On February 10, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC) (Press release, Tempest Therapeutics, FEB 10, 2025, View Source [SID1234650138]).

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"We are thrilled to receive Fast Track designation from the FDA," said Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest. "This designation, following the Orphan Drug designation granted last month, reinforces the promise of amezalpat as a potential treatment option for patients affected by HCC. We look forward to working closely with the FDA and foreign regulatory agencies to develop amezalpat with the goal of bringing this promising therapy to patients."

This is the second regulatory designation granted to amezalpat. The company announced in January that the U.S. FDA granted amezalpat with Orphan Drug Designation (ODD) following positive data across multiple key study efficacy and safety endpoints in 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 the addition of 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.ii Every year, more than 900,000 people worldwide are diagnosed with HCC.iii Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US.iv In the US, HCC represents the fastest-rising cause of cancer-related death. iii

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.v

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

About Amezalpat

Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist. Data suggests 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 in first-line HCC patients, amezalpat in combination with atezolizumab and bevacizumab showed clinical superiority across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to patients receiving 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 Fast Track Designation

Fast Track designation is intended to help rapidly advance the development and review processes for promising therapeutic candidates for serious conditions that may fill an unmet medical need. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process and may also be eligible for accelerated approval and priority review when relevant criteria are met.

On February 10, 2025 Beyond Air, Inc. (NASDAQ: XAIR) ("Beyond Air" or the "Company"), a commercial stage medical device and biopharmaceutical company focused on harnessing the power of nitric oxide (NO) to improve the lives of patients, reported its financial results for the fiscal third quarter ended December 31, 2024, and provided a corporate update (Press release, Beyond Air, FEB 10, 2025, View Source [SID1234650139]).

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"Our commercial team continues to make significant progress as we expand the number of U.S. hospitals utilizing LungFit PH. While this is the culmination of years of work, it was approximately nine months ago that we started to make significant headway in our commercial efforts with the fully updated system. We expect to continue generating double digit sequential quarterly revenue growth for the foreseeable future," said Steve Lisi, Chairman and Chief Executive Officer of Beyond Air.

"We ended calendar year 2024 on a high note with the receipt of CE Mark for LungFit PH in Europe, opening a significant portion of the global market outside of the U.S. for our current and future distribution partners to launch their commercial programs for LungFit PH. With the advantage of real-world customer experience and feedback from our U.S. program, combined with our distribution partners’ established sales teams and customer networks, we anticipate a faster commercial ramp-up in these regions compared to what we experienced in the U.S.," concluded Mr. Lisi.

Commercial Execution, Portfolio Highlights and Upcoming Milestones

LungFit PH Commercial Execution
Increased demand for LungFit PH:
A 34% increase in revenue for the quarter ended December 31, 2024, compared with the quarter ended September 30, 2024
Six new hospital starts in the U.S. in the quarter ended December 31, 2024
Received market authorization in Australia in January 2025 and expect to have regulatory approvals in eight countries covered under the partnership with Getz Healthcare by calendar year-end, with initial commercial shipments occurring in 2025.
Signed partnerships with two distributors in the Middle East in January 2025, with initial shipments expected in the 1H of calendar year 2025.
Pipeline Highlights
Received CE Mark for LungFit PH, which allows Beyond Air to market LungFit PH in the European Union and all other countries that recognize this certification.
Indications under CE Mark certification include:
The treatment of infants >34 weeks gestation with hypoxic respiratory failure associated with clinical or echocardiographic evidence of pulmonary hypertension, in order to improve oxygenation and to reduce the need for extracorporeal membrane oxygenation; and
The treatment of peri- and post-operative pulmonary hypertension in adults and newborn infants, infants and toddlers, children and adolescents, ages 0-17 years in conjunction to heart surgery, in order to selectively decrease pulmonary arterial pressure and improve right ventricular function
CE Mark certification triggered a $1 million milestone payment, payable in the fiscal fourth quarter of 2025, from the Company’s Asia-Pacific partner, Getz Healthcare, the leading distributor of medical equipment, devices and consumables in Asia Pacific region – providing access to hospitals in Australia, New Zealand, Thailand, Philippines, Taiwan, Hong Kong, Malaysia, Pakistan, Singapore and Vietnam
Beyond Air will also receive double-digit royalty payments based on LungFit PH net sales by Getz Healthcare
Cardiac surgery PMA supplement review ongoing at FDA
Currently no FDA approved nitric oxide system is labeled for cardiac surgery
Approval should increase LungFit PH’s rate of market penetration
LungFit PH transport-ready PMA supplement submission to FDA anticipated in the coming months
Beyond Cancer – Solid Tumor Program

