Andrew Hirsch is wasting no time at his new role running C4 Therapeutics, and, just days after getting the top job, the biotech is gunning for an initial public offering.
The Watertown, Massachusetts-based preclinical biotech has been pretty secretive on the specifics of its programs, but its filing with the Securities and Exchange Commission as it preps for a potential IPO has seen it open up.
One of its leading product candidates is CFT7455, an orally bioavailable degrader targeting IKZF1/3 for multiple myeloma (MM), peripheral T-cell lymphoma (PTCL) and mantle cell lymphoma (MCL).
The biotech is planning an IND with the FDA in the fourth quarter and expects to kick-start a phase 1/2 test next year, according to its S-1 filing.
“We believe CFT7455 could eventually replace therapies based in the class of molecules known as IMiDs as the standard of care in multiple indications, including MM. IMiD therapies have been estimated to represent worldwide sales of approximately $15 billion in 2020, including MM as well as MCL, marginal zone lymphona [sic], and follicular lymphona [sic],” said C4 in its filing.
This test will run as an open-label dose escalation study of CFT7455 in around 18 to 30 subjects with MM or NHL. The trial will focus on safety and tolerability of CFT7455, and key secondary endpoints will be to characterize CFT7455’s PK/PD profile and anti-tumor activity.
“We expect the results from this clinical trial will help us better understand the disease characteristics of those patients who may derive benefit from CFT7455, which will enable us to more effectively design future clinical trials for this product candidate.”
It’s also working on another early candidate, CFT8634, an orally bioavailable degrader of a protein target called BRD9, for synovial sarcoma and SMARCB1-deleted solid tumors. An IND for this is slated for the second half of 2021 with a clinical trial projected by the end of 2021.
“We expect to design our first-in-human phase 1/2 clinical trial for this product candidate to be an open-label dose escalation/expansion study in both synovial sarcoma and solid tumors with SMARCB1 loss,” the company said.
Further back in the pipeline, it is also developing degraders specifically targeting V600E mutant BRAF to treat melanoma, non-small cell lung cancer, colorectal cancer and other solid malignancies that harbor this mutation, as well as degraders targeting RET to treat lung cancer, sporadic medullary thyroid cancers and other solid malignancies that harbor oncogenic RET lesions.
“We expect to have our lead product candidates, CFT7455 and CFT8634, in the clinic by the end of 2021, and product candidates from our two other lead programs, BRAF V600E and RET, in the clinic by the end of 2022,” it added.
The biotech will also assess whether its platform can help against certain neurodegenerative disorders, after engineering degraders in preclinical models that “have successfully achieved blood-brain barrier penetration,” a key step in developing drugs in this tough to treat area.
C4’s so-called TORPEDO (Target ORiented ProtEin Degrader Optimizer) platform aims to develop small-molecule treatments that harness the body’s naturally occurring pathways for dismantling and eliminating proteins within the cell.
By tagging disease-causing proteins for demolition by the cell’s own proteasomes, the company aims to reach classes of targets that have been difficult to reach with traditional means.
The former Fierce 15 winner has formed collaborations with Roche, Calico and most recently Biogen—with a deal inked last January totaling up to $415 million for research into neurodegenerative diseases such as Alzheimer’s and Parkinson’s.
Last year, it hired Adam Crystal, M.D., Ph.D., a senior director at the Novartis Institutes for BioMedical Research, to be its new chief medical officer and help carry its targeted protein degraders closer to the clinic.
And, three months back, it nabbed $150 million in a series B equity round co-led by existing investor Cobro Ventures and new investor Perceptive Advisors, while also getting $20 million in venture debt from Perceptive Advisors.
Just this week, it hired former Bind Therapeutics CEO Hirsch as its new chief, who is clearly wasting no time in moving the biotech into its next phase of becoming a publicly traded, clinical biotech.
C4 follows a long line of early-stage biotechs marching toward the public markets this year, fueled by COVID-19 woes that have hit every other industry but kept life science companies largely in the green. Many have started off asking for $100 million but gone on to raise much more.
In one of the more interesting and clever ticker symbols, C4 is planning to list on the Nasdaq under the symbol "CCCC."