On September 25, 2023 Shuttle Pharmaceuticals Holdings, Inc. (Nasdaq: SHPH), a discovery and development stage specialty pharmaceutical company focused on improving the outcomes of cancer patients treated with radiation therapy (RT), reported receipt of written responses to questions submitted for a Type B pre-Investigational New Drug Application (PIND) meeting with the U.S. Food and Drug Administration (FDA) (Press release, Shuttle Pharmaceuticals, SEP 25, 2023, View Source [SID1234635393]). The FDA’s positive feedback and guidance on the Company’s Chemistry, Manufacturing, and Controls (CMC) and clinical protocol design for Ropidoxuridine provides a pathway to IND application submission in the fourth quarter of 2023 to initiate the Phase 2 clinical trial.

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"The FDA’s written responses provide clarification and guidance on key aspects to our Phase 2 clinical design and CMC protocols, paving the way to Shuttle’s clinical development of Ropidoxuridine as a radiation sensitizer for treating glioblastoma," commented Shuttle Pharma’s Chairman and CEO, Anatoly Dritschilo, M.D.

Ropidoxuridine (IPdR) is Shuttle’s lead candidate radiation sensitizer for use in combination with RT to treat brain tumors (glioblastoma), a deadly malignancy of the brain with no known cure. Shuttle has received Orphan Drug Designation from the FDA, providing potential marketing exclusivity upon first FDA approval for the disease.

On September 25, 2023 Ascletis Pharma Inc. (HKEX: 1672, "Ascletis") reported the completion of enrollment of 120 patients in the Phase III registration clinical trial of ASC40 combined with bevacizumab for treatment of recurrent glioblastoma (rGBM) (Press release, Ascletis, SEP 25, 2023, View Source [SID1234635392]). ASC40 is an oral, selective small molecule inhibitor of fatty acid synthase (FASN), a key enzyme which regulates de novo lipogenesis (DNL). ASC40 inhibits energy supply and disturbs membrane phospholipid composition of tumor cells by blocking de novo lipogenesis [1].

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The Phase III registration clinical trial (ClinicalTrials.gov Identifier: NCT05118776) is a randomized, double-blind, placebo-controlled, multi-center clinical trial in China to evaluate progression-free survival (PFS), overall survival (OS) and safety of patients with rGBM. Approximately 180 patients will be randomized at the ratio of 1:1 to Cohort 1 (oral ASC40 tablet, once daily + Bevacizumab) and Cohort 2 (matching placebo tablet, once daily + Bevacizumab). Based on prespecified interim analysis condition, 120 patients are likely to lead sufficient events for interim analysis of PFS. The interim analysis will be conducted after 93 PFS events are observed.

Glioblastoma (GBM) is the most aggressive diffuse glioma of astrocytic lineage and is considered a grade IV glioma based on the World Health Organization (WHO) classification [2]. Research shows that glioblastoma (GBM) accounts for 57% of gliomas and has an incidence rate of approximately 2.85 to 4.56 per 100,000 population in China per year, suggesting approximately 40,000 to 64,000 new cases of GBM per year [3]. In the U.S., GBM represents 56.6% of gliomas and has an incidence rate of approximately 3.21 per 100,000 population per year [4]. Over 90% GBM patients will relapse after surgery, radiation and chemotherapies. Effective treatments are extremely limited for patients with rGBM.

"Lipid metabolism is now recognized as an important pathway in cancer [1]. The pivotal role of FASN in lipid metabolism makes it an attractive target in the clinic research." said Dr. Jinzi J. Wu, Founder, Chairman and CEO of Ascletis. "With 120 patients enrolled in the Phase III clinical trial of FASN inhibitor ASC40, we are likely to have sufficient events for the interim analysis of PFS. It’s a milestone of the Phase III study and we are looking forward to the results."

On September 25, 2023 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence (AI) company developing targeted and transformative cancer therapies using its proprietary AI and machine learning (ML) platform, RADR, with multiple clinical stage drug programs, reported the dosing of the first patient in the Phase 1 clinical trial evaluating Lantern’s investigational new drug LP-184 in patients with advanced solid tumors (Press release, Lantern Pharma, SEP 25, 2023, View Source [SID1234635391]).