Clinical Development Execution
Phase 1a trial (monotherapy) – Part A of the trial evaluating ultra-high concentration Nitric Oxide (UNO) therapy in subjects with advanced, relapsed or refractory unresectable, primary or metastatic cutaneous and subcutaneous solid tumors at a dose of 25,000 ppm has been completed, and at the dose of 50,000 ppm is ongoing.
Phase 1b trial (combination therapy) – Received regulatory approval in Israel for Part B of the trial, which will assess the intratumoral administration of 25,000 ppm low volume (LV) Nitric Oxide (UNO) in patients with unresectable cutaneous or subcutaneous histologically confirmed primary or metastatic lesions, who have shown disease progression or prolonged stable disease (12 weeks) after receiving a single agent anti-PD-1 containing treatment. Topline data from the Phase 1b portion of the study are anticipated late in calendar 2025 or early-2026.
NeuroNOS – Autism Spectrum Disorder (ASD) Program

NeuroNOS appointed Professor Roger D. Kornberg to its Scientific Advisory Board (SAB). Dr. Kornberg was awarded the Nobel Prize in Chemistry in 2006 for his groundbreaking work in molecular biology and is a renowned leader in the field of eukaryotic gene transcription. Professor Kornberg will assist NeuroNOS in accelerating the development of treatments for ASD and potentially other neurological disorders.
Financial Results for the Fiscal Third Quarter Ended December 31, 2024

Revenues for the fiscal quarter ended December 31, 2024 were $1.1 million compared to $0.4 million for the fiscal quarter ended December 31, 2023 and $0.8 million for the previous quarter ended September 30, 2024.

Cost of revenue of $1.3 million was recognized for the three months ended December 31, 2024, compared to $0.7 million for the three months ended December 31, 2023. Cost of revenue exceeded revenue primarily driven by depreciation of LungFit devices and one-time upgrade costs to systems.

Research and development expenses for the three months ended December 31, 2024 were $3.0 million as compared to $6.8 million for the three months ended December 31, 2023. The decrease of $3.8 million was primarily attributed to a decrease in salaries, stock-based compensation and to a lesser extent from clinical and pre-clinical studies expenses.

Selling, general and administrative expenses for the three-month periods ended December 31, 2024 and 2023 were $7.7 million and $9.8 million, respectively. The decrease of $2.1 million was attributed primarily to a reduction in salaries and stock-based compensation cost.

Other expense for the three months ended December 31, 2024 was $2.4 million compared to $0.3 million for the three months ended December 31, 2023. The increase in other expense of $2.1 million was mainly due to a non-cash loss recorded upon the retirement of the Avenue Capital debt.

Cash burn in the fiscal quarter ended December 31, 2024, excluding the impacts of financing and the extinguishment of debt, was $7.6 million.

As of December 31, 2024, the Company reported cash, cash equivalents, and marketable securities of $10.9 million, and total debt outstanding of $11.8 million. Debt repayment does not begin until October 2026. The Company’s cash, cash equivalents, and marketable securities, along with its cash conservation strategy and anticipated revenue growth are expected to provide sufficient cash runway to support current operating plans well into calendar 2026.

Conference Call & Webcast
Monday, February 10th @ 4:30 PM ET

Domestic: 1-877-407-0784
International: 1-201-689-8560
Conference ID: 13750714
Webcast: A webcast of the live conference call can be accessed by visiting the Events section of the Company’s website (click here) or directly (click here). An online recording of the conference call will be available on the Company’s website or via the direct link an hour after the call.

On February 10, 2025 Convergent Therapeutics Inc., a clinical-stage biotechnology company focused on the development of next-generation radiopharmaceuticals for the treatment of cancer, reported clinical trial updates for its lead asset, CONV01-α (225Ac-J591), a prostate-specific membrane antigen (PSMA)-targeted monoclonal antibody linked to actinium-225 (225Ac) (Press release, Convergent Therapeutics, FEB 10, 2025, View Source;at-the-american-society-of-clinical-oncology-asco-genitourinary-cancers-symposium-2025-302372722.html [SID1234650140]). Data will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium, which will take place February 13-15, 2025, in San Francisco, California.

