locally advanced or metastatic non-small cell lung cancer (NSCLC) with a KRASG12C mutation in December 2022, the company’s website misleadingly implied that Krazati improved the disease control rate and “depth of response” in patients, despite the study not being designed to demonstrate this. Krazati was granted accelerated approval for adult patients with KRASG12C-mutated locally advanced or metastatic colorectal cancer in June this year.BMY shares closed at $46.72 on Friday, down by 0.28%.On Tuesday, the FDA approved Servier’s Vorasidenib for Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. Following the acquisition of Agios Pharmaceuticals’ oncology business in 2021, Vorasidenib is the first and only FDA-approved targeted treatment for Grade 2 IDH-mutant glioma. The drug is priced at $39,881 per month.Novartis AG (NVS) announced on Thursday that it has received accelerated approval from the FDA for Fabhalta, a groundbreaking complement inhibitor designed to reduce proteinuria in adults with immunoglobulin A nephropathy (IgAN) who face a high risk of rapid disease progression. IgAN is a progressive and rare autoimmune disease where the immune system attacks the kidneys, leading to glomerular inflammation and proteinuria. This marks the second accelerated approval for Fabhalta; last December, the FDA approved the drug for treating adults with paroxysmal nocturnal hemoglobinuria, a rare blood disorder.NVS concluded Friday’s trading session at $111.89, showing an increase of 0.78%.### Acquisition Trends and Merger StrategiesG1 Therapeutics Inc. (GTHX), a commercial-stage oncology company, announced on Wednesday an agreement to be acquired by privately-held Pharmacosmos A/S for $7.15 per share in cash, totaling roughly $405 million in equity value. This acquisition aims to expand and strengthen Pharmacosmos’s global commercial portfolio, especially in developing treatments for iron deficiency and iron deficiency anemia. The transaction is expected to close at the end of the third quarter of 2024.GTHX ended Friday’s trading at $7.09.Nevro Corp. (NVRO) disclosed during its Q2 earnings call on Wednesday that it is reviewing all strategic options to diversify, accelerate profitability, and maximize stockholder value. The potential strategies include partnerships, mergers, or even a sale of the company. In the second quarter of 2024, Nevro’s worldwide revenue declined to $104.2 million from $108.8 million in the same period of 2023 due to competitive pressures and ongoing weakness in the U.S. spinal cord stimulation market. For the third quarter of 2024, the company projects worldwide revenue to decline further to a range of $92 million to $94 million.NVRO ended Friday’s trading at $6.01, an increase of 6%.Eisai Co. Ltd. (ESALF.PK) and SEED Therapeutics Inc. announced on Tuesday a strategic research collaboration aimed at discovering and developing novel molecular glue degraders for neurodegenerative and oncology indications. Eisai will pay SEED up to $1.5 billion in upfront and milestone payments, along with tiered royalties, contingent on the exercise of their exclusive rights under the collaboration.ESALF.PK concluded Friday’s trading at $37.84.Recursion Pharmaceuticals Inc. (RXRX) and Exscientia plc (EXAI) disclosed on Thursday a definitive agreement to form a combined entity, creating a technology-first, end-to-end drug discovery platform. Recursion, a clinical-stage biotech company focused on industrializing drug discovery, and Exscientia, a technology-driven clinical-stage drug design and development firm, will merge. Recursion shareholders will own approximately 74%, while Exscientia shareholders will own about 26% of the new combined company, which will retain the name Recursion. The new entity will be headquartered in Salt Lake City, Utah, and continue to trade on NASDAQ while maintaining a significant presence in the U.K.RXRX finished Friday’s trading at $6.29, down 5.27%.Merck (MRK) has agreed to acquire CN201, a novel investigational bispecific antibody for treating B-cell associated diseases, from privately-held Curon Biopharmaceuticals. Merck will pay an upfront amount of $700 million in cash and up to $600 million in milestone payments linked to the