On October 22, 2024 Scholar Rock Holding Corporation (Nasdaq: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, reported that on October 16, 2024, the underwriters of its previously announced underwritten public offering of common stock and pre-funded warrants, which closed on October 10, 2024, exercised their option in full to purchase an additional 1,592,920 shares at the public offering price of $28.25 per share, resulting in additional gross proceeds of approximately $45 million, before deducting underwriting discounts and commissions and offering expenses payable by Scholar Rock (Press release, Scholar Rock, OCT 22, 2024, View Source [SID1234647324]). The closing of the underwriters’ exercise of their option to purchase 1,592,920 additional shares occurred on October 18, 2024.

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J.P. Morgan Securities LLC, Jefferies and Piper Sandler & Co. acted as joint book-running managers for the offering. BMO Capital Markets Corp., Wedbush Securities Inc. and Raymond James & Associates, Inc. acted as co-managers for the offering.

An automatically effective shelf registration statement on Form S-3 relating to the offering of the shares of common stock and pre-funded warrants described above was filed with the Securities and Exchange Commission (SEC) on October 7, 2024. A preliminary prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC on October 7, 2024, and are available on the SEC’s website located at www.sec.gov. The final prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC on October 9, 2023 and may be obtained by contacting: J.P. Morgan Securities LLC, c/o: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by email at [email protected] and [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at 877-821-7388, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at 800-747-3924, or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction.

On October 22, 2024 SOTIO Biotech, a clinical-stage immuno-oncology company owned by PPF Group, reported it will present three posters at the 2024 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Meeting taking place November 6 – 10, 2024, in Houston, TX (Press release, SOTIO, OCT 22, 2024, View Source [SID1234647323]).

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Data to be presented include the investigation of a novel chimeric PGC-1α transgene to enhance CAR T cell efficacy in patients with solid tumors. The VICTORIA-01 study is currently evaluating the safety, tolerability and initial efficacy of SOT201 monotherapy for the treatment of advanced solid tumors, as well as data around SOT201, a next-generation clinical stage PD-1-targeting immunocytokine.

Poster details are as follows:

Title: "Chimeric PGC-1α expression in CAR-T cells improves metabolic function and anti-tumor efficacy in solid tumors"
Abstract Number: 273
Date: Thursday, November 7 and Friday, November 8, 2024
Presenting Author: Amy Jensen-Smith

Title: "VICTORIA-01: A multicenter, open-label, phase 1 study to evaluate the safety and preliminary efficacy of SOT201 in patients with advanced or metastatic solid tumors"
Abstract Number: 675
Date: Friday, November 8, 2024
Presenting Author: Aung Naing

Title: "SOT201, a novel cis-acting PD-1/IL-15 mutein-based immunocytokine that reinvigorates anti-tumor immunity qualitatively superior to PD-1/IL-2v-based IL-2/15Rβγ agonism"
Abstract Number: 940
Date: Saturday, November 9, 2024
Presenting Author: Irena Adkins

On October 22, 2024 NeoGenomics, Inc. (NASDAQ: NEO), a leading oncology testing services company, reported that the New York State Department of Health (NYSDOH) has granted the company conditional approval for its Neo Comprehensive Solid Tumor assay and NeoTYPE DNA & RNA Lung, allowing for immediate commercial access in the state of New York (Press release, NeoGenomics Laboratories, OCT 22, 2024, View Source [SID1234647322]). These next-generation sequencing (NGS) tests deliver better diagnostic value and cost-effectiveness than single gene testing and direct the first-line treatment of diverse solid tumors, including non-small cell lung cancer.

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New York State is known for having stringent validation standards for laboratory-developed tests. Clinical laboratories testing specimens from New York residents must obtain a clinical laboratory permit from NYSDOH to help ensure the accuracy and reliability of clinical tests.

"Receiving approval for Neo Comprehensive Solid Tumor and NeoTYPE DNA & RNA Lung from NYSDOH marks a significant milestone for NeoGenomics, confirming the company’s high standards for developing new tests, the quality of our lab results, and the impact these tests have on guiding management of diverse cancers," said Warren Stone, CCO of NeoGenomics. "More importantly, NeoGenomics can now bring these tests to more healthcare providers and their patients across New York state, continuing our mission of transforming care for cancer patients."

Each year, 116,000 people are diagnosed with cancer in New York State, with lung cancer representing the leading cause of cancer-related death. Neo Comprehensive offers physicians a pan-cancer genomic profiling solution that aligns with National Comprehensive Cancer Network (NCCN) guidelines for diverse cancers. NeoTYPE DNA & RNA Lung leverages the same technology as Neo Comprehensive Solid Tumor but in the context of a more focused NGS panel that addresses an unmet need in lung cancer testing across the state. These tests analyze a broad panel of genes, which detect less common genetic markers that cancer-specific profiles or single-gene tests may miss.

