Weekly Review: ANTX Downsizes, ALDX Aces Dry Eye Disease Trial, FDA Oks 1st Epinephrine Nasal Spray

(RTTNews) - The week saw FDA reprimanding a pharma giant for making false and misleading claims about its non-small cell lung cancer drug and a developer of MDMA therapy for PTSD facing a major setback. On the drug development front, acute myeloid leukemia drug candidate Iomab-B faced a significant setback while a DMD drug candidate produced exciting data in a study. The healthcare sector saw an increase in acquisitions and licensing deals, while simultaneously facing challenges as several companies announced workforce reduction as part of their restructuring efforts.

Layoffs and Impact on R&D

Precigen Inc. (PGEN), a biopharmaceutical company developing gene and cell therapies, on Wednesday, announced it is reducing 20% of its workforce as part of a strategic reprioritization of its clinical portfolio and streamlining of resources. The company will now focus on potential commercialization of its PRGN-2012 AdenoVerse gene therapy for the treatment of recurrent respiratory papillomatosis. PRGN-2012 is on track for a rolling BLA submission under an accelerated approval pathway.

PGEN closed Friday's trading at $1.10, down 13.39%.

Entero Therapeutics Inc. (formerly First Wave BioPharma, Inc.) (ENTO) is terminating all non-essential employees and is vacating its Boca Raton office. The company has also paused any non-essential research and development activities. The Chief Executive Officer James Sapirstein, and Board members Charles Casamento and Terry Coelho are also among those who are departing the company. These drastic measures are being taken in response to a Default notice from Mattress Liquidators Inc, which has demanded the immediate repayment of all outstanding obligations.

ENTO closed Friday's trading at $0.23, up 3.80%.

AN2 Therapeutics Inc. (ANTX) is all set to terminate approximately 50% of its employee workforce as part of a strategic restructuring to extend cash runway through 2027. The company has decided to discontinue the EBO-301 study, a phase II/III trial, evaluating drug candidate Epetraborole for treatment-refractory mycobacterium avium complex (MAC) lung disease due to lackluster results.

ANTX closed Friday's trading at $1.03, down nearly 61%.

FDA Decisions and Warnings

ARS Pharmaceuticals Inc.'s (SPRY) neffy has been granted FDA approval, becoming the first and only needle-free treatment for type I allergic reactions, including anaphylaxis for adults and children who weigh =30 kg. Epinephrine, generally given as intramuscular (IM) injection, administered by an epinephrine auto-injector (EAI) has so far been the only first-line treatment for anaphylaxis. The approval of neffy, an intranasal epinephrine product, marks the first significant innovation in the delivery of epinephrine in more than 35 years. The product is expected to be available in the U.S. within eight weeks.

SPRY closed Friday's trading at $10.88, up 10.01%.

The FDA has declined to approve Lykos Therapeutics' Midomafetamine capsules for the treatment of post-traumatic stress disorder in adults and has requested the company to run another phase III trial to gather more safety and efficacy data of Midomafetamine. The company intends to focus on addressing the FDA's concerns in the coming months and provide an update regarding the resubmission of the drug candidate. Midomafetamine, widely referred to as MDMA or Ecstasy, is a Schedule I drug under the Controlled Substances Act, prevented from being used for recreational or medical use.

Lykos Therapeutics is a public benefit corporation founded by the Multidisciplinary Association for Psychedelic Studies.

Bristol Myers Squibb Co. (BMY) has been issued a letter by the FDA's Office of Prescription Drug Promotion for making false or misleading claims and representations about the benefits of Krazati, which was granted accelerated approval for locally advanced or metastatic non-small cell lung cancer (NSCLC) with a KRASG12C Mutation in Dec.2022. The company's website misleadingly suggests that Krazati improves disease control rate and "depth of response" in patients with KRAS G12C-mutated locally advanced or metastatic NSCLC, even though the study was not designed to demonstrate this, reveals the OPDP's letter. The drug was granted accelerated approval by the FDA for adult patients with KRASG12C -mutated locally advanced or metastatic colorectal cancer as recently as June of this year.

