The target of AllRock Bio’s research is already validated by FDA-approved medicines — for the eyes. The biotech startup is expanding that science to the lungs with a drug candidate licensed from Sanofi offering the potential for a first-in-class approach to cardiopulmonary and fibrotic diseases. AllRock is now out of stealth, revealing its strategy along with $50 million for its clinical trial plans.
AllRock’s Series A financing announced Tuesday was co-led by Versant Ventures and Westlake BioPartners.
AllRock’s drug candidate, ROC-101, targets and inhibits two Rho kinases (ROCKs), enzymes that play key roles in cellular functions. Overactivity of these enzymes can lead to vascular problems. For example, improper draining of fluid from the eye leads to glaucoma. Just as ROCK inhibitors treat glaucoma by widening blood vessels to reduce intraocular pressure, this approach can also address vascular problems of the lungs, said AllRock Chief Medical Officer Bill Marshall.
The lead disease target of Natick, Massachusetts-based AllRock is pulmonary arterial hypertension (PAH), a form of high blood pressure that develops in the arteries carrying blood from the heart to the lungs. For this indication, ROCK inhibition could offer more than vasodilation, Marshall said. In PAH, arteries clog due to the buildup of inflammatory cells, formation of scar tissue that’s called fibrosis, and the proliferation of muscular cells.
“A lot of drugs that are already approved (for PAH) just address the arterial constriction,” Marshall said. “It’s unusual to find drugs like a ROCK inhibitor that has ability to address the proliferation of cells, the fibrosis, the muscularization (of blood vessels).”
AllRock is led by co-founder and CEO Catherine Pearce. She and her team bring cardiovascular drug development experience from CinCor Pharma, a company acquired by AstraZeneca two years ago in a $1.3 billion deal centered on the hypertension drug candidate baxdrostat.
Pearce’s awareness of ROC-101 goes back about a decade. The molecule was originally developed by Kadmon Holdings, which Sanofi acquired in 2021. Kadmon’s main asset was Rezurock, a ROCK2 inhibitor approved for graft-versus-host disease. The pan-ROCK inhibitor now known as ROC-101 was Kadmon’s second internally developed compound and it was one of the molecules Pearce discussed with Kadmon for a potential partnership. After CinCor was sold, she saw the opportunity to get the molecule. Pearce brought the old CinCor team together to form AllRock in 2023 and license ROC-101, which she said did not fit Sanofi’s portfolio.
“They’re not a company that does pulmonary hypertension,” Pearce said of the pharma giant. “However, the science and the preclinical package (from Kadmon) was beautifully done. So there wasn’t anything other than us picking it up from that starting point and continuing on.”
AllRock has completed a Phase 1 test of the oral small molecule in healthy volunteers. Results are set to be revealed later this month during the European Respiratory Society Congress in Amsterdam. Details about the pharmacokinetics, safety, and target engagement of ROC-101 remain under embargo, but Marshall said he can say the drug was safe and well tolerated. He added that the key concern was inhibition of both ROCK1 and ROCK2 could causes hypotension, or low blood pressure. Marshall said there was no evidence of hypotension in the 90-patient study.
Standard PAH therapies include old vasodilators as well as drugs that inhibit excessive growth of cells in the blood vessels of the lungs. Last year, FDA approval of Merck’s sotatercept, brand name Winrevair, introduced a new class of PAH medicines. Subcutaneously injected Winrevair is a fusion protein engineered to trap a protein whose elevated levels are associated with proliferation of cells that thicken pulmonary vessels. The drug, which came from Merck’s $11.5 billion acquisition of Acceleron Pharma in 2021, is projected to become a blockbuster seller.
AllRock does not aim to replace existing PAH drugs, including Winrevair, Pearce said. ROC-101 could complement those products, giving patients additional benefit beyond available PAH therapies. The 24-week Phase 2 trial is designed with an arm to test standard drugs plus ROC-101 and another arm testing standard of care and Winrevair plus ROC-101. Marshall said the trial will enroll patients who, despite taking currently available drugs, still have high pulmonary pressures.
In addition to enrolling PAH patients, the Phase 2 test will recruit those with pulmonary hypertension with interstitial lung disease (PH-ILD), a type of high blood pressure in the lungs due to interstitial lung disease, a progressive lung disorder. United Therapeutics’ inhaled vasodilator Tyvasso, which was initially approved for PAH, has become a standard treatment for PH-ILD. Roivant Sciences subsidiary Pulmovant is pursuing this indication with an inhalable drug candidate licensed from Bayer. Pearce said AllRock can stand apart with a pill formulation.
Bringing pan-ROCK inhibition to arterial disorders has precedent. The vasodilative effects of the decades-old pan-ROCK inhibitor fasudil lowers blood pressure to treat cerebral vasospasm, a narrowing of brain arteries, Marshall said. This intravenously administered drug is only approved in Japan and China, but off-label use of the product has shown efficacy in PAH. Additional evidence from investigator-initiated clinical testing showed better results than approved PAH drugs, Marshall said.
Fasudil’s data from Asia provide clinical validation of pan-ROCK inhibition, which made the hypothesis that those results would translate to ROC-101 clear and derisked, Alica Levey, venture partner at Versant, said in an email. Versant’s investment was also a wager on the AllRock team.
“At CinCor, they had tremendous success identifying a diamond-in-the-rough asset and efficiently generating the data that drove pharma interest and ultimately an acquisition,” Levey said. “We are using the same strategy here and are betting on a similar outcome.”
CinCor came from CinRx, a biotech incubator. Pearce and her team are taking a similar approach with AllRock, which is the first startup from JucaBio, a biopharmaceutical company that scouts for promising drug assets and forms subsidiaries to advance them. Roivant and BridgeBio Pharma have similar “hub-and-spoke” business models. Pearce, who is also CEO of JucaBio, said ROC-101 was the first asset the company was able to acquire. Under AllRock, it has progressed from preclinical stage to Phase 2-ready in 16 months. Pearce said she hopes this drug is just the first of several for JucaBio.
“We’ve been efficient with our capital, and we’d like to continue to do that on repeat,” Pearce said.
Image: Jackie Niam, Getty Images
