CPS1

Boston-area biotechs Upstream Bio and CAMP4 Therapeutics are headed to the Nasdaq on Friday, the latest companies to IPO in what appears to be a recovery for startups trying to raise capital on the public market. Upstream Bio priced at $17 per share, the high end of the $15 to $17 range it set earlier this week . By selling 15 million shares — 2.5 million more than originally planned — it will raise $255 million in new cash. CAMP4, meanwhile, priced its shares $CAMP at $11 apiece, $3 below the low end of the $14 to $16 range it marketed to investors. It sold 6.82 million shares versus an initial plan to offer 5 million and will raise $75 million . The IPO window is “showing signs of recovery,” Oppenheimer bankers wrote in a quarterly analysis this week. GPCR biotech Septerna is also in the IPO queue, and Bain-backed obesity startup Rivus Pharmaceuticals is also considering a listing this fall, Bloomberg reported this week. For Upstream $UPB the offering will add to its coffers as it runs Phase 2 and later-stage trials of its TSLP receptor antagonist verekitug. The experimental treatment, licensed from Astellas, is being studied for severe asthma and chronic rhinosinusitis with nasal polyps . Upstream hopes to enter a similar market as Amgen and AstraZeneca’s Tezspire, as well as Blackstone-backed Uniquity Bio’s solrikitug . CAMP4 is working on regulatory RNA and has a Phase 1 trial of its lead drug, a CPS1-targeting medicine for urea cycle disorders. The IPO comes a few weeks after CAMP4 added BioMarin to its partnership lineup, which already includes Fulcrum Therapeutics and Eli Lilly. Earlier in its startup days, CAMP4 had pacts with Biogen and Alnylam.

Regulatory RNA startup CAMP4 Therapeutics has inked another collaboration deal as it prepares for an initial public offering. The Cambridge, MA-based startup has lined up a pact with BioMarin to create RNA-targeting antisense oligonucleotides, or ASOs, the companies said Tuesday morning. The deal comes with just $1 million upfront, but as much as $370 million in potential future payments if BioMarin goes all the way through commercialization with two programs, according to an earlier SEC filing . For BioMarin, it lets the company put a small bet on new technology after recently trimming its pipeline , cutting workers and changing leadership . CAMP4 unveiled its IPO ambitions on Sept. 20, and it could price and start trading within the next few weeks. The startup, which emerged in 2018, had tie-ups with Alnylam and Biogen in its early days, but those agreements were scrapped as CAMP4 shifted from a focus on bioinformatics to restoring gene expression through regulatory RNAs. It has since forged a license and discovery agreement with Fulcrum Therapeutics and an R&D pact for up to three ASOs with Eli Lilly. The company is currently in Phase 1 with a CPS1-targeting experimental drug for urea cycle disorders. It expects initial data in the first quarter of next year. The experimental medicine, dubbed CMP-CPS-001, has orphan drug and rare pediatric disease tags from the FDA. The 64-employee biotech is in the IPO queue alongside respiratory biotech Upstream Bio . If they follow through with their Nasdaq listings, they’d tack onto a relatively busy fall of debuts from Bicara, Zenas, MBX and BioAge .

CAMBRIDGE, Mass., Sept. 17, 2024 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics, a clinical-stage biotechnology company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels across a range of genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CMP-CPS-001 for the treatment of urea cycle disorders (UCDs). “Today, there are no disease-modifying therapies available for patients with UCDs, and this community is in desperate need of innovative approaches to help mitigate potentially devastating health outcomes, including neurologic disability, seizures, and death,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “Based on our preclinical studies, we believe CMP-CPS-001 has the potential to shift the paradigm in how patients with UCDs are treated. Our preclinical studies have demonstrated that by modulating the activity of the target regRNA, CMP-CPS-001 increases the levels of a key enzyme in the urea cycle, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges.” About Orphan Drug DesignationOrphan Drug Designation is granted by the FDA and is designed to support the development of new therapeutics for rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies companies for certain incentives, which may include tax credits for qualified clinical trials, exemption from FDA user fees, and potential eligibility for seven years of market exclusivity in the United States upon FDA approval. Drugs for rare diseases are held to the same rigorous scientific review processes as any other drug for approval or licensing. About CMP-CPS-001CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate for the treatment of UCDs targeting carbamoyl phosphate synthetase 1 (CPS1), an enzyme that catalyzes the first step of the urea cycle, which converts ammonia to urea. CMP-CPS-001 is designed to upregulate CPS1 gene expression by binding to a CPS1-specific regulatory RNA sequence to ultimately increase CPS1 protein levels. CMP-CPS-001 is currently under evaluation in a Phase 1 clinical trial in healthy volunteers (NCT06247670). CMP-CPS-001 has been granted Rare Pediatric Disease Designation by the FDA. About CAMP4 TherapeuticsCAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic ASO drug candidates target regulatory RNAs (regRNAs), which act locally on transcription factors and are the master regulators of gene expression. CAMP4’s proprietary RAP Platform™ enables the mapping of regRNAs and generation of therapeutic candidates designed target the regRNAs of haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. Learn more about us at www.CAMP4tx.com and follow us on LinkedIn and X. Contacts Media:Jason Braco, Ph.D. LifeSci Communicationsjbraco@lifescicomms.com(646) 876-4932