On November 20, 2025 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that it will host an R&D Day to discuss its pipeline on Dec. 2, 2025, at 9:30 a.m. EST in New York City. The event will also be webcast.

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Matthew B. Klein, M.D., Chief Executive Officer, and PTC research and development team leaders will provide an update on the company’s proprietary small molecule splicing and inflammation platforms, including new targets and programs.

To register for the webcast, please visit the Events and Presentations page on the Investors section of the PTC Therapeutics website at View Source A replay will be available for approximately 30 days following the event.

(Press release, PTC Therapeutics, NOV 20, 2025, View Source [SID1234660850])

On November 20, 2025 Pasithea Therapeutics Corp. (Nasdaq: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic oral MEK inhibitor for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), reported positive interim Phase 1 data from its ongoing first-in-human trial evaluating PAS-004 in patients with MAPK pathway-driven advanced solid tumors with a documented RAS, NF1 or RAF mutation, or in patients who have failed prior BRAF/MEK inhibition.

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Dr. Tiago Reis Marques, CEO of Pasithea said, "Today’s updated interim results from our advanced cancer trial demonstrate the safety, PK and anti-tumor activity of PAS-004, and support its potential to be a best-in-class MEK inhibitor for the treatment of NF1-PN. Achieving a monotherapy partial response in an advanced cancer patient who had previously received a MEK + BRAF inhibitor combination therapy, and whose prior best response had been stable disease, is very promising. At our highest reported cohort (30mg capsule), we are seeing significant drug exposures (Area Under the Curve (AUC) greater than 5,400 ng·h/mL), with a relatively flat PK curve, suggesting sustained pathway inhibition. We believe this profile is well aligned with what is needed to drive meaningful clinical responses in NF1-PN patients. Published clinical data has shown that tumor response in NF1-PN is positively correlated with drug exposure (AUC), reinforcing the relevance of these findings."

Dr. Rebecca Brown, Director of the Neurofibromatosis (NF) and Schwannomatosis (SWN) Program at University of Alabama Birmingham (UAB) and Scientific Advisory Board member of Pasithea, stated: "I find it very encouraging that even when used as a monotherapy in advanced recurrent cancer patients, PAS-004 has demonstrated early signals of efficacy, but more importantly exhibited such a favorable safety profile that no dose interruptions or modifications were required. Maintaining NF1-PN patients on treatment for extended periods of time is paramount to achieving maximum tumor control. I believe that PAS-004’s early efficacy signals combined with the low rate of adverse side effects may translate into better tolerability and longer time-on-treatment for plexiform neurofibromas associated with NF1, compared with current FDA-approved therapies discontinuation rates estimated as high as 40-50% before year two."

Interim Phase 1 Results for PAS-004:

Initial Signals of Clinical Activity

Among 21 efficacy evaluable patients (as per RECIST1.1):

Partial Response:
A BRAF V600E melanoma patient in Cohort 4A (15mg capsule) achieved an unconfirmed partial response with a –31.9% tumor reduction and remains on trial for >11 months; prior best response when treated with a MEK + BRAF combination therapy was stable disease
Disease Control Rate (DCR):
71.4% (5 of 7) of patients identified with BRAF-mutated tumors achieved stable disease or partial response
42.8% (9 of 21) of patients achieved stable disease or partial response
Durable Stable Disease:
A second BRAF V600E melanoma patient previously treated with MEK + BRAF combination therapy in Cohort 6 (30mg capsule) remains on trial for >6 months with a stable disease and tumor shrinkage of -1.6%
Safety and Tolerability

Among 27 dosed patients through the Dose Limiting Toxicity (DLT) period (Day 28) through the cutoff date of November 10, 2025:

PAS-004, dosed once daily (QD), has been well-tolerated across all dose levels
No dose-limiting toxicities (DLTs), and no discontinuations have been reported.
All treatment-related adverse events (TRAEs) at least possible related to PAS-004 were Grade 1 or 2, with limited rash (7.4%), nausea (18.5%), vomiting (14.8%), diarrhea (7.4%), and no ocular retinal abnormalities or cardiovascular toxicities observed.
Pharmacokinetics (PK)