BMY closed Friday's trading at $46.72, down 0.28%.

The FDA, on Tuesday, approved privately held Servier's Vorasidenib for two types of brain cancer, Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. Vorasidenib came under Servier's fold, following the acquisition of Agios Pharmaceuticals' (AGIO) oncology business in 2021. Voranigo is the first and only FDA-approved targeted treatment in Grade 2 IDH-mutant glioma. The drug is said to cost $39,881 per month.

Novartis AG (NVS), on Thursday, announced it has secured accelerated approval from the FDA for Fabhalta, a first-in-class complement inhibitor for the reduction of proteinuria in adults with immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. IgAN is a progressive, rare disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria. This is the second indication for which the drug has been granted accelerated approval. Last December, Fabhalta was granted accelerated approval by the FDA for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria.

NVS closed Friday's trading at $111.89, up 0.78%.

Deal Or No Deal - Acquisition Trends and Merger Strategies

G1 Therapeutics Inc. (GTHX), a commercial-stage oncology company, on Wednesday, agreed to be acquired by privately held Pharmacosmos A/S, for $7.15 per share in cash for a total equity value of roughly $405 million. The deal is expected to expand and strengthen the global commercial portfolio of Pharmacosmos, which is involved in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anemia. The transaction is slated to close late in the third quarter of 2024.

GTHX closed Friday's trading at $7.09.

Nevro Corp. (NVRO), on Wednesday, during its Q2 earnings call, revealed that it is exploring all possible strategic options as part of an attempt to diversify, accelerate profitability and maximize stockholder value. The strategic options "may include, but are not limited to, partnerships, mergers or even a sale of the company". For the second quarter of 2024, the company's worldwide revenue declined to $104.2 million from $108.8 million in the second quarter of 2023, hurt by competitive dynamics and ongoing softness in de novo U.S. spinal cord stimulation market. For the third quarter of 2024, Nevro expects worldwide revenue to further decline to a range of approximately $92 million to $94 million.

NVRO closed Friday's trading at $6.01, up 6%.

Eisai Co. Ltd. (ESALF.PK) and SEED Therapeutics Inc., on Tuesday, entered into a strategic research collaboration to discover and develop novel molecular glue degraders for neurodegeneration and oncology indications. Eisai will pay SEED up to $1.5 billion in upfront and milestone payments, along with tiered royalties, upon exercising their exclusive rights under the strategic research collaboration.

ESALF.PK closed Friday's trading at $37.84.

Recursion Pharmaceuticals Inc. (RXRX) and Exscientia plc (EXAI), on Thursday, entered into a definitive agreement to create a combined entity that will be technology-first, end-to-end drug discovery platform. Recursion is a clinical stage technology-enabled biotech company decoding biology to industrialize drug discovery, while Exscientia is a technology-driven clinical stage drug design and development company, committed to creating more effective medicines for patients, faster. Recursion shareholders will own about 74% and Exscientia shareholders will own approximately 26% of the combined company. The combined company, which will be named Recursion, will continue to be headquartered in Salt Lake City, Utah, and trade on the NASDAQ, while maintaining a significant presence in the U.K.

RXRX closed Friday's trading at $6.29, down 5.27%.

Merck (MRK) has agreed to acquire CN201, a novel investigational clinical-stage bispecific antibody for the treatment of B-cell associated diseases, from Curon Biopharmaceutical, a privately held biotechnology company. Merck will pay an upfront payment of $700 million in cash and up to $600 million in milestone payments associated with the development and regulatory approval of CN201 to Curon. CN201 is currently being evaluated in Phase 1 and Phase 1b/2 clinical trials for the treatment of patients with relapsed or refractory non-Hodgkin's lymphoma (NHL) and relapsed or refractory B-cell acute lymphocytic leukemia (ALL), respectively. This is the fourth time this year that Merck has loosened its purse strings to make an acquisition.

