On June 5, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company with a pipeline of first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC) (Press release, Tempest Therapeutics, JUN 5, 2025, View Source [SID1234653742]).

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"We’re incredibly pleased to receive Orphan Drug Designation from the EMA, building on the momentum of regulatory support we’ve already received from the FDA," said Stephen Brady, president and chief executive officer of Tempest. "These designations reflect the significant unmet need in liver cancer and reinforce our belief in the potential of amezalpat to make a meaningful difference for patients and families affected by this devastating disease."

The company announced earlier this year that the FDA had granted both ODD and Fast Track Designation (FTD) to amezalpat to treat patients with HCC. These three designations follow positive data across multiple key study efficacy and safety endpoints from a global, randomized Phase 1b/2 clinical study evaluating amezalpat plus standard-of-care atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone in the first-line treatment of patients with unresectable or metastatic HCC. Notable positive outcomes of the randomized comparison include a six-month improvement in median overall survival (OS) with a hazard ratio (HR) of 0.65 for patients receiving the amezalpat combination therapy. In addition, a survival benefit from patients receiving amezalpat was preserved in key sub-populations including PD-L1 negative disease, which is consistent with amezalpat’s proposed mechanism of action to target both the tumor cells directly and the patient’s immune system.

About Hepatocellular Carcinoma

HCC is an aggressive cancer with rising mortality and is projected to become the third leading cause of cancer death by 2030.2 Every year, more than 900,000 people worldwide are diagnosed with HCC.3 Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US.4 In the US, HCC represents the fastest-rising cause of cancer-related death.3

Nine out of ten cases of HCC are caused by chronic liver disease, which includes chronic hepatitis B and C infection, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), alcohol-related liver disease (ALD) and cirrhosis resulting from these conditions.5

Even if diagnosed in the early stage, an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery.6 Early recurrence is associated with poorer prognosis and shorter survival.5,7 Tumor size, number of tumors, and portal vein invasion are associated with an increased risk of recurrence.6

About Amezalpat

Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist. Data suggest that amezalpat treats cancer by targeting tumor cells directly and by modulating immune suppressive cells and angiogenesis in the tumor microenvironment. In a global randomized Phase 1b/2 study of amezalpat in combination with atezolizumab and bevacizumab in first-line patients with advanced HCC, the amezalpat arm showed clinical superiority across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to atezolizumab and bevacizumab alone, the standard of care. These randomized data were supported by additional positive results observed in the Phase 1 clinical trial in patients with heavily pretreated advanced solid tumors, including renal cell carcinoma and cholangiocarcinoma.

About Orphan Drug Designation

Orphan Designation is granted to therapies intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases that affect no more than two in 10,000 people in the European Union (EU) and for which no satisfactory therapy is available. The treatment must also provide significant benefit to those affected by the condition. EMA orphan drug designation provides certain benefits, including the potential for 10 years of market exclusivity following regulatory approval in the EU, reduction in regulatory fees and a centralized EU approval process.

On June 5, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported the appointment of two distinguished oncology leaders to its Scientific Advisory Board (SAB) (Press release, Sellas Life Sciences, JUN 5, 2025, View Source [SID1234653741]). The new members – Philip C. Amrein, MD, and Alex Kentsis, MD, PhD bring decades of expertise in cancer research, clinical oncology, and translational medicine, further strengthening the company’s strategic guidance as it advances its therapeutic pipeline.

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"We are honored to welcome Drs. Amrein and Kentsis to our Scientific Advisory Board," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Their extensive experience in clinical development and hematology-oncology research will be pivotal as we reach significant milestones with both of our assets –full topline Phase 2 data of SLS009 in acute myeloid leukemia (AML), and the final analysis of our Phase 3 pivotal REGAL trial of GPS in AML. With those inflection points expected this year, their guidance will help ensure we continue to advance our lead assets towards potential regulatory filings and commercialization, while also supporting our efforts in translational and precision medicine to maximize clinical impact. We are thrilled to collaborate with them, leveraging their exceptional expertise to drive groundbreaking success."

