On November 12, 2024 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 company received a "Study May Proceed" letter from the U.S. Food and Drug Administration (FDA) to evaluate amezalpat (TPST-1120) in combination with atezolizumab and bevacizumab, the current standard of care, versus the standard of care alone in a pivotal randomized Phase 3 trial for the first-line treatment of unresectable or metastatic hepatocellular carcinoma (HCC) (Press release, Tempest Therapeutics, NOV 12, 2024, View Source [SID1234648216]).

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"The clinical and regulatory team at Tempest are thrilled to receive this notice from FDA with respect to the planned pivotal Phase 3 trial to evaluate amezalpat as a potential treatment for first-line liver cancer," said Sam Whiting M.D., Ph.D., chief medical officer and head of R&D of Tempest. "Previously reported positive Phase 2 data underscore amezalpat’s potential to improve the survival of patients facing this life-threatening disease, and our team is dedicated to advancing the program and bringing amezalpat to patients."

About the TPST-1120-301 Study (NCT06680258)

The planned Phase 3 study is a global, blinded, 1:1 randomized study of amezalpat plus atezolizumab and bevacizumab versus placebo plus atezolizumab and bevacizumab, the standard of care, for the first-line treatment of patients with unresectable or metastatic HCC. In August 2024, the company received agreement from the FDA on its Phase 3 study design, dose of amezalpat, and the statistical plan, including a pre-specified efficacy analysis that could shorten the time to primary analysis. The company is working to enable a Phase 3 study to start in the first quarter of 2025.

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 an ongoing 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.

On November 12, 2024 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, reported financial results and corporate updates from the third quarter of 2024 (Press release, BeiGene, NOV 12, 2024, View Source [SID1234648158]).

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"Our exceptional third-quarter results underscore the Company’s global oncology leadership driven by our unique R&D and clinical advantages as well as the tremendous launch trajectory of BRUKINSA," said John V. Oyler, Co-Founder, Chairman and CEO at BeiGene. "In the U.S., BRUKINSA, with the broadest label of any BTK inhibitor, is now the leader in new patient starts in both frontline and relapsed/refractory (R/R) CLL in addition to all other approved B-cell malignancies. As the cornerstone of our hematology franchise, BRUKINSA shows tremendous promise for patients as a monotherapy and as a backbone for best-in-class combinations with our late-stage BCL2 inhibitor, sonrotoclax, and BTK degrader BGB-16673. In the solid tumor area, we’re expanding access to our PD-1 inhibitor, TEVIMBRA, for patients worldwide and building global commercial capabilities to support our prolific pipeline of exciting potential cancer medicines. We are laying the foundation for future franchises in breast, lung, and gastrointestinal cancers across three signature platform technologies including multi-specific antibodies, protein degraders, and antibody-drug conjugates. This progress not only highlights our achievements but also emphasizes our commitment to positively impacting patients’ lives globally, fostering hope and advancements in the fight against cancer."

On November 12, 2024 Nuvation Bio Inc. (NYSE: NUVB), a late clinical-stage, global biopharmaceutical company tackling some of the greatest unmet needs in oncology, reported that David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio, will present at the Jefferies London Healthcare Conference in London, U.K., on Tuesday, November 19, 2024, at 3:30 a.m. ET/8:30 a.m. GMT (Press release, Nuvation Bio, NOV 12, 2024, View Source [SID1234648202]).

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A live webcast of the presentation will be available on the Nuvation Bio website at View Source An archived recording will be available for 90 days following the event.

On November 12, 2024 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended September 30, 2024, and provided a corporate update (Press release, Tempest Therapeutics, NOV 12, 2024, View Source [SID1234648217]).

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"During the third quarter, the team continued to move amezalpat successfully towards the pivotal study, achieving important milestones on both the regulatory and business fronts," said Stephen Brady, president and chief executive officer of Tempest. "Based on the positive randomized Phase 2 data and a Phase 3 plan we believe is designed for success, we were thrilled to receive broad agreement with the FDA. Coupled with Roche’s support for the Phase 3 study, the third quarter further solidified the foundation of a pivotal study that we hope will result in a new and meaningful therapy for first-line HCC patients."

Recent Highlights

Amezalpat (TPST-1120) (clinical PPARα antagonist):
Received a "Study May Proceed" letter from the U.S. Food and Drug Administration (FDA) to evaluate amezalpat in combination with atezolizumab (Tecentriq) and bevacizumab (Avastin), the current standard of care for unresectable or metastatic hepatocellular carcinoma (HCC), in a pivotal Phase 3 trial for the first-line treatment of unresectable or metastatic hepatocellular carcinoma.
Announced an agreement with F. Hoffmann-La Roche Ltd. (Roche) to advance the evaluation of amezalpat in combination with atezolizumab and bevacizumab into a pivotal Phase 3 trial for the first-line treatment of unresectable or metastatic HCC, a form of liver cancer with high unmet need.
Under the agreement, Roche will supply atezolizumab globally and Tempest will sponsor and lead the pivotal study. The agreement builds on a clinical collaboration between the companies pursuant to which amezalpat was combined with atezolizumab and bevacizumab in first-line HCC patients and compared to atezolizumab and bevacizumab alone in a randomized Phase 1b/2 study. Tempest retains all development and commercial rights to amezalpat.
Announced positive feedback from the end-of-Phase 2 meeting with the FDA for amezalpat in combination with atezolizumab and bevacizumab to treat first-line unresectable or metastatic HCC. Key outcomes included:
Agreement on Phase 3 study design, including the standard-of-care control arm and the primary and secondary study endpoints.
Agreement on appropriateness of the current amezalpat dose and schedule for the Phase 3 study.
Agreement on the Phase 3 statistical plan, including a pre-specified early efficacy analysis that the company currently estimates could shorten the time to primary analysis by up to 8 months.
Corporate:
Expanded leadership team to strengthen global clinical expertise with the appointments of Troy M. Wagner as Vice President of Quality Assurance and Sheldon Mullins as Vice President of Regulatory Affairs.
Other Potential Future Milestones

TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist)
Plan to advance TPST-1495 into a Phase 2 study in patients with Familial Adenomatous Polyposis (FAP) in 2024 or early 2025 under the auspices of the Cancer Prevention Clinical Trials Network and funded by the National Cancer Institute (NCI) Division of Cancer Prevention.
Expect to disclose data from the TPST-1495 Phase 1 combination arm in patients with advanced endometrial cancer in 2025.
Financial Results

Third Quarter 2024

Tempest ended the quarter with $22.1 million in cash and cash equivalents, compared to $39.2 million on December 31, 2023. Subsequent to September 30, 2024, Tempest raised an additional $19.9 million in net proceeds through the sale of 17 million shares of common stock under the Company’s at-the-market (ATM) program.
Net loss and net loss per share for the quarter ended September 30, 2024, were $10.6 million and $0.41, respectively, compared to $6.8 million and $0.48, respectively, for the same period in 2023.
Research and development expenses for the quarter were $7.6 million compared to $4.2 million for the same period in 2023. The $3.4 million increase was primarily due to an increase in costs incurred from contract research and manufacturing organizations.
General and administrative expenses for the quarter were $3.0 million compared to $2.4 million for the same period in 2023. The $0.6 million increase was primarily due to an increase in stock-based compensation, and consulting and professional services.
Year-to-Date

Cash used in operating activities for the nine months ended September 30, 2024 was $22.9 million.
Net loss and net loss per share for the nine months ended September 30, 2024 were $28.0 million and $1.19, respectively, compared to $22.0 million and $1.57, respectively, for the same period in 2023.
Research and development expenses for the nine months ended September 30, 2024 were $17.7 million compared to $13.3 million for the same period in 2023. The $4.4 million increase was primarily due to an increase in costs incurred from contract research and manufacturing organizations, as well as an increase in stock-based compensation.
General and administrative expenses for the nine months ended September 30, 2024 were $10.4 million compared to $8.3 million for the same period in 2023. The $2.1 million increase was primarily due to an increase in stock-based compensation as well as legal and consulting services.

On November 12, 2024 Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, reported it will initiate a pivotal Phase 2 study in the first quarter of 2025 for the company’s potential first-in-class, investigational, anti-CD7 CAR-T cell therapy, WU-CART-007, in patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL) (Press release, Wugen, NOV 12, 2024, View Source [SID1234648291]).

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"T-ALL/LBL is an aggressive cancer with a significant need for new therapies given that many patients will progress on standard of care," said Kumar Srinivasan, Ph.D., Wugen president and chief executive officer. "Wugen is well positioned for this critical stage of research and development for WU-CART-007. Recently, we generated positive Phase 1/2 results; received significant new regulatory designations – RMAT and PRIME – to expedite our clinical development path in the United States and Europe; and deepened our cell therapy expertise with Cherry T. Thomas, M.D. joining our team as chief medical officer."

The pivotal Phase 2 study is a single-arm trial evaluating the efficacy and safety of WU-CART-007 in patients with R/R T-ALL/LBL and T-ALL/LBL. The study will evaluate the efficacy of WU-CART-007 in two groups: a R/R cohort and an exploratory minimum residual disease (MRD)-positive cohort. The trial will enroll pediatric and adult patients at oncology centers in the United States, Europe, Asia and Australia.

"With a 20-year void of new medicines to treat patients with relapsed or refractory T-ALL/LBL, many of whom are young, we believe off-the-shelf allogeneic CD7-targeted CAR-T cell therapies may offer hope and innovation," said Dr. Thomas, Wugen’s chief medical officer.

"Wugen’s progress in advancing WU-CART-007 also includes a robust, scalable and efficient commercial manufacturing process, developed with our partners to create a CD7-targeted therapy for off-the-shelf use," said Matt Cooper, Ph.D., Wugen’s co-founder and chief scientific officer, who invented WU-CART-007 as a researcher at Washington University in St. Louis, MO.

Wugen previously announced positive results from a Phase 1/2 cohort expansion study showing clinically manageable safety and evidence of anti-leukemic activity (overall response rate of 91%) in heavily pre-treated patients with R/R T-ALL/LBL. Researchers will present a data update at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) 2024, Dec. 7-10 in San Diego.

Follow Wugen’s LinkedIn page to see abstracts to be presented at ASH (Free ASH Whitepaper) 2024 for WU-CART-007 and for WU-NK-101, Wugen’s investigational off-the-shelf memory natural killer cell therapy.

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). More information on the Phase 1/2 trial is available on clinicaltrials.gov, identifier NCT# 04984356 and on the Phase 2 pivotal trial on clinicaltrials.gov, identifier NCT06514794.

WU-CART-007 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and Priority Medicines (PRIME) Scheme designation in the European Union for the treatment of relapsed/refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need.