On January 13, 2025 Avalo therapeutics presented its corporate presentation (Presentation, Avalo Therapeutics, JAN 13, 2025, View Source [SID1234649645]).

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On January 13, 2025 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company seeking to develop and commercialize more effective and better tolerated therapies for cancer patients, reported a positive update from its Phase 2a arm studying IMM-1-104 in combination with modified FOLFIRINOX (mFFX) in first-line pancreatic cancer, and provided a corporate update (Press release, Immuneering, JAN 13, 2025, View Source [SID1234649661]).

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"We are thrilled to report two more responses in our Phase 2a arm studying IMM-1-104 in combination with modified FOLFIRINOX in first-line pancreatic cancer," said Ben Zeskind, Ph.D., CEO of Immuneering. "The response rates emerging from both of our Phase 2a combination arms in first-line pancreatic cancer are comparable to one another, and speak not only to IMM-1-104’s potential to drive a new standard of care in pancreatic cancer, but also its potential as a first-of-its-kind, well-tolerated MEK inhibitor that could be safely used in a variety of combinations to drive better outcomes for patients across a range of indications."

Zeskind continued: "Building on our positive January 7, 2025 data update, we strengthened our cash balance with nearly $14 million in net proceeds raised through our ATM facility. We began 2025 strong and intend to sustain that momentum throughout the year, including with a planned second quarter 2025 Phase 2a trial data update, the anticipated initiation of new combination trial arms, and planning for a pivotal trial of IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel."

Since the Company’s prior update on January 7, 2025 (which used a data cutoff date of December 5, 2024), two new partial responses (PRs) have been reported.
As of January 6, 2025, there were six evaluable patients in the Phase 2a arm evaluating IMM-1-104 with modified FOLFIRINOX in first-line pancreatic cancer; three patients achieved partial responses (one unconfirmed) for an overall response rate of 50% (3/6). Four patients remain on treatment. The historic benchmark ORR is 32% for FOLFIRINOX alone.
The combination of IMM-1-104 plus modified FOLFIRINOX (mFFX) was observed to be generally well tolerated.
The Company is currently evaluating the 320 mg QD dose of IMM-1-104 in combination with modified FOLFIRINOX.
Additionally, Immuneering reported that it raised net proceeds of $13.7 million through utilization of its ATM facility following its January 7, 2025 update from its ongoing Phase 2a clinical trial.

Near-Term Milestone Expectations

IMM-1-104

Further IMM-1-104 Phase 2a data expected in the second quarter of 2025
Initiation of Phase 2a arms of IMM-1-104 in combination with a BRAF inhibitor, as well as IMM-1-104 in combination with a checkpoint inhibitor, planned for 2025

On January 13, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported its preliminary fourth quarter and full year 2024 U.S. net product revenue for OGSIVEO (nirogacestat) and provided additional company updates ahead of its presentation at the 43rd Annual J.P. Morgan Healthcare conference (Press release, SpringWorks Therapeutics, JAN 13, 2025, View Source [SID1234649677]).

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Preliminary Fourth Quarter and Full Year 2024 Financial Results* and Recent Business Highlights

Preliminary fourth quarter and full-year 2024 U.S. net product revenue for OGSIVEO were $61.5 million and $172.0 million, respectively.
As of December 31, 2024, total preliminary cash, cash equivalents, and marketable securities was $461.9 million. SpringWorks expects its cash position to fund operations through profitability, which the Company anticipates achieving in the first half of 2026.
Presented long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors in the fourth quarter of 2024. These results showed that longer-term treatment with nirogacestat (median duration of treatment: 34 months) was associated with further reductions in tumor size, increase in objective response rate with additional partial responses and complete responses, sustained improvements in desmoid tumor symptoms including pain, and a consistent safety profile.
Obtained an exclusive, global license from Rappta Therapeutics Oy for a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes. PP2A mutations represent a class of targetable oncogenic drivers in molecularly defined subsets of uterine cancer patients with high unmet need. In preclinical models of PP2A mutant uterine cancer, SW-3431 (formerly RPT04402) showed rapid, deep and durable tumor regressions as a monotherapy. In exchange for the license, Rappta received a $13 million upfront payment and is eligible to receive further clinical, regulatory and commercial milestone payments, and tiered single digit royalties on net sales.
* The preliminary fourth quarter and full year 2024 financial results are unaudited and do not present all information necessary for an understanding of the Company’s results of operations and financial position for the fourth quarter and full year 2024. Such financial results are subject to adjustment and could differ from the Company’s announcement of complete financial results in February 2025.

2025 Priorities and Anticipated Milestones

OGSIVEO (nirogacestat)

Continue strong commercial execution of the OGSIVEO launch in the U.S.
Secure regulatory approval for OGSIVEO in the European Union (EU) and launch OGSIVEO following reimbursement authorization in individual EU countries, beginning with Germany in mid-2025.
Publish long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors in a peer-reviewed journal by the end of 2025.
Report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with ovarian granulosa cell tumors in the first half of 2025.
Continue to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
Mirdametinib (NF1-PN)

Secure FDA approval in adults and children with NF1-associated plexiform neurofibromas, or NF1-PN (PDUFA: February 28, 2025), and launch in the U.S.
Obtain regulatory approval in the EU for mirdametinib for the treatment of adults and children with NF1-PN and begin initial launch in 2025.
Emerging Pipeline

SpringWorks expects additional data to be presented by MapKure from the brimarafenib monotherapy trial in the second half of 2025.
Continue enrolling patients in Phase 1 trial of SW-682 in Hippo-mutant solid tumors.
File an Investigational New Drug (IND) application for SW-3431 by the end of 2025.
"2025 is set up to be another transformative year for SpringWorks. With the potential launch of our second medicine and global expansion to serve patients with two devastating diseases, we are energized by the opportunity to continue delivering on the commitments we have made to the patient communities we are dedicated to serving," said Saqib Islam, Chief Executive Officer of SpringWorks. "In parallel with our commercial launches, we are advancing our pipeline of oncology programs for heavily underserved patient populations and are confident that our strong foundation will drive our long-term growth and success."