development and regulatory approval of CN201. Presently, CN201 is undergoing Phase 1 and Phase 1b/2 clinical trials for treating patients with relapsed or refractory non-Hodgkin’s lymphoma (NHL) and relapsed or refractory B-cell acute lymphocytic leukemia (ALL). This marks Merck’s fourth acquisition this year.MRK closed Friday’s trading session at $114.55, up 0.55%.Gilead Sciences Inc. (GILD) announced on Friday its decision to pay $320 million to Janssen Pharmaceutica NV to acquire the global royalties for Seladelpar. Seladelpar, a PPARδ agonist currently under FDA review for the treatment of Primary Biliary Cholangitis, is set for an approval decision by August 14, 2024. Gilead secured Seladelpar through its acquisition of CymaBay Therapeutics in February. In 2006, CymaBay entered into an exclusive, worldwide, royalty-bearing license agreement with Janssen, which entitled Janssen to up to an 8% royalty on net sales of PPARδ products. With this buyout, Gilead will no longer owe any royalties to Janssen should Seladelpar receive FDA approval.GILD concluded Friday’s trading session at $73.66, reflecting a decrease of 2.55%.### Breakthroughs and Setbacks**Actinium Pharmaceuticals Inc. (ATNM)** received feedback on Monday from the FDA stating that its Phase III SIERRA trial is insufficient to support a Biologics License Application (BLA) for Iomab-B, despite achieving a statistically significant primary endpoint. Iomab-B is being developed for patients with active, relapsed, or refractory acute myeloid leukemia. The FDA has requested an additional head-to-head randomized clinical trial to demonstrate an overall survival benefit with Iomab-B. Actinium intends to seek a strategic partner for Iomab-B in the U.S.ATNM’s stock has plummeted nearly 67% since the announcement, closing Friday’s trading at $2.05, down almost 15%.**Aldeyra Therapeutics Inc. (ALDX)** reported on Thursday that its Phase III trial of the investigational 0.25% Reproxalap ophthalmic solution for dry eye disease met its primary endpoint. The study showed that Reproxalap was statistically superior to the vehicle in alleviating ocular discomfort, an FDA-accepted symptom of dry eye disease. This marks the first positive Phase III clinical trial in a dry eye chamber with a symptom as the primary endpoint. The FDA had previously declined to approve Reproxalap for dry eye disease last November, requiring an additional trial to demonstrate efficacy in treating symptoms. With the new trial now meeting its goal, Aldeyra plans to resubmit the New Drug Application for Reproxalap this year.ALDX closed Friday’s trading at $5.10, up 21.72%.**Pulse Biosciences Inc. (PLSE)** announced on Thursday the treatment of its first two patients with atrial fibrillation in the Netherlands as part of a first-in-human feasibility study using its novel Cardiac Surgery System for the ablation of cardiac tissue. The procedure was well tolerated, according to Dr. Gan Dunnington, Chief Medical Officer for Cardiac Surgery at Pulse Biosciences. The trial is set to enroll up to 30 patients. The nano-PFA Cardiac Surgical System received FDA Breakthrough Device Designation in early July 2024, and the company plans to seek IDE approval from the FDA to commence U.S. clinical trials.PLSE closed Friday’s trading at $15.65, up 3.64%.**Avidity Biosciences Inc. (RNA)** on Friday reported promising data from its Phase 1/2 clinical trial of Del-zota for treating Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping, known as EXPLORE44. Del-zota is formulated to deliver phosphorodiamidate morpholino oligomers (PMOs) to muscle and heart tissues, enabling dystrophin production. The trial demonstrated outstanding delivery of PMO (200 nM) to skeletal muscle, significantly reduced creatine kinase levels, and achieved a greater than 80% reduction from baseline levels. The study also indicated a statistically significant 37% increase in exon 44 skipping, reaching up to 66%, and a 25% increase to normal dystrophin production, with total dystrophin restoration up to 54% of normal levels.RNA ended Friday’s session at $46.95, up 12.13%.The material has been provided by InstaForex Company – www.instaforex.com
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