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The first presentation, titled "Mature phase 1 follow up of alpha emitter 225Ac-J591 with 177Lu-PSMA-I&T in advanced prostate cancer," reported clinical follow-up of a Phase I dose-escalation trial exploring the addition of CONV01-α to 177Lu-PSMA-I&T, a PSMA-targeted beta emitting small molecule for the treatment of advanced prostate cancer. The study population presented included 18 patients (six at each dose level of CONV01-α) with progressive metastatic castration-resistant prostate cancer (mCRPC). Relatively low doses of CONV01-α were used.

Data showed that the median overall survival was 29.8 months, with 10 patients still alive at the time of submission. Five patients were free from PSA progression at one year including one patient progression-free at 34 months on no active therapy. A decline in prostate-specific antigen (PSA) levels was seen in 94% of patients, 64% of patients achieved a PSA50 and 28% achieved a PSA90 response. Additionally, circulating tumor cell count changes were seen, with 80% of evaluable patients converting from "unfavorable" to "favorable." High grade adverse events were rare.

"The impressive clinical activity is consistent with our pre-clinical data demonstrating that the antibody/ligand combination delivers a synergistic dose to tumor. Importantly, the synergistic tumor dose is achieved in the absence of additive toxicity due to the non-overlapping normal organ biodistribution of the ligand and antibody. This was also confirmed by the benign safety profile in the trial. The dosimetry and safety profile suggest the potential for additional cycles beyond the two cycles used in this trial. That the overall survival in this trial was double that of the Phase III VISION trial of 177Lu PSMA-617 further increases our enthusiasm for future trials of this combination," said Neil Bander, MD, Convergent’s Co-Founder and Chief Scientific Officer and Professor Emeritus of Urology at Weill Cornell Medicine.

The second presentation, titled "CONVERGE-01: Dosimetry, randomized dose optimization, dose escalation, and efficacy of Ac-225 rosopatamab tetraxetan in participants with PSMA-positive castration-resistant prostate cancer," is a "Trials in Progress" presentation of the three-part company sponsored Phase II trial evaluating the safety and efficacy of CONV01-α (225Ac-J591) monotherapy in patients with castration-resistant prostate cancer. Philip Kantoff, MD, Co-Founder and CEO said, "CONVERGE-01 is intended to confirm the high response rate and durability of CONV01-a monotherapy and solidify the path forward to our pivotal trial."

About CONV01-α
CONV01-α, Convergent’s alpha-emitting radioantibody, combines the precision and pharmacokinetics of antibodies with the tumor-killing potential of alpha-emitting radionuclides. Specifically, CONV01-α uses a humanized monoclonal antibody targeted at prostate-specific membrane antigen (PSMA), which is highly overexpressed in prostate cancer cells. Since PSMA is a validated target, several therapeutics are directed at this antigen, and CONV01-α is differentiated by its use of both an antibody and alpha emitter. CONV01-α is linked to a powerful radionuclide called Ac-225, which releases alpha particles that kill cancer cells through DNA double-strand breaks. Unlike other radioactive sources, alpha particles deliver high-energy radiation over very short distances, thereby minimizing radiation exposure to healthy neighboring cells and tissues. Pairing highly selective antibodies with such a powerful, yet precise, payload offers the ideal combination to treat many types of cancers. Convergent Therapeutics is currently enrolling into the CONVERGE-01 Phase II monotherapy trial to confirm the high response rate, durability and safety of CONV01-a pre and post radioligand beta.

On February 10, 2025 TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical-stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and other indications, reported the first Cohort B patient in the ACHIEVE Phase 2B UK clinical trial, evaluating TCB008 in Acute Myeloid Leukemia, has completed the full dosing regiment (Press release, TC Biopharm, FEB 10, 2025, View Source [SID1234650141]).

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The ACHIEVE trial is an open-label Phase II study dedicated to evaluating the efficacy and safety of TCB008. This trial is focused on assessing the treatment’s effectiveness and tolerability on patients suffering from acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS/AML) with challenging cases such as refractory or relapsed conditions. Cohort B recruits patients who have achieved remission following previous treatment yet continue to have a detectable or minimal residual disease (MRD).

Recruitment into Cohort B was initiated during the fourth quarter of 2024, ahead of the Company’s anticipated schedule. The first Cohort B patient received their dose in October 2024. The initial Cohort B patient has completed the dosing regiment, receiving all four planned doses of TCB008, and is expected to receive an additional fifth dose. Enrolment of a second Cohort B patient has also been initiated.