Presentation at the 43rd Annual J.P. Morgan Healthcare Conference

SpringWorks will webcast its presentation from the 43rd Annual J.P. Morgan Healthcare Conference today, Monday, January 13, 2025 at 11:15 a.m. PT (2:15 p.m. ET). To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

On January 13, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, reported that Clay Siegall, Ph.D., President and CEO of Immunome, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 9:45 a.m. PT (Press release, Immunome, JAN 13, 2025, View Source [SID1234649693]).

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Interested parties can access the live audio webcast for this conference from the Investor Relations section of the company’s website at www.immunome.com. The webcast replay will be available after the conclusion of the live presentation for approximately 30 days.

The presentation will cover Immunome’s pipeline of targeted therapeutics, including varegacestat (formerly known as AL102), a once-daily oral gamma secretase inhibitor in the Phase 3 RINGSIDE clinical trial for the treatment of desmoid tumors. Immunome expects to report topline data in the second half of 2025.

Immunome also announced the FDA clearance of its IND submission for IM-1021, a ROR1-targeted ADC. Immunome anticipates submitting an IND for IM-3050, its FAP-targeted radioligand therapy, by the end of the first quarter of 2025.

Immunome additionally disclosed three novel ADCs for solid tumor indications that are in preclinical development: IM-1617, IM-1340 and IM-1335.

On January 13, 2025 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported the company’s key 2025 milestones ahead of its presentation at J.P. Morgan’s 43rd annual healthcare conference (Press release, Cogent Biosciences, JAN 13, 2025, View Source [SID1234649646]).

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"2025 will be a transformational year at Cogent Biosciences," said Andrew Robbins, President and Chief Executive Officer. "During the year, we plan to report top-line results from all three registration-directed bezuclastinib studies, and if successful, move forward with our first New Drug Application (NDA) submission by the end of 2025. With both SUMMIT and PEAK enrollment finishing several months ahead of schedule, we are confident that physicians and patients are highly aware of bezuclastinib’s potential and are also eagerly awaiting these clinical trial results. We believe bezuclastinib could change the lives of thousands of patients fighting SM and GIST and has the potential to be the first potent, CNS-sparing, selective KIT mutant inhibitor. In addition, we continue to advance our pipeline of novel small molecule programs, including an ongoing Phase 1 study of our novel FGFR2 inhibitor, CGT4859, and plan to file INDs for both our ErbB2 and PI3Kα programs during the year. With a strong balance sheet, we are well positioned to prepare Cogent for our evolution into a commercial-stage company."

In 2025, the Company plans to achieve the following milestones:

Bezuclastinib – Systemic Mastocytosis (SM)

Report top-line results in July 2025 from the SUMMIT trial. SUMMIT is a registration-directed, global, randomized, placebo-controlled trial of bezuclastinib in patients with Non-Advanced Systemic Mastocytosis (NonAdvSM).

Report top-line results during the second half of 2025 from the APEX trial. APEX is a registration-directed, global, open-label trial of bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM).

Submit the first bezuclastinib New Drug Application (NDA) by the end of 2025.

Bezuclastinib – Gastrointestinal Stromal Tumors (GIST)

Report top-line results by the end of 2025 from the pivotal Phase 3 PEAK trial. PEAK is a global, blinded, randomized clinical trial studying the combination of bezuclastinib and sunitinib versus sunitinib alone in patients with imatinib-resistant gastrointestinal stromal tumors (GIST).

Bezuclastinib – Expanded Access Program

During Q1 2025, initiate Expanded Access Programs (EAP) in the U.S. for SM and GIST patients to receive investigational bezuclastinib after meeting certain eligibility criteria.

CGT4859 (FGFR2 inhibitor)

Enroll patients in the ongoing Phase 1 trial with CGT4859, a reversible, selective FGFR2 inhibitor in patients with documented FGFR mutations, including advanced cholangiocarcinoma. The trial is designed to explore the safety, tolerability and clinical activity of escalating doses of CGT4859 with a goal of selecting an active and well tolerated dose for further clinical investigation.

Preclinical Pipeline

Submit an IND application for CGT4255, a potent, selective ErbB2 inhibitor, highlighted by potential best-in-class brain-penetrant properties.

Submit an IND application for CGT6297, a potent allosteric inhibitor of PI3Kα, with 25-fold selectivity over PI3Kα WT.

J.P. Morgan Presentation Details
Cogent will participate in a presentation and Q&A session at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025, beginning at 7:30 a.m. PT (10:30 a.m. ET). A live webcast will be accessible in the "Investors & Media" section of the company’s website, www.cogentbio.com, and will be archived for 30 days following the event.