NeoGenomics operates Clinical Laboratory Improvement Amendments (CLIA) certified and College of American Pathologists (CAP) accredited laboratories for full-sample processing across the U.S. Widely recognized as the highest standard for clinical laboratory accreditations, the CAP Laboratory Accreditation Program is designed to ensure laboratories meet stringent requirements and standards of quality, safety, and accuracy.

On October 22, 2024 CEL-SCI Corporation (NYSE American: CVM) reported the potential positive impact on the clinical development of its immunotherapy Multikine (Leukocyte Interleukin, Injection)* resulting from a recent U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) meeting, a public forum (Press release, Cel-Sci, OCT 22, 2024, View Source [SID1234647321]).

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FDA advisory committees provide independent expert advice to the FDA on the safety and effectiveness of new and marketed drugs and help the agency make sound and informed decisions. Advisory committees make non-binding recommendations to the FDA, which generally follows the recommendations but is not legally bound to do so.

The September 27, 2024 ODAC meeting evaluated the use of checkpoint inhibitors on patients with various cancers. PD-L1 is the biomarker most often used for patient selection for checkpoint inhibitors, the most successful class of cancer drugs including Keytruda and Opdivo. The FDA sought the ODAC’s opinion on the following:

adequacy of PD-L1 expression as a predictive biomarker for patient selection in this patient population
differing risk-benefit assessments in different subpopulations defined by PD-L1 expression
adequacy of the cumulative data to restrict the approvals of immune checkpoint inhibitors based on PD-L1 expression
Following a thorough analysis of peer-reviewed published data, the panel of experts on the ODAC voted 10-2 and 11–1 against the risk-benefit profile for PD-L1 inhibitors in various cancers in two separate votes. Most ODAC members expressed concerns about the lack of benefit demonstrated for patients with low PD-L1 expression, while some members pointed to evidence that the use of the immune checkpoint inhibitors may add unnecessary toxicities for patients while also increasing financial burdens on patients.

Checkpoint inhibitors were an estimated $48 billion global market in 2023, with PD-L1 inhibitors representing 73% of the market. Current labeling for approved checkpoint inhibitors in the indications evaluated by the FDA’s ODAC include broad approvals for all patients, regardless of PD-L1 expression.

"The FDA advisory committee’s findings and public statement on the risk-benefit of checkpoint inhibitors based on PD-L1 expression is potentially a turning point in cancer treatment, pointing to a major treatment gap in low and negative PD-L1 patients. While the approved blockbuster checkpoint inhibitors, including Keytruda which is expected to be the biggest selling drug in 2024, work well in high PD-L1 expressing patients, to our knowledge, Multikine is the only immunotherapy that is targeted to and has shown survival benefit in the low and negative PD-L1 population. CEL-SCI has already identified in our prior study low PD-L1 expression as a predictive biomarker, representing about 70% of head and neck cancer patients, which will be used as a selection criterion for our upcoming FDA confirmatory Registration Study of Multikine in the treatment of treatment-naïve resectable locally advanced head and neck cancer," stated CEL-SCI CEO Geert Kersten.

"The FDA’s ODAC decision further supports our belief that Multikine holds strong potential in treating patients across a wider range of solid tumor cancer indications alone and in combination regimens," Kersten concluded.

On October 22, 2024 Oncodesign Precision Medicine (OPM) (ISIN: FR001400CM63; Mnemonic: ALOPM), a biopharmaceutical company specializing in precision medicine for the treatment of resistant and metastatic cancers, reported details of the final results of its Phase 1 clinical trial with OPM-101, its RIPK2 inhibitor and drug candidate administered orally to healthy volunteers, first reported on July 16, 2024 (Press release, Oncodesign Precision Medicine, OCT 22, 2024, View Source [SID1234647320]). This clinical trial began in February 2023, the database was frozen in June 2024, and the full results and final study report became available, as scheduled in the study timetable, in October 2024.

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OPM-101 is an experimental, powerful and selective small molecule inhibitor of the RIPK2 kinase. OPM-101 is designed to modulate the pro-inflammatory signal transmission pathway of this kinase, which is responsible for the development of inflammatory diseases, and has the potential to treat diseases in the fields of IBD (Chronic Inflammatory Bowel Disease) and immuno-oncology.

This randomized, double-blind, placebo-controlled phase 1 study was designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of OPM-101 (EudraCT: 2022-003122-50) in 104 healthy volunteers:

– In the SAD part of the trial, 72 HV (mean age = 34 years) received a single oral dose of placebo or 5, 20, 60, 150, 300, 600 or 1,000 mg of OPM-101, with one cohort dedicated to the high-fat meal effect and another to the gender effect, also receiving 150mg.

– In the MAD part of the trial, 32 HV (mean age = 36 years) received an oral dose of 75, 150 or 300 mg of OPM-101 or placebo twice daily for 14 consecutive days, including a cohort dedicated to the gender effect, receiving 150mg twice a day.

