On February 15, 2024 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported the first patient has been dosed in the Company’s Phase 1 LIONS (PLK4 Inhibitor in Advanced Solid Tumors) clinical trial evaluating RP-1664, a potential first-in-class, highly selective, oral polo-like kinase 4 (PLK4) inhibitor, for the monotherapy treatment of adult and adolescent patients enriched for TRIM37-high solid tumors (Press release, Repare Therapeutics, FEB 15, 2024, View Source [SID1234640172]).

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"RP-1664 exhibited deep tumor growth inhibition and regressions in multiple TRIM37-high solid tumor and neuroblastoma xenograft models, both internally and in collaboration with Children’s Hospital of Philadelphia. After evaluating safety in the LIONS clinical trial, we expect to move rapidly into a Phase 1/2 clinical trial in high risk, recurrent pediatric neuroblastoma, in which patients have a high prevalence of TRIM37-altered tumors and limited treatment options," said Maria Koehler, MD, PhD, Executive Vice President and Chief Medical Officer of Repare. "RP-1664 is Repare’s third internally-developed clinical therapeutic candidate, a testament to the productivity of our platform."

The LIONS clinical trial (NCT06232408) is a first-in-human, multicenter, open-label Phase 1 study to investigate safety, pharmacokinetics, pharmacodynamics and the preliminary efficacy of RP-1664. The clinical trial is expected to enroll approximately 80 patients with molecularly selected advanced solid tumors, including those with gain or amplification of TRIM37, among other genetic alterations. The primary endpoints are to determine the safety, tolerability, dose and schedule of RP-1664 and assess any early antitumor activity.

About RP-1664

RP-1664 is a potential first-in-class, highly selective, oral PLK4 inhibitor designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors. Tumors rely on PLK4 for centriole biogenesis in S-phase of the cell cycle when TRIM37, an E3 ligase that reduces pericentriolar material, is high. Preclinical studies demonstrate that RP-1664 selectively inhibits PLK4 and drives potent synthetic lethality in TRIM37-high tumor models, both in vitro and in vivo. Elevated TRIM37 is a feature found across a range of solid tumors and in approximately 80% of all high-grade neuroblastomas. RP-1664 is the only selective PLK4 inhibitor known to be in the clinic.

On February 15, 2024 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended December 31, 2023, as well as key recent clinical and corporate developments (Press release, Cel-Sci, FEB 15, 2024, View Source [SID1234640171]).

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Clinical and Corporate Developments include:

CEL-SCI identified the target head and neck cancer patient population for Multikine (Leukocyte Interleukin, Injection)* that will be the basis for the Company’s regulatory filings for marketing clearance. In October 2023, the new data were presented at the 2023 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. The target population, which saw its 5-year risk of death cut in half, can be identified prior to surgery upon diagnosis with tests that physicians routinely use in cancer screenings, a key finding for Multikine, which is a neoadjuvant therapy. A summary of Multikine’s results in the target population include the following:
73% survival for Multikine vs 45% in the control at 5 years
28% absolute survival benefit
Statistically significant p = 0.0015 and hazard ratio = 0.35
Tumor reduction rate >13% and tumor downstaging >35%
No safety signals or toxicities vs standard of care
Target population of an estimated 145,000 patients (global, annual) with newly diagnosed squamous cell carcinoma of the head and neck (SCCHN) who present with:
No lymph node involvement (via PET scan)
Low PD-L1 tumor expression (TPS<10) (via biopsy).
Physicians routinely assess these features at baseline; no extra tests needed. These features make it easy to write a label for Multikine, which is essential for drug approval
CEL-SCI estimates that low PD-L1 patients represent about 70% of locally advanced primary SCCHN patients
CEL-SCI issued a comprehensive Letter to Shareholders detailing the data reported on the efficacy of Multikine in the head and neck cancer target patient population
CEL-SCI’s cGMP state-of-the-art dedicated manufacturing facility commissioning was completed, a significant milestone toward a planned Biologics License Application (BLA) with several regulatory agencies for approval of Multikine. The Company’s manufacturing trade secrets, capabilities, and know-how are high-value key strategic assets that are very difficult for others to replicate.
The United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE) selected Multikine to be evaluated as the potential new standard of care for SCCHN. NICE posted a detailed report from the UK’s National Institute for Health and Care Research (NIHR) regarding Multikine, its clinical data, and its potential to become a better standard of care in treating newly diagnosed head and neck cancer in the UK.
The European Medicines Agency’s (EMA) Paediatric Committee granted CEL-SCI a product-specific waiver of strict requirements for commercialization of cancer drugs in the European Union (EU). The waiver is a big step forward for Multikine, as it removes a major hurdle on the path towards commercialization in Europe.
CEL-SCI plans to submit the target population data to the U.S. Food and Drug Administration (FDA) this quarter. Health Canada advised CEL-SCI to request advance consideration for approval under a Notice of Compliance with Conditions (NOCC) policy. Meetings with the UK regulators and the EMA are expected H1 2024.
"Following the identification of our focused patient population, backed by robust efficacy data, we have made progress with global regulators, including in the UK and EU where we expect meetings in the coming months. Our manufacturing facility is fully commissioned and should soon be ready for commercial-scale production," stated CEL-SCI CEO, Geert Kersten. "We are optimistic about working with regulators to get Multikine to the patients who need it. Statistically significant data demonstrate our target patient population can benefit from a longer life with Multikine."