About the New SAB Members:

Philip C. Amrein, MD
Harvard Medical School – Massachusetts General Hospital (MGH)
Dr. Amrein is an Assistant Professor of Medicine at Harvard Medical School and a physician at the MGH, where he specializes in leukemia. He is part of the Cancer Center, Leukemia, Cellular Immunotherapy, and Hematology/Oncology departments. Dr. Amrein treats adult patients with acute and chronic leukemia, myelodysplasia, and myeloproliferative neoplasms, leading numerous clinical trials and exploring new treatment approaches. Dr. Amrein earned his MD from Johns Hopkins University School of Medicine and completed his residency at Yale Waterbury Hospital, followed by a fellowship at MGH. He is board-certified in Internal Medicine and Medical Oncology.

Alex Kentsis, MD, PhD
Memorial Sloan Kettering (MSK) Cancer Center
Dr. Kentsis is the founding Director of the MSK Tow Center for Developmental Oncology, a practicing pediatric oncologist at MSK Kids, a Member of the Sloan Kettering Institute, and Professor of Pediatrics, Pharmacology, and Physiology & Biophysics at Weill Cornell Medical College of Cornell University. His research focuses on improving understanding of blood and solid tumors, particularly in children and young adults. He and his team have identified new therapeutic targets in leukemias and solid tumors, as well as mechanisms of treatment resistance and evasion. The Kentsis Lab uses modern technologies to explore genomic plasticity, epigenetic signaling, and adaptive responses to targeted therapies. He holds an MD from Mount Sinai School of Medicine, a PhD from New York University, and completed his clinical training at the Boston Children’s Hospital and Dana-Farber Cancer Institute at Harvard Medical School.

On June 5, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viruses, reported the release of its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast discussing the final data from its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases (MB-107) (Press release, Moleculin, JUN 5, 2025, View Source [SID1234653740]).

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For the event, Walter Klemp, Chairman and Chief Executive Officer, and Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin are joined by Key Opinion Leaders: Mohamad Cherry, MD, Medical Director of Hematology at Atlantic Health System; Prof. Bernd Kasper, Sarcoma Unit of the Mannheim Cancer Center (MCC) at the Mannheim University Medical Center, University of Heidelberg; and Sant P. Chawla, MD, Director, Sarcoma Oncology Center, Director, Cancer Center of Southern California.

The on-demand video webcast is now available on the Events page of the Investors section of the Moleculin website, moleculin.com and will be accessible for 90 days.

On June 5, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or the "Company"), an oncology-focused biotechnology company developing innovative therapies for difficult-to-treat cancers, reported that the first patient has been dosed in a Phase 1b clinical trial sponsored by Kazia (Press release, Kazia Therapeutics, JUN 5, 2025, View Source [SID1234653739]). The study evaluates paxalisib, the Company’s dual PI3K/mTOR inhibitor, in combination with olaparib or pembrolizumab for patients with advanced breast cancer.

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This multi-center, open-label, randomized trial is designed to assess the safety, tolerability, and preliminary efficacy of multiple paxalisib-based treatment combinations. The study also includes deep biomarker profiling to support future development and early signals of clinical activity.

"The start of patient dosing in this Kazia-sponsored study marks an important milestone in the evolution of paxalisib beyond brain cancer and into broader solid tumor applications," said Dr. John Friend, Chief Executive Officer of Kazia. "By leveraging the dual inhibition of PI3K and mTOR, this trial builds on compelling preclinical data showing epigenetic modulation in aggressive breast cancer pre-clinical models. We believe the combinations explored here may provide a more effective therapeutic strategy by simultaneously targeting tumor metabolism, DNA repair, and immune evasion."

About the Study

This Phase 1b trial (ACTRNXXX) will enroll patients with advanced breast cancer into two treatment arms:

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Arm A: Patients are randomized to receive either 15mg or 30mg of paxalisib (once daily) in combination with olaparib (300mg orally, twice daily), administered in 28-day cycles.