Safety assessments (physical examination, vital signs, blood tests, ECGs, holters) were performed regularly throughout the trial. In addition, blood samples were collected to measure circulating concentrations of OPM-101 and engagement of RIPK2, the pharmacological target of OPM-101 (measured by inhibition of stimulated TNFα production).

Robust safety data, no toxicity on cardiac parameters

OPM-101 was very well tolerated after administration in SAD and MAD. No severe or serious adverse events were reported, and no volunteer discontinued the study. All treatment-emergent adverse events considered to be at least possibly drug- and study-related were mild to moderate. Fourteen healthy volunteers (13.5%) reported a total of 18 adverse events considered related to study treatment (OPM-101 or placebo). Among them, 12 healthy volunteers (15%) reported a total of 15 adverse events considered to be related to OPM-101, the majority of which were headaches. 80% of these events were mild and 20% moderate. There were no clinically significant changes in safety-related laboratory tests reported during the treatment periods for all OPM-101 dose cohorts included in the analysis, with the exception of one volunteer who received a dose of 150mg twice daily and experienced a moderate (3x normal) increase in ALT (liver enzyme) during MAD, leading to discontinuation of her treatment on the morning of study day 12. In this volunteer, further increases in liver proteins were not observed.

Cardiac function was monitored in detail with daily 12-lead ECGs, particularly at the time of peak circulating concentrations. 24-hour ECG recordings (holters) and cardiac ultrasound were also performed. All these examinations showed normal results, without any relation to circulating OPM-101 concentrations, and without any clinically significant changes. This type of monitoring is common at this stage of development, particularly for kinase inhibitors. The results obtained make OPM-101 a first-rate molecule in this field.

Promising pharmacokinetic and pharmacodynamic results

OPM-101 significantly inhibits the RIPK2 pathway. Target engagement kinetics and pharmacodynamic results showed a fast and marked onset of inhibitory effect, already observed 2-6h after first administration, with maximal inhibition of 90-100%, and minimal inhibition of 65%-85% maintained over 14 days of dose-dependent administration. Inhibition remained marked (50% to 80%) 24 hours after the last administration on day 14 in the MAD part.

The pharmacokinetic parameters of OPM-101 showed consistent results between the SAD and MAD parts, and characteristics suitable for use in patients. OPM-101 is rapidly absorbed, with a Tmax observed between 2-4h, a terminal half-life of around 12h, steady-state reached after 3-4 days and dose-dependent exposure with repeated administration.

On the basis of the PK/PD relationship determined from the results of the MAD part of the study, we anticipate that a very significant target commitment (≥80%) can be achieved and maintained with a residual OPM-101 plasma concentration (Cmin) in the range 150-300 ng/mL. In addition, population pharmacokinetic modelling has enabled us to simulate different dose regimes for a future clinical study, and the target dose could be 150mg twice daily.

OPM and Professor Peyrin-Biroulet, presented the phase 1 results at the United European Gastroenterology Week (UEGW) 2024 held from October 12 to 15, 2024 in Vienna, Austria.

Based on the results presented today, OPM plans to submit a protocol for a phase 1b/2a clinical study in the fourth quarter of 2024.

"We are very pleased with the progress and results of this clinical trial with OPM-101, which provided convincing results for all primary, secondary and exploratory endpoints included in this study," said Philippe Genne, Chief Executive Officer of OPM. "We are pleased to demonstrate the safety of our candidate and the strong pharmacodynamic correlation that exists. High target engagement is demonstrated at tolerated doses of OPM-101 throughout the treatment period. The modulation of TNFα production ex vivo can be considered as a key biomarker of target engagement for future clinical trials. The clinical results reported today not only highlight the consistency with the immunomodulatory effect of OPM-101 observed in preclinical studies, but also validate OPM-101 as a safe and effective inhibitor of the RIPK2 pathway. We are currently in the process of identifying with our scientists and clinicians’ experts the first clinical indication that we will explore in a phase 1b/2a study before the end of the year in order to provide a first clinical proof of concept in a patient population capable of generating significant added value for our asset".

"These complete Phase 1 results confirm and validate OPM-101 as a highly specific, effective and well-tolerated inhibitor of the RIPK2 immune pathway," added Jan Hoflack, Deputy CEO and Chief Scientific Officer of OPM. "The field related to this therapeutic approach is currently booming with new high quality preclinical and clinical scientific publications mentioning a potential role for an inhibitor like OPM-101 in multiple immuno-oncology indications, in addition to the already well-established rational for the treatment of IBD and other inflammatory diseases. Our team is currently working to validate the different therapeutic options available for OPM-101, with the aim of launching a proof-of-concept clinical trial in relevant patients rapidly and efficiently before the end of the year. The current idea of a safe and effective RIPK2 inhibitor like OPM-101 suggests significant potential in both IBD and immuno-oncology, two of today’s largest pharmaceutical markets with significant unmet needs".