C4 Therapeutics’ myeloma drug shows 50% response rate in trial

Published 20/09/2025, 16:08
C4 Therapeutics’ myeloma drug shows 50% response rate in trial

WATERTOWN, Mass. - C4 Therapeutics, Inc. (NASDAQ:CCCC), a clinical-stage biopharmaceutical company with a market capitalization of $225 million, reported Saturday that its investigational oral drug cemsidomide, when combined with dexamethasone, achieved a 50% overall response rate at the highest dose level in patients with relapsed/refractory multiple myeloma. The announcement has contributed to the stock’s impressive 19% gain over the past week, according to InvestingPro data.

The Phase 1 clinical trial results showed the drug achieved a 50% response rate at the 100 μg dose and 40% at the 75 μg dose in heavily pre-treated patients who had received a median of seven prior therapies. The median duration of response was 9.3 months across all dose levels, with the two highest doses not yet reaching median duration. While the company maintains a strong liquidity position with a current ratio of 5.06 and more cash than debt on its balance sheet, InvestingPro analysis indicates the company is rapidly burning through its cash reserves.

The study enrolled 72 patients, with 75% having previously received BCMA-targeted therapy and 75% having prior CAR-T or T-cell engager therapy. According to the company, cemsidomide demonstrated a favorable safety profile with no treatment discontinuations related to the drug and minimal dose reductions.

"Cemsidomide’s clinical trial results to date have shown compelling anti-myeloma activity, a differentiated safety and tolerability profile and immunomodulatory effects across all dose levels," said Len Reyno, chief medical officer of C4 Therapeutics, in the press release.

The company plans to pursue two distinct development paths that could lead to accelerated approval. A Phase 2 registrational trial evaluating cemsidomide with dexamethasone is expected to begin in Q1 2026, with initial data anticipated in the second half of 2027. Additionally, a Phase 1b trial studying cemsidomide and dexamethasone in combination with a BCMA BiTE is planned for Q2 2026.

The drug works by degrading IKZF1/3 proteins, which are targeted by established multiple myeloma treatments. Multiple myeloma affects approximately 36,000 people annually in the United States and remains incurable despite treatment advances.

C4 Therapeutics hosted a webcast for investors and analysts on Saturday to discuss the findings. Analyst price targets for the company range from $5 to $50 per share, reflecting diverse views on the company’s potential. For deeper insights into C4 Therapeutics’ financial health and growth prospects, including 12 additional exclusive ProTips, visit InvestingPro.

In other recent news, C4 Therapeutics has been the focus of several analyst updates and company developments. Barclays has reiterated its Overweight rating on C4 Therapeutics, maintaining a price target of $8.00 per share, reflecting a positive outlook on the company’s specialization in targeted protein degradation therapeutics. Additionally, Stephens upgraded the company’s stock from Equal Weight to Overweight, setting a price target of $6.00, citing recent progress and changes in the multiple myeloma treatment landscape. Guggenheim also initiated coverage on C4 Therapeutics with a Buy rating and a price target of $8.00, highlighting the potential of C4’s oral drug, cemsidomide, for multiple myeloma. C4 Therapeutics plans to host a webcast to discuss data from its Phase 1 dose escalation trial of cemsidomide in multiple myeloma patients. The webcast will follow an oral presentation by Dr. Binod Dhakal at the International Myeloma Society Annual Meeting. These developments indicate growing interest and potential in C4 Therapeutics’ therapeutic offerings and market strategies.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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