On November 20, 2024 The Cancer Prevention & Research Institute of Texas (CPRIT) reported that it plans to award Orphagen a $10.2 M product development grant over three years to partially fund the final steps towards IND filing and a Phase 1 clinical trial of OR-449, a novel small molecule and orally-bioavailable therapeutic candidate for an intractable cancer, adrenocortical cancer (ACC) (Press release, Orphagen, NOV 20, 2024, View Source [SID1234649065]). OR-449 may be developed subsequently in head & neck and lung squamous cancers as a precision therapy for the 3-10% of these tumors where its target is highly expressed. Details of today’s announcement are here: CPRIT.grants.announcement.Nov.20.2024.

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A major site for the Phase 1 trial would be the MD Anderson Cancer Center in Houston, internationally recognized for its expertise in treatment of ACC. To initiate and fully comply with the CPRIT grant, Orphagen will raise additional matching funds and move its headquarters to Texas in 2025.

Scott Thacher, Orphagen’s CEO and founder, stated, "This is an important step forward for our programs. OR-449 is the first of our internal programs that is on the path to a Phase 1 trial."

"Further OR-449 represents a significant opportunity for improved therapy of ACC patients, whose options for medical therapy are limited. The target of OR-449 is the orphan nuclear receptor SF-1, now recognized as a potential driver of ACC."

Orphagen Pharmaceuticals is a preclinical stage biopharmaceutical company focused on discovering and developing first-in-class therapeutics for cancer and inflammatory disease. Orphagen is a pioneer in the screening, discovery and development of ligands that modulate orphan or unexplored members of the nuclear receptor family. On a per target basis, the nuclear receptors are one of the most successful target classes known to the pharmaceutical industry.

Its first-in-class SF-1 antagonist (OR-449) has practically completed IND-enabling studies. Potential expansion indications for OR-449 include other cancers where SF-1 expression is highly elevated in such a way to suggest these depend on SF-1 activity for tumor growth.

On November 20, 2024 Valora Therapeutics Inc. ("Valora"), a biotechnology company pioneering a novel approach to immunotherapy, reported the successful closing of its seed funding round (Press release, Valora Therapeutics, NOV 20, 2024, View Source [SID1234648535]). The investment was co-led by Avalon BioVentures, a top-tier investor group with a long history of nurturing groundbreaking life science companies from inception to exit, together with Bregua Corporation and TigerGene, investors recognized for their expertise in supporting innovative biotech companies. Additional participation was provided by Alexandria Venture Investments and Correlation Ventures. This significant investment will propel the advancement of Valora’s proprietary AbLec platform to develop novel immunotherapeutics.

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"The AbLec platform represents a significant leap forward in manipulating glycoimmunology and unlocking new paradigms in immunotherapy. I look forward to providing scientific support to Valora as it advances lead molecules toward clinical development."

Valora’s AbLec (Antibody-Lectin chimera) platform is expected to offer a fresh perspective on restoring proper immunological competency. By precisely targeting specific sugar molecules on cells, AbLecs modulate glyco-immune checkpoints—a key control point in the body’s immune response. This innovative approach holds significant potential for developing first-in-class and best-in-class therapeutics in oncology, autoimmune diseases, and other therapeutic areas. The technology is exclusively licensed from Stanford University. It builds upon the transformative work performed at the Stanford laboratory of Dr. Carolyn Bertozzi, recipient of the 2022 Nobel Prize in Chemistry, and a world leader in glycobiology, together with Dr. Jessica Stark, who is currently an Assistant Professor of Biological Engineering and Chemical Engineering at the Massachusetts Institute of Technology. Both Drs. Carolyn Bertozzi and Jessica Stark are scientific founders and advisors for Valora.

"We are incredibly honored to partner with highly recognized investors and Valora’s scientific founders, Dr. Carolyn Bertozzi and Dr. Jessica Stark, to develop the AbLec platform as a trailblazing approach for next-generation immunomodulators," said Miguel Garcia-Guzman, Ph.D., Valora’s CEO. "This funding allows us to accelerate our R&D efforts, optimizing the AbLec platform and advancing AbLec therapeutics toward the clinic. We take great pride in the work we do at Valora and are deeply grateful for the support of our investors."

Dr. Stark added, "The AbLec platform represents a significant leap forward in manipulating glycoimmunology and unlocking new paradigms in immunotherapy. I look forward to providing scientific support to Valora as it advances lead molecules toward clinical development."

The seed funding will enable Valora to further explore and develop the full potential of the AbLec platform, validating specific mechanisms of action, and advancing lead molecules into preclinical development. Valora has established its R&D operations at the Avalon BioVentures Accelerator in San Diego, California, providing access to a supportive ecosystem of biotech experts and resources.

Sandy Madigan, Ph.D., Managing Partner at Avalon BioVentures and a Board Member of Valora Therapeutics, stated, "We are excited to co-lead the seed funding round for Valora. We believe Valora has the team and resources to lead the development of novel AbLec therapeutics and to rapidly advance this groundbreaking technology toward the clinic."