MRK closed Friday's trading at $114.55, up 0.55%.

Gilead Sciences Inc. (GILD), on Friday, announced it is paying $320 million to Janssen Pharmaceutica NV to buy-out the global Seladelpar royalties. Seladelpar, a PPARd agonist, for the proposed indication of Primary biliary cholangitis, is under FDA review, with a decision due on Aug.14, 2024. Gilead gained control over Seladelpar, following the acquisition of CymaBay Therapeutics in February of this year. In 2006, CymaBay had inked a License agreement with Janssen Pharma, receiving an exclusive worldwide, royalty-bearing license to Seladelpar and certain other PPARd compounds. Under the terms of the agreement Janssen was entitled to receive up to an 8% royalty on net sales of PPARd Products. Now that Gilead has bought out the global Seladelpar royalties, it is not required to pay any royalty to Janssen if Seladelpar is approved.

GILD closed Friday's trading at $73.66, down 2.55%.

Breakthroughs and Setbacks

Actinium Pharmaceuticals Inc. (ATNM), on Monday, was informed by the FDA that its phase III SIERRA trial is not adequate to support a BLA filing for Iomab-B despite its statistically significant primary endpoint. Iomab-B is being developed for patients with active relapsed or refractory acute myeloid leukemia. The U.S. regulatory agency has required the company to conduct an additional head-to-head randomized clinical trial demonstrating overall survival benefit with Iomab-B. The company intends to seek a strategic partner for Iomab-B for the U.S.

ATNM has lost nearly 67% since the news to close Friday's trading at $2.05, down nearly 15%.

Aldeyra Therapeutics Inc. (ALDX), on Thursday, announced that its phase III trial of investigational 0.25% Reproxalap ophthalmic solution in dry eye disease has achieved the primary endpoint. In the study, Reproxalap was statistically superior to vehicle for the prespecified primary endpoint of ocular discomfort, an FDA-accepted symptom of dry eye disease. According to the company, this is the first positive phase 3 clinical trial in a dry eye chamber with a symptom as a primary endpoint. The FDA had declined to approve Reproxalap for the treatment of dry eye disease last November, requiring the company to conduct an additional trial demonstrating "efficacy in treating ocular symptoms associated with dry eyes". Now that the new trial has achieved the primary goal, the company is planning to resubmit the New Drug Application of Reproxalap in dry eye disease this year.

ALDX closed Friday's trading at $5.10, up 21.72%.

Pulse Biosciences Inc. (PLSE), on Thursday, announced it has treated the first two patients with atrial fibrillation in Netherlands in the first-in-human feasibility study using its novel Cardiac Surgery System for the ablation of cardiac tissue for the treatment of atrial fibrillation. The procedure was well tolerated by the two patients, according to Dr. Gan Dunnington, Chief Medical Officer, Cardiac Surgery of Pulse Biosciences. The trial is a multicenter study of up to 30 patients. The nano-PFA Cardiac Surgical System received FDA Breakthrough Device Designation in early July 2024. The company intends to submit for IDE approval from the FDA to begin U.S. clinical work.

PLSE closed Friday's trading at $15.65, up 3.64%.

Avidity Biosciences Inc. (RNA) on Friday, announced exciting data from its Phase 1/2 clinical trial evaluating investigational drug Del-zota for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping, dubbed EXPLORE44. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping. According to the trial findings, Del-zota demonstrated unsurpassed delivery of PMO of 200 nM in skeletal muscle and a reduction in creatine kinase levels to near normal with greater than 80% reduction compared to baseline. The study also demonstrated statistically significant 37% increase in exon 44 skipping and up to 66% exon 44 skipping and statistically significant increase of 25% of normal in dystrophin production and restored total dystrophin up to 54% of normal with Del-zota.

RNA closed Friday's trading at $46.95, up 12.13%.