Financial Results

The Company incurred a net operating loss of approximately $6.5 million for the three months ended December 31, 2023, approximately $2.6 million of which was non-cash expenses.

During the three months ended December 31, 2023, research and development expenses decreased by approximately $1.0 million, or 19%, compared to the three months ended December 31, 2022. During the three months ended December 31, 2023, general and administrative expenses decreased by approximately $0.1 million, or 6%, compared to the three months ended December 31, 2022.

CEL-SCI raised $5 million in November 2023 and $7.75 million in February 2024, both through public offerings of common stock.

On February 15, 2024 Incyte (Nasdaq:INCY) reported that it will present at the Cowen 44th Annual Health Care Conference on Monday, March 4, 2024 at 9:50 a.m. (EST) in Boston (Press release, Incyte, FEB 15, 2024, View Source [SID1234640170]).

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The presentation will be webcast live and can be accessed at Investor.Incyte.com and will be available for replay for 30 days.

On February 15, 2024 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported the company will participate in the following investor conferences (Press release, Guardant Health, FEB 15, 2024, View Source [SID1234640169]).

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Citi 2024 Unplugged Medtech and Life Sciences Access Day in New York
Fireside Chat on Thursday, February 29 at 1:00 p.m. Eastern Time / 10:00 a.m. Pacific Time
TD Cowen 44th Annual Health Care Conference in Boston
Fireside Chat on Tuesday, March 5 at 9:50 a.m. Eastern Time / 6:50 a.m. Pacific Time

Interested parties may access live and archived webcasts of the sessions on the "Investors" section of the company website at: www.guardanthealth.com.

On February 15, 2024 Kurome Therapeutics Inc. reported that the U.S. Food and Drug Administration (FDA) has cleared the IND for KME-0584, allowing the company to proceed with a Ph1 clinical trial in relapsed/refractory (R/R) AML and high-risk (HR) MDS patients (Press release, Kurome Therapeutics, FEB 15, 2024, View Source [SID1234640168]). Kurome plans to initiate the clinical trial in the latter half of 2024.

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"This is a major milestone for the Kurome team and our collaborators and validates our unique approach to AML and MDS as well as the suitability of KME-0584 for initial clinical testing," said Jan Rosenbaum, Ph.D., CEO and CSO at Kurome. "We look forward to getting our clinical trial underway and testing our approach of targeting dysregulated immune signalling in the setting of AML and HR-MDS by targeting both IRAK1 and IRAK4 together to improve efficacy in this difficult to treat R/R patient population."

About KME-0584
KME-0584 is a potent and highly selective small molecule inhibitor of interleukin 1 receptor associated kinases (IRAK)1, IRAK4, and all mutations of FMS-like receptor tyrosine kinase-3 (FLT3), for the treatment of R/R AML or HR-MDS. KME-0584 is intended for oral administration as a monotherapy, and as combination therapy with azacitidine or venetoclax.

About the Phase 1 Clinical Trial
The Phase I study will evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of KME-0584 alone or in combination with venetoclax or azacitidine in patients with AML and HR MDS. The study will include a dose escalation and an expansion phase with up to 100 total participants. Kurome Therapeutics is planning to start the study in 2024 across multiple investigative sites in the US.