Stefan Heller, Ph.D., who represents Bregua Corporation on the Board of Valora Therapeutics, added, "We are excited to collaborate with Valora’s team to advance the AbLec platform for developing novel therapies. This innovative approach has promising potential for creating leading-edge treatments across a range of therapeutic areas."

Audrey Warner, Partner at TigerGene and Director of Valora Therapeutics concluded, "We are very excited to partner with Valora’s team to explore the promise of glyco-immune checkpoint modulation and deliver innovative biological therapeutics to alleviate patient suffering across a multitude of indications."

On November 20, 2024 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported that its management team will participate at the following investor conferences (Press release, Personalis, NOV 20, 2024, View Source [SID1234648534]):

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– Piper Sandler 36th Annual Healthcare Conference
Date: Tuesday, December 3, 2024
Fireside Chat Time: 12:30 pm Eastern Time
Location: The Lotte New York Palace in New York, NY

– TD Cowen Diagnosing Tomorrow: Tools & Technologies for the Next Decade
Date: Thursday, December 12, 2024
Panel Topic: MRD – The Future Tech Stack
Panel Time: 1:30 pm Eastern Time
Location: One Vanderbilt in New York, NY

On November 20, 2024 Schrodinger, Inc. (Nasdaq: SDGR) reported that management will participate in a fireside chat at the Piper Sandler 36th Annual Healthcare Conference (Press release, Schrodinger, NOV 20, 2024, View Source [SID1234648533]). The live presentation will take place on Wednesday, December 4, 2024 at 8:30 a.m. ET.

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The live webcast can be accessed in the "Investors" section of Schrödinger’s website and will be archived for approximately 90 days following the event.

On November 20, 2024 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported the initiation of its ALISertib in CAncer (ALISCA-Breast1) Phase II trial (PUMA-ALI-1201; NCT06369285) of alisertib in combination with endocrine therapy for the treatment of patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-negative) recurrent or metastatic breast cancer who have been previously treated with CDK 4/6 inhibitors and received at least two prior lines of endocrine therapy in the recurrent or metastatic setting (Press release, Puma Biotechnology, NOV 20, 2024, View Source [SID1234648532]). The ALISCA-Breast1 trial will enroll up to 150 patients who will be randomized (1:1:1) to receive alisertib dosed at either 30 mg, 40 mg or 50 mg twice daily on days 1-3, 8-10 and 15-17 in a 28-day cycle in combination with the endocrine therapy of the investigator’s choice. Patients must provide blood and tissue specimens so that biomarkers can be analyzed.

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"Additional therapies are needed for patients with HER2-negative, HR+ metastatic breast cancer whose disease progresses on CDK4/6 inhibitors in the first-line setting"

The primary objective of the trial is to determine the optimal alisertib dose in combination with selected endocrine therapy. The primary endpoints of the trial include objective response rate, duration of response, disease control rate, progression-free survival, and overall survival. As a secondary endpoint, Puma will evaluate each of these efficacy endpoints within biomarker subgroups in order to determine whether any biomarker subgroup correlates with response. Puma will perform its biomarker analysis of the ALISCA-Breast1 trial in parallel with the execution of the clinical trial. Puma plans to perform an initial interim analysis for the evaluation of safety and efficacy.

Based upon the outcomes of the trial, Puma anticipates meeting with the U.S. Food and Drug Administration to explore the potential for an approval pathway for alisertib in HER2-negative, HR+ metastatic breast cancer. Once the optimal alisertib dose is identified, Puma plans to engage with global regulatory agencies regarding the design of a pivotal (Phase III) trial, which it anticipates will be a randomized trial of alisertib plus investigator’s choice endocrine therapy versus placebo plus investigator’s choice endocrine therapy in patients with HER2-negative, HR+ metastatic breast cancer.

"Additional therapies are needed for patients with HER2-negative, HR+ metastatic breast cancer whose disease progresses on CDK4/6 inhibitors in the first-line setting," said Joyce A. O’Shaughnessy, M.D., the Celebrating Women Chair in Breast Cancer Research at Baylor University Medical Center, Texas Oncology, Sarah Cannon Research Institute in Dallas, Texas. "The results from the TBCRC 041 trial indicated that alisertib has impressive clinical activity in the setting of endocrine therapy and CDK4/6 inhibitor-resistant metastatic breast cancer, with good tolerability. I look forward to the further evaluation of alisertib in the ALISCA-Breast1 trial to definitively determine the clinical impact of this treatment."

Alan H. Auerbach, Chief Executive Officer, President and Founder of Puma, stated, "We are excited to initiate this Phase II trial and to move forward with the development of alisertib in HER2-negative HR+ metastatic breast cancer. We believe that the data from the previous trial of alisertib monotherapy (published in Lancet Oncology) as well as the TBCRC 041 trial (published in JAMA Oncology), which tested alisertib alone and with fulvestrant, and the randomized trial of alisertib plus paclitaxel versus paclitaxel alone (published in JAMA Network Open) have demonstrated that alisertib is active in patients with HER2-negative, HR+ metastatic breast cancer and in biomarker focused subgroups. We look forward to enrollment in the ALISCA-Breast1 trial and anticipate that we should have initial data from this trial in 2025."