On November 20, 2025 Syntara Limited (ASX: SNT), a clinical-stage drug development company, reported the opening of MDS05/D3 MESSAGE, a Phase 2 multi-centre trial investigating amsulostat (SNT-5505) in combination with hypomethylating agent ASTX727 for the treatment of transfusion dependent, low and intermediate risk Myelodysplastic Syndromes (MDS): ALLG | New MDS05 MESSAGE Trial | Transfusion Dependent MDS.

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The Principal Investigator, Associate Professor Anoop Enjeti, commented: "One of the inspirations for this study came from some ground breaking pre-clinical work recently conducted by Heidelberg University that showed a significant increase in red blood cell production when the Syntara drug, amsulostat, was added to a hypomethylating agent that is commonly used in high risk MDS patients. Amsulostat has since been shown to be safe and well tolerated when used in patients with another type of haematological cancer, myelofibrosis, for up to 12 months so we are excited about the potential it might hold for patients with MDS who have very few treatment options".

The multi-centre study will open at 10 hospitals across Australia with up to 30 patients to be recruited for treatment. It aims to reduce the reliance on fortnightly blood transfusions typically required by MDS patients, improving the survival outcomes and lessening the treatment burden.

The trial is being led by the Australasian Leukaemia & Lymphoma Group (ALLG), while being funded by the Australian Government’s Medical Research Future Fund, with contributions from Syntara and Taiho as industry partners.

MDS is a form of blood cancer where patient’s bone marrow fails to produce enough healthy blood cells, causing bone marrow failure. This high treatment burden in transfusion dependent MDS greatly impacts both quality of life for patients as well as placing a heavy load on blood transfusion supply.

Patients with this type of MDS also experience far worse outcomes, with a 5-year survival rate of just 37.3%, and a high risk of progression from MDS to acute myeloid leukaemia (AML), an aggressive and often fatal blood cancer.

Associate Professor Anoop Enjeti said: "Transfusion dependent myelodysplasia has no approved treatments currently available for Australian patients. The MDS05 MESSAGE trial’s new treatment combination aims to improve survival rates and quality of life for patients with this type of MDS by reducing the reliance on regular blood transfusions. Another unique aspect will be the combination of 2 oral medications making this trial more accessible to patients."

ALLG Chief Executive Officer, Delaine Smith, said: "The ALLG is the only collaborative blood cancer clinical trial group in Australasia, conducting clinical trials into MDS, AML and other leukaemias, lymphomas and myeloma. Our independent, clinician-led network enables us to conduct research into all areas of blood cancers, including rarer cancers and areas of high unmet need. We’re excited to launch the MESSAGE clinical trial to improve the outcomes and quality of life for transfusion-dependant MDS patients and delighted to work with Syntara whose original research underpins this new treatment concept."

The commencement of this trial follows (see ASX announcement 18 July 2025) the initiation of AZALOX, a Phase 1b/2 multi-centre study in Germany evaluating amsulostat in combination with 5-Azacitidine for the treatment of high-risk Myelodysplastic Neoplasms (MDS) and Chronic Myelomonocytic Leukemia (CMML). Updates on recruitment and interim safety and efficacy data are anticipated as both studies progress in 2026.

Syntara CEO Gary Phillips commented: "The MESSAGE trial allows us an opportunity to prove the potential of amsulostat in another form of blood cancer, complementary to our lead program in myelofibrosis and the AZALOX study in related MDS indications in Germany. We appreciate the support of the ALLG, Taiho, Professor Enjeti in conducting the trial and the financial support from the MRFF. We look forward to seeing initial results from this study in 2026 and, in the longer term, making a difference for these patients with limited treatment options."

(Press release, Syntara, NOV 20, 2025, https://mcusercontent.com/add2e2fa70ec3d0eeaf2a93cc/files/bfd40536-38a4-84f6-6c22-0325db62adbd/03026278.pdf [SID1234660852])

On November 20, 2025 Inhibikase Therapeutics, Inc. (NASDAQ: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing therapeutics to modify the course of pulmonary arterial hypertension ("PAH"), reported the pricing of its underwritten public offering of 46,091,739 shares of common stock and pre-funded warrants to purchase 22,873,779 shares of common stock. The shares of common stock are being sold at an offering price of $1.45 per share, and the pre-funded warrants are being sold at an offering price of $1.449 per pre-funded warrant, which represents the per share offering price for the common stock less the $0.001 per share exercise price for each such pre-funded warrant. In addition, Inhibikase has granted the underwriters a 30-day option to purchase up to an additional 10,344,827 shares of its common stock at the public offering price, less underwriting discounts and commissions. The aggregate gross proceeds to Inhibikase from this offering are expected to be approximately $100.0 million, before deducting underwriting discounts and commissions and other offering expenses, excluding the exercise of any pre-funded warrants. All of the securities being sold in the offering are being offered by Inhibikase. The offering is expected to close on November 24, 2025, subject to the satisfaction of customary closing conditions.

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Jefferies, BofA Securities and Cantor are acting as joint book-running managers for the offering. LifeSci Capital and Oppenheimer & Co. are acting as co-lead managers for the offering. H.C. Wainwright & Co. and Ladenburg Thalmann & Co. Inc. are acting as co-managers for the offering.

The securities described above are being offered by Inhibikase pursuant to a shelf registration statement on Form S-3 (No. 333-288213) that was previously filed with the Securities and Exchange Commission ("SEC") and declared effective on June 27, 2025. This offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A final prospectus supplement and accompanying prospectus related to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may also be obtained, when available, by contacting: Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; BofA Securities, Inc., Attention: Prospectus Department, 201 North Tryon Street, NC1-022-02-25 Charlotte, NC 28255- 0001, or by email at [email protected]; or Cantor Fitzgerald & Co., Attention: Capital Markets, 110 East 59th Street, 6th Floor, New York, NY 10022, or by email at [email protected].

This press release does not constitute an offer to sell or a solicitation of an offer to buy these securities, 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 that state or jurisdiction.

(Press release, Inhibikase Therapeutics, NOV 20, 2025, View Source [SID1234660851])

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])