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"The first patient dosing in our Phase 1 trial of LP-184 is a critical milestone and underscores the commitment of our team to advancing our pipeline of therapies to patients," stated Panna Sharma, Lantern’s President and CEO. "This milestone is about more than advancing a novel drug candidate. It also validates our unique approach of leveraging AI and machine learning to expedite drug development. Insights from our proprietary AI and ML platform, RADR, were instrumental in our development of LP-184 and aided in understanding its mechanism of action, identifying and prioritizing its cancer indications, and generating machine learning biomarker signatures to assist with patient selection in future clinical trials. The rapid advancement of LP-184 into a first-in-human Phase 1 trial underscores the immense potential that we see in this drug candidate, as well as for our approach to AI-enabled drug development. We believe LP-184 has blockbuster potential for patients with multiple types of advanced solid tumors and CNS cancers, many of which have no or limited effective therapeutic options, and we are excited to envision the impact this could have in transforming the lives of patients."

The single arm multicenter Phase 1 trial (NCT05933265) is assessing the safety and tolerability of escalating doses of LP-184 to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D) in patients with advanced solid tumors and recurrent high-grade gliomas, including glioblastoma (GBM). The study has been designed as a 35 patient trial with patients receiving LP-184 infusion on Day 1 and Day 8 of each 21-day cycle, for a minimum of two cycles. Patients will be monitored for safety, pharmacokinetics, and clinical activity, and dose escalation is planned with minimum of three patient cohorts. Lantern has already activated two clinical trial sites and plans to activate multiple additional sites in the US over the next 100 days. Lantern anticipates the Phase 1A portion of the trial to be completed in the first half of 2024.

After the Phase 1 trial is completed, Lantern plans to advance LP-184 into additional clinical trials for multiple solid tumor indications, and Lantern’s subsidiary, Starlight Therapeutics, will advance the clinical development of LP-184 for all brain and CNS indications under the name STAR-001. Globally, the aggregate annual market potential of LP-184/STAR-001’s programs is estimated to be approximately $11-13 billion, consisting of $6-7 billion for solid tumors and $5-6 billion for CNS cancers.

About LP-184:

LP-184 is a unique small molecule that utilizes its powerful mechanism of action, known as synthetic lethality, to exploit common vulnerabilities in solid tumor and CNS cancers with DNA damage repair (DDR) deficiencies. The anti-tumor potential of LP-184 has been demonstrated across an extensive number of in-vitro and in-vivo cancer models, including pancreatic, bladder, triple-negative breast cancer (TNBC), glioblastoma (GBM), brain metastases, and ATRT. In addition to LP-184’s promise as a single agent, its antitumor potency has the potential to be enhanced when used in combination with existing FDA-approved agents and other treatment modalities including spironolactone, PARP inhibitors, and radiation therapy. Results validating LP-184’s anti-tumor potential have been published at leading conferences and journals including, the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, the Society for Neuro-Oncology annual meeting, the San Antonio Breast Cancer Symposium, and the Frontiers in Drug Discovery Journal.

On September 25, 2023 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported that management will participate in a fireside chat at the Cantor Global Healthcare Conference on Tuesday, September 26th at 1:00 pm ET (Press release, Celldex Therapeutics, SEP 25, 2023, View Source [SID1234635390]). A webcast of the presentation will be available on the "Events & Presentations(opens in a new tab)" page of the "Investors & Media(opens in a new tab)" section of the Celldex website. A replay will be available for 90 days following the event.

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On September 25, 2023 Pierre Fabre Laboratories and Vernalis (R&D) Ltd ("Vernalis"), a fully owned subsidiary of HitGen Inc., reported a long-term partnership to identify pre-clinical candidates against multiple oncology targets (Press release, Vernalis, SEP 25, 2023, View Source;utm_medium=rss&utm_campaign=pierre-fabre-laboratories-and-vernalis-announce-a-drug-discovery-collaboration-in-oncology [SID1234635389]).

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Under the terms of the agreement, Vernalis will use its expertise to enable drug discovery against an undisclosed portfolio of targets and use fragment and structure-based methods to identify small molecules that modulate their activity. This research will be combined with the oncology experience of Pierre Fabre Laboratories to identify drug candidates for treatment of a range of cancers for further development and commercialisation by Pierre Fabre Laboratories. The research at Vernalis will be funded by Pierre Fabre Laboratories who will also pay research and clinical milestones and royalties on marketed products. The detailed terms of the agreement are not disclosed.

James Murray, Research Director of Vernalis said: "This strategic partnership is built on the Vernalis track record for innovation and success in enabling drug discovery and identifying clinical candidates for a wide variety of therapeutic targets. We look forward to building this long-term partnership with Pierre Fabre Laboratories, leveraging their deep expertise in cancer biology and clinical development."

Francesco Hofmann, Head of Research and Development for Medical Care at Pierre Fabre Laboratories said: "Partnering with Vernalis represents an important milestone for us. Leveraging the expertise of Vernalis in key areas of Structure Based Drug Design to deliver high quality clinical candidates on multiple Oncology targets is a real asset to develop our Small Molecule Drug Discovery pipeline".