The safety objectives and endpoints of ACHIEVE evaluate patient responses to TCB008, including; grading of adverse events experienced and the incidence and severity of cytokine release syndrome and neurotoxicity. In a review of preliminary data, there are no drug-related adverse events following cumulative infusions of TCB008, containing up to a billion cells. These data continue to support the positive safety profile of TCB008 and the ACHIEVE UK study safety objectives and endpoints.

"The ACHIEVE study progressed at an incredible rate in 2024," stated Alison Bracchi, EVP of Clinical Operations. "Thanks to the hard work and dedication of both the TC BioPharm team and Clinical sites, we’ve reached a significant study milestone in under six months. We’re seeing a fantastic safety profile from our initial data review and exciting efficacy signals that indicate cellular recovery and a reduction of inflammation in AML patients. The TC BioPharm team and I are inspired by the progress to date and look forward to sharing further updates on Cohort B."

"The progression of ACHIEVE, with dosing underway in the second cohort, is a key milestone in the clinical development of our gamma delta therapy candidate TCB008. We believe it has potential to serve as an efficacious treatment for AML patients, whom still have significant unmet needs," said Bryan Kobel, CEO of TC BioPharm. "The therapy has been well-tolerated with no unexpected events or toxicities observed and promising efficacy results observed in some patients, with additional data being collected and analyzed. Cohort B is an extremely compelling patient population for TCB008 for many reasons, including patients having a more intact immune system to amplify the impact of TCB008 and the lack of true treatment options for these patients who are unfortunately on a path to relapse. We expect to complete enrolment in the second cohort in the first half of 2025, with data readout anticipated later this year."

On February 10, 2025 PHC Corporation reported to have signed a Master Collaboration Agreement with CCRM to work together on the development of primary T-cell(*1) expansion culture processes that will seek to accelerate the manufacturing of cell and gene therapy (CGT) products (Press release, CCRM, FEB 10, 2025, View Source [SID1234650119]). This joint initiative will integrate "LiCellGrowTM(*2), PHC’s cell expansion system under development, with CCRM’s deep knowledge of regenerative medicine and biomanufacturing to establish new culture processes to improve cell culture efficiency and quality for CGTs.

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Primary T-cells are used in process development and manufacturing for CGTs, such as in CAR-T cell therapy.(*3) However, primary T-cells derived directly from patients often exhibit significant variability in growth rates and quality, making it challenging for researchers to ensure stable cell counts and maintain quality throughout the culture process. To address these challenges and improve the quality of cell-based therapeutics, better cell culture processes are needed.

Chikara Takauo, Director of PHC and Head of the Biomedical Division that leads the company’s Life Science business, commented: "We are delighted to begin this joint research and development initiative with CCRM, a leader with 14 years of experience in the commercialization of regenerative medicine and CGT. By combining the technologies and expertise of both of our organizations, we aim to advance the manufacturing processes for cell-based therapeutics and cell culture technologies, contributing to the early practical application of CGT."

PHC has developed proprietary In-Line monitoring technology to track key indicators of cell metabolism in real-time, which can help researchers address issues like cell quality and reproducibility, and establish optimal cell culture methods. This technology enables precise, continuous measurement of glucose uptake and lactate production during cell culture, providing a more precise understanding of changes in cell metabolism over time than is possible to observe using traditional sampling methods. In 2024, PHC launched the live-cell metabolic analyzer "LiCellMoTM(*4)" incorporating this technology in the United States, Canada, Europe and some Asian markets including Japan, China, Singapore and Taiwan.

Building on this technology, the company is also developing "LiCellGrow," a cell expansion system designed to exchange media automatically based on the metabolic state of the cells and to maintain the culture environment in an optimal state. PHC aims to further expand its product lineup to seamlessly support research, process development, and commercial manufacturing of cell-based therapeutics.

"We are excited to collaborate with PHC to unlock new possibilities in cell culture," explained Michael May, President and CEO of CCRM. "Technology development partnerships, like this one, are key to advancing the industry and making CGT more cost effective, and therefore more accessible to patients around the world."

The joint research with CCRM will allow PHC to analyze culture conditions using "LiCellGrow" to establish optimal culture processes for primary T-cells. The collaboration will seek to accelerate LiCellGrow’s development, contributing to improved cell quality, enhanced manufacturing efficiency, and cost reduction in the production of cell-based therapeutics.