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Arm B: Patients are randomized to receive paxalisib (15mg or 30mg once daily) and pembrolizumab (200mg IV every 21 days) alongside standard-of-care chemotherapy: either nanoparticle albumin-bound paclitaxel or a gemcitabine-carboplatin regimen, depending on clinical indication.

Participants will be evaluated for:

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Adverse events and overall tolerability

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Changes in circulating tumor cells ("CTCs") and CTC clusters

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Immune cell signatures and overall immune functio

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Clinical activity and tumor response signals

Strategic Significance

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For investors, this trial expands the clinical footprint of paxalisib into solid tumors beyond the central nervous system, targeting a significant commercial opportunity in advanced and metastatic breast cancers, including triple-negative breast cancer.

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For potential partners and acquirers, the novel biomarker-driven design provides an early window into how paxalisib may enhance or sensitize tumors to immune checkpoint inhibitors and DNA repair-targeted therapy.

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For the scientific community, the study is structured to generate translational data that may clarify the mechanistic interactions between dual PI3K/mTOR inhibition, immune modulation, and chemotherapy-induced cytotoxicity.

"The integration of paxalisib into combination regimens reflects our strategy of building value through differentiated science and high-quality collaborations," added Dr. Friend. "As this study progresses, we aim to share interim updates that may further underscore the potential of paxalisib to impact multiple indications with poor prognoses."

On June 5, 2025 GlycoMimetics, Inc. (Nasdaq: GLYC) ("GlycoMimetics") reported that its stockholders have approved the proposed merger (the "Merger") with Crescent Biopharma, Inc. ("Crescent"), along with all proposals related to the Merger (Press release, Crescent Biopharma, JUN 5, 2025, View Source [SID1234653738]). The proposals were voted upon at GlycoMimetics’ special meeting in lieu of the annual meeting of stockholders held on June 5, 2025 (the "Special Meeting"), including a reverse stock split of GlycoMimetics’ common stock to be effected at the discretion of the board of directors of GlycoMimetics (the "Board") within the parameters approved by GlycoMimetics’ stockholders.

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On June 5, 2025, following the Special Meeting, the Board approved a reverse stock split of GlycoMimetics’ common stock at a ratio of 1-for-100. Following the anticipated closing of the Merger, the combined company’s common stock is expected to begin trading on a post-reverse stock split basis on The Nasdaq Capital Market ("Nasdaq") on June 16, 2025, under the new name "Crescent Biopharma, Inc.", ticker symbol "CBIO", CUSIP number 38000Q201 and ISIN number US38000Q2012.

The reverse stock split is expected to reduce the number of GlycoMimetics’ outstanding common stock from approximately 64.5 million shares to approximately 0.6 million shares. The number of shares of GlycoMimetics’ authorized common stock will not be affected by the reverse stock split. At the Special Meeting, GlycoMimetics’ stockholders approved an increase in the number of shares of GlycoMimetics’ authorized common stock from 150,000,000 shares to 175,000,000 shares in connection with the anticipated closing of the Merger. No fractional shares will be issued if, as a result of the reverse stock split, a stockholder would otherwise become entitled to a fractional share because the number of shares of GlycoMimetics common stock they hold before the reverse stock split is not evenly divisible by the split ratio. Instead, each stockholder will be entitled to receive a cash payment in lieu of such fractional share. The cash payment to be paid will be equal to the fraction of a share to which such stockholder would otherwise be entitled multiplied by the closing price per share as reported by The Nasdaq Stock Market LLC on June 12, 2025, the trading day prior to the date the charter amendment to effect the reverse stock split is expected to be filed with the Secretary of State of the State of Delaware (with such price proportionately adjusted to give effect to the reverse stock split).

As a result of the reverse stock split, proportionate adjustments will be made to the exercise prices and number of shares of GlycoMimetics’ common stock underlying GlycoMimetics’ outstanding equity awards. There will be no change to the par value per share.

Following the closing of the Merger, the combined company’s total issued and outstanding common stock is expected to be approximately 14.8 million shares, or approximately 25.3 million shares on a fully-diluted basis.