On January 14, 2025 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported its anticipated 2025 milestones as it continues its evolution into becoming a fully integrated biotechnology company focused on orally bioavailable degraders (Press release, C4 Therapeutics, JAN 14, 2025, View Source [SID1234649701]).
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"Stellar execution in 2024 has set up 2025 to be a pivotal year for the company as we work to generate important data that will position us to advance programs and bring degrader medicines to patients searching for new therapeutic options," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "The cemsidomide data presented at the ASH (Free ASH Whitepaper) Annual Meeting in December support a potentially best-in-class profile and we are preparing for the next phase of development of this molecule. We continue to progress the CFT1946 Phase 1/2 study and will leverage data from the tumor specific cohorts to determine the development path for this program. In addition, we expect data from the CFT8919 Phase 1 dose escalation study run by our partner Betta Pharmaceuticals in China, which will define its potential for non-small cell lung cancer patients with the EGFR L858R mutation. We are excited about the degrader rationale for these programs, which we believe have the potential to deliver value for patients, caregivers, physicians and shareholders."
ANTICIPATED 2025 MILESTONES
Cemsidomide: Cemsidomide is an oral degrader of IKZF1/3 for the potential treatment of relapsed/refractory (R/R) multiple myeloma (MM) and R/R non-Hodgkin’s lymphoma (NHL).
Multiple Myeloma
Enable initiation of the next phase of clinical development to investigate cemsidomide in combination with dexamethasone in the late-line setting, and in combination with other MM agents for earlier lines of treatment. These new studies are expected to initiate in early 2026.
Complete Phase 1 dose escalation and present data in the second half of 2025.
Non-Hodgkin’s Lymphoma
Complete Phase 1 dose escalation and present data in the second half of 2025.
Open expansion cohort(s) of the current Phase 1/2 trial in patients with peripheral T-cell lymphoma (PTCL) in the second half of 2025.
Enable initiation of the next phase of clinical development to investigate cemsidomide monotherapy in later lines of therapy in PTCL. This new study is expected to initiate in early 2026.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600 mutations for the potential treatment of solid tumors including colorectal cancer (CRC), melanoma and other malignancies with V600 mutations.
Complete monotherapy Phase 1 dose escalation in BRAF V600 mutant solid tumors in the first half of 2025.
Generate data from the Phase 1 cohorts exploring monotherapy CFT1946 in melanoma, CFT1946 in combination with cetuximab in CRC and CFT1946 in combination with trametinib in melanoma. Data from these cohorts will define and enable the next phase of development.
Present Phase 1 data in the second half of 2025. These presentations will include: (1) monotherapy in BRAF V600 mutant solid tumors, (2) monotherapy expansion cohorts in melanoma, and (3) in combination with cetuximab in CRC.
CFT8919: CFT8919 is an oral degrader targeting EGFR bearing an oncogenic L858R mutation for the potential treatment of non-small cell lung cancer (NSCLC).
Evaluate data from the Phase 1 dose escalation study in Greater China, which is led by partner Betta Pharmaceuticals, in patients with locally or advanced metastatic NSCLC harboring an EGFR L858R mutation. These data will be used to determine the next phase of development.
Discovery: C4T will continue to utilize its TORPEDO platform to develop orally bioavailable degraders for oncology and non-oncology targets for internal research and collaboration programs. To further highlight its deep expertise in drug discovery, C4T plans to:
Present and publish preclinical work from its internal pipeline and TORPEDO platform.
Advance internal and collaboration programs to key milestones.
2024 ACCOMPLISHMENTS
Cemsidomide: C4T advanced the Phase 1/2 clinical trial and delivered data reinforcing the potential of cemsidomide to become a backbone therapy of choice in MM and NHL where IKZF1/3 degradation is warranted.
Multiple Myeloma
At ASH (Free ASH Whitepaper), presented data on cemsidomide in combination with dexamethasone. As of the data cutoff date of October 11, 2024, the dose level exploring 75 µg once daily (QD) achieved an overall response rate (ORR) of 36 percent. Cemsidomide was well-tolerated across all dose levels.
The maximum tolerated dose has not yet been reached. Patients are enrolling in the 100 µg QD cohort.
Non-Hodgkin’s Lymphoma
At ASH (Free ASH Whitepaper), presented data on cemsidomide monotherapy. As of the data cutoff date of October 11, 2024, cemsidomide demonstrated a 38 percent ORR across all subtypes and doses studied. In PTCL, cemsidomide achieved a 44 percent ORR and a 25 percent complete metabolic response rate.
The maximum tolerated dose has not yet been reached. Patients are enrolling in the 75 µg QD cohort.
CFT1946: C4T advanced the Phase 1/2 clinical trial across multiple arms and delivered monotherapy data demonstrating proof of mechanism and early evidence of proof of concept.
At the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, presented monotherapy data demonstrating CFT1946 is well tolerated across all dose levels and demonstrates initial signs of anti-tumor activity across all dose levels.
At the TPD Summit, presented new preclinical data demonstrating CFT1946 has the ability to cross the blood-brain barrier, with Kpu,u values in the range of 0.34 to 0.88.
Progressed the Phase 1 monotherapy dose escalation study. Began enrolling patients across multiple exploratory cohorts: CFT1946 monotherapy in melanoma, CFT1946 in combination with trametinib in melanoma, and CFT1946 in combination with cetuximab in CRC.
CFT8919: Betta Pharmaceuticals, with C4T support, initiated the Phase 1 clinical trial of CFT8919 in Greater China.
Discovery: C4T further validated its TORPEDO platform and advanced key research efforts.
Delivered two development candidates for non-oncology targets to collaborator Biogen.
Established a new collaboration with Merck KGaA, Darmstadt, Germany focused on two critical oncogenic proteins.
Continued to progress its internal discovery portfolio of orally bioavailable degraders.
Corporate: C4T further strengthened its leadership across its management team and Board of Directors to supports its evolution into a fully integrated biotechnology company.
Paige Mahaney, Ph.D., was appointed chief scientific officer. Dr. Mahaney is an experienced drug developer who has helped leading pharmaceutical companies build clinical portfolios across a wide range of disease indications and treatment modalities.
C4T continued to evolve its governance by appointing three new members to its Board of Directors who bring deep experience across drug discovery, commercialization and lifecycle management.
Cash Guidance
C4T expects that its cash, cash equivalents and marketable securities as of December 31, 2024, together with anticipated collaboration expense reimbursements, but excluding any collaboration option or milestone payments, will enable the company to fund its operating plan into 2027.
JP Morgan Presentation
C4T will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025 at 2:15 pm PST (5:15 pm EST). A live webcast will be available under "Events & Presentations" in the Investors section of the company’s website at www.c4therapeutics.com. A replay of the webcast will be archived on the C4T website for at least two weeks following the presentation.