Recursion Pharmaceuticals Advances First AI-Discovered Neuroscience Target with Genentech, Lowers Cash Burn Guidance
核心洞察
Genentech exercised the first validated target option under its neuroscience collaboration with Recursion, advancing a previously unexplored target identified via AI-powered whole-genome CRISPR screening of over 1 trillion iPSC-derived neuronal cells.
REC-4881, an oral MEK1/2 (搜索) inhibitor for familial adenomatous polyposis (搜索), demonstrated a median 43% reduction in polyp burden at three months in the Phase 2 TUPELO trial, with additional data expected at CGA-IGC in November 2026.
The FDA cleared the IND for REC-7735 (搜索), a mutant-selective PI3Kα (搜索) H1047R inhibitor with >100-fold selectivity over wild-type, with the Phase 1/2 ZINNIA trial set to begin in the second half of 2026.
Recursion Pharmaceuticals has reached what CEO Najat Khan, Ph.D., described as a "pivotal point" in the company's evolution, as its AI-native drug discovery platform delivered a novel neuroscience target that partner Genentech has now advanced into a joint early discovery program. The milestone, announced alongside second-quarter 2026 financial results, provides early evidence that Recursion's approach can identify and experimentally validate previously unexplored biology in one of medicine's most challenging therapeutic areas.
"The advancement of the first unexplored neuroscience target from our collaboration with Roche and Genentech into an early discovery program is an important proof point," Khan said. "Finding new targets in neuroscience has historically been challenging, and this milestone highlights our ability to uncover novel biology in areas where conventional approaches have struggled."
Genentech Collaboration Validates AI Platform
The neuroscience target was identified through a collaboration that leveraged Recursion's proprietary platform to build the first whole-genome CRISPR knockout map from a subset of over 1 trillion internally manufactured iPSC-derived neuronal cells. The company also generated hundreds of billions of microglial cells, with its models evaluating biological signatures across more than 17,000 genes and tens of millions of data points.
Candidate targets progressed through successive stages of pathway validation, functional validation, and disease validation, with only targets demonstrating compelling evidence across each stage advancing into a validation package. Next steps include small molecule design, hit generation, and validation using Recursion's AI-native chemistry platform.
To date, Recursion has achieved $216 million in upfront and milestone payments from the Roche and Genentech collaboration. The agreement encompasses up to 40 potential small molecule discovery programs, each carrying the potential for more than $300 million in development, commercialization, and net sales milestones, plus tiered royalties up to high single digits.
REC-4881 Shows Promise in FAP
Chief Medical Officer Vicki Goodman highlighted progress with REC-4881, an oral MEK1/2 (搜索) inhibitor being developed for familial adenomatous polyposis (搜索) (FAP), a disease that causes patients to develop hundreds or thousands of polyps in the gastrointestinal tract and often requires colectomy.
In the ongoing Phase 2 TUPELO study, patients receiving REC-4881 after colectomy showed a median 43% reduction in polyp burden after three months of treatment. Reductions were sustained after three months off treatment and were observed in both the upper and lower gastrointestinal tract. The safety profile was characterized as manageable, with predominantly mild-to-moderate adverse events consistent with other MEK inhibitors.
Goodman noted there are no approved systemic therapies to alter the course of FAP in post-colectomy patients. Recursion estimates more than 50,000 diagnosed patients across the U.S. and EU5, representing a potential addressable market exceeding $10 billion. REC-4881 has received both Orphan Drug Designation and Fast Track Designation from the FDA.
The company is continuing enrollment in TUPELO, including a dose-optimization cohort, and additional Phase 2 safety and efficacy data contextualized with real-world registry data will be presented at the Presidential Plenary session at the CGA-IGC Annual Meeting on November 2, 2026. Regulatory discussions with the FDA were initiated in the first half of 2026, with an update on the registrational path expected in the second half of the year.
REC-7735 (搜索) IND Cleared
Recursion also announced FDA clearance of its investigational new drug application for REC-7735 (搜索), a precision-designed PI3Kα (搜索) inhibitor targeting cancers with the PIK3CA H1047R (搜索) mutation. The company intends to initiate the Phase 1/2 ZINNIA trial in the second half of 2026.
Goodman said REC-7735 (搜索) was designed to be more than 100-fold selective for the H1047R mutation relative to wild-type PI3Kα (搜索). Existing PI3K inhibitors can inhibit wild-type PI3K and cause hyperglycemia, which can limit dosing and potentially lead to hyperinsulinemia that reactivates the PI3K signaling pathway.
The development candidate was delivered in 10 months following the synthesis of 242 compounds across 13 design cycles. Recursion said its platform identified a previously unpublished binding pocket and found no identified off-target liabilities in preclinical work. The initial dose-escalation portion of ZINNIA will enroll patients with PIK3CA H1047R (搜索)-mutant solid tumors, with subsequent dose optimization in ER-positive, HER2-negative breast cancer. First dose-escalation data are expected in the first half of 2028.
AI Platform and Financial Position
Recursion reported it has generated and aggregated more than 50 petabytes of multimodal biological data. The company's chemistry platform has advanced candidates using roughly 330 compounds over approximately 18 months, compared with an industry benchmark of roughly 2,500 compounds over four years for small-molecule discovery.
The company also detailed deployment of AI agents across biology, chemistry, and clinical development. A target-discovery tool enables scientists to interrogate biological maps in hours rather than weeks, while a chemistry design agent has reduced structural-analysis work from roughly four hours to about 30 minutes. In clinical development, agent-supported enrollment strategies have contributed to a 1.3 to 1.6-fold increase in enrollment rates versus historical benchmarks.
Recursion named Dr. Hoifung Poon, formerly of Microsoft, as chief AI officer, and Dr. Donovan Chin as senior vice president of drug design.
CFO Ben Taylor said the company lowered its 2026 full-year cash operating expense guidance to below $375 million, a $15 million reduction from prior guidance and representing a nearly 40% reduction from comparable 2024 pro forma expenses. Recursion ended the quarter with approximately $557 million in cash and equivalents, providing operating runway through early 2028.
Net loss for the second quarter of 2026 was $131.0 million, compared to $171.9 million for the same period in 2025. Research and development expenses decreased to $89.6 million from $128.6 million, primarily due to lower platform costs and improved operating efficiency.