PAS-004 has demonstrated through Cohort 6:

Linear PK and dose-proportionality
PK curve with Cmax/Cmin ratio <2, with Cmax and Cmin above the IC50 (half-maximal inhibitory concentration) from our cellular assay.
Long half-life (~60 hours)
Cohort 6 (30mg capsule) has demonstrated:
AUC: ~5,480 ng·h/mL
Cmax: 249 ng/mL
Cmin: 215 ng/mL

(Press release, Pasithea Therapeutics, NOV 20, 2025, View Source [SID1234660849])

On November 20, 2025 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported the closing of its previously announced underwritten public offering of 11,500,000 shares of its common stock, which includes the full exercise of the underwriters’ option to purchase 1,500,000 additional shares of common stock, at a price to the public of $19.00 per share. The gross proceeds to Olema from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $218.5 million.

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TD Cowen, Evercore ISI, Guggenheim Securities, LifeSci Capital, Oppenheimer & Co. and H.C. Wainwright & Co. acted as book-running managers for the offering.

The offering was made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed with the Securities and Exchange Commission (the "SEC") on January 6, 2025 and declared effective on January 15, 2025, and a related registration statement on Form S-3MEF that was filed with the SEC pursuant to Rule 462(b) on November 18, 2025, which became automatically effective on November 18, 2025. Copies of the final prospectus supplement and accompanying prospectus relating to the offering can be accessed at no charge through the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained from: TD Securities (USA) LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, Olema Oncology, NOV 20, 2025, View Source [SID1234660848])

On November 20, 2025 GENFIT (Nasdaq and Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, reported its third quarter 2025 financial results1 and provided a corporate update.

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Cash Position

As of September 30, 2025, the Company’s cash and cash equivalents amounted to €119.0 million compared with €107.5 million as of June 30, 2025, and €81.8 million as of December 31, 2024.

We expect that our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements beyond the end of 2028, enabling the Company to further develop its pipeline focused on Acute on-Chronic Liver Failure (ACLF) and support general corporate purposes. This is based on current assumptions and programs and does not include exceptional events. This estimation assumes (i) our expectation to receive significant future commercial milestone revenue pursuant to the license agreement with Ipsen and Ipsen meeting its sales-based thresholds and (ii) drawing down all additional installments under the Royalty Financing agreement with HCRx.

In the first nine months of 2025, cash utilization is mainly the result of our research and development efforts in our pipeline focused on ACLF (notably VS-01, G1090N2, SRT-015, CLM-022, and VS-02 HE), as well as GNS561 in cholangiocarcinoma (CCA). Cash utilization was notably offset by a €26.5 million milestone payment received in July 2025 under the Licensing and Collaboration Agreement with Ipsen, following pricing and reimbursement approvals for Iqirvo (elafibranor) in three major European markets3.

Revenue

Revenue4 for the first nine months of 2025 amounted to €39.2 million compared to €59.7 million for the same period in 2024.

Revenue for the first nine months of 2025 was primarily driven by the Licensing and Collaboration Agreement with Ipsen, including (i) royalty revenue from worldwide sales (excluding Greater China) of Ipsen’s Iqirvo (elafibranor) totaling €12.6 million and (ii) milestone revenue from pricing and reimbursement approval of Iqirvo (elafibranor) in three major European countries3 totaling €26.5 million.

Program highlights

ACLF pipeline

G1090N2 – A Phase 1 First-in-Human study in healthy volunteers is currently underway with safety data expected at the end of this year. Early signals of efficacy from ex-vivo functional assays are also expected at the same time.

SRT-015 – Current work on an improved formulation aims at increasing exposure. Pending positive development, the launch of a first-in-human trial could be initiated as early as the second half of 2026.

CLM-022 – Current experiments aim at confirming therapeutic efficacy using different disease models relevant for AD and ACLF as well as starting formulation development and first toxicological studies in 2025. Pending further positive developments, a first-in-human trial could be initiated in the first half of 2027.

VS-02-HE – We intend to develop VS-02-HE as a unique oral formulation designed to act in the gut where ammonia is primarily produced, minimizing systemic absorption of ammonia while reducing glutamine levels in the brain. Completion of Investigational New Drug-enabling nonclinical studies and formulation development are expected by the end of 2025. A first-in-human trial could be initiated in the second half of 2027.

Other life-threatening diseases

GNS561 in CCA – Data readout from the ongoing Phase 1b clinical trial are expected by the end of 2025.

VS-01-HAC in Urea Cycle Disorders & Organic Acidemias (pediatric indication) – Data from the pivotal juvenile toxicology study in Göttingen Minipigs are expected before the end of 2025. Following discontinuation of VS-01 in ACLF, additional preclinical work will be conducted before moving into the clinic. Update on the ongoing preclinical work and potential clinical development is expected before the end of 2025.

Primary Biliary Cholangitis (PBC)

As reported in Ipsen’s third quarter financial results5, Iqirvo (elafibranor) continues to show solid growth across both the U.S. and European markets in PBC.

(Press release, Genfit, NOV 20, 2025, https://ir.genfit.com/news-releases/news-release-details/genfit-reports-third-quarter-2025-financial-information-and [SID1234660847])

On November 20, 2025 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) reported the first patient has been randomized in the pivotal Phase 3 CAREFNDR trial, a multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of once-daily, oral paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The Phase 3 study builds on positive Phase 2 data in which paltusotine demonstrated rapid and sustained reductions in the frequency and severity of carcinoid syndrome symptoms, including flushing episodes and bowel movements.

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"Patients with carcinoid syndrome need a treatment option that can consistently manage symptoms without the burden of painful monthly injections," said Dana Pizzuti, M.D., Chief Medical and Development Officer of Crinetics. "Based on our encouraging Phase 2 results and data from our open-label extension, we believe paltusotine has the potential to transform the treatment landscape with a once-daily, oral therapy that may offer meaningful benefits for patients managing the challenging day-to-day impacts of carcinoid syndrome. The initiation of our Phase 3 CAREFNDR trial represents a significant milestone in our commitment to address this critical unmet need."

The Phase 3 study is designed to enroll 141 adults, both naïve and previously treated patients, with carcinoid syndrome. Participants will be randomized in a 2:1 ratio to receive either once-daily paltusotine 80 mg or matching placebo. The primary endpoint will measure the change in flushing episodes per day from baseline to Week 12, with change in bowel movements per day as a key secondary endpoint. Following the 16-week randomized controlled period, the trial includes a 104-week open-label extension (OLE) to evaluate long-term efficacy, safety and additional clinical outcomes. The OLE will also monitor long-term tumor control (progression free survival).

The Phase 3 CAREFNDR trial represents the latest step in Crinetics’ goal of expanding the value of PALSONIFY (paltusotine) beyond its lead indication for the treatment of acromegaly. Crinetics is also investigating meeting the unmet needs of NETs patients through CRN09682, the first candidate from its nonpeptide drug conjugate platform. CRN09682, an investigational anti-tumor candidate targeting SST2-expressing neuroendocrine and other solid tumors, received Investigational New Drug clearance earlier this year. This broader development strategy reflects Crinetics’ dedication to developing transformative therapies that address serious endocrine disorders and endocrine-related cancers.

For more information about the CAREFNDR trial, visit View Source Global enrollment in CAREFNDR is expected throughout 2025 and 2026.

About Paltusotine
Paltusotine, a selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide, is in Phase 3 clinical development for carcinoid syndrome associated with neuroendocrine tumors (CAREFNDR). Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel movement frequency, which are the most common symptoms of carcinoid syndrome. PALSONIFY (paltusotine) is currently approved in the U.S. for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.

About Carcinoid Syndrome
Carcinoid syndrome is found in approximately 20% of patients with neuroendocrine tumors (NETs). NETs are a rare, slow-growing type of cancer that arise most often in the digestive tract. When these tumors metastasize to the liver, carcinoid syndrome can occur and is most commonly characterized by diarrhea and flushing. While injectable depot somatostatin receptor ligand therapies are mainstay treatments for carcinoid syndrome, these injections are associated with considerable treatment burden and offer inadequate relief of carcinoid syndrome symptoms for many patients.

(Press release, Crinetics Pharmaceuticals, NOV 20, 2025, View Source [SID1234660846])