Absci (NASDAQ:ABSI) used a presentation at the 44th JPMorgan Healthcare Conference to outline recent platform updates and provide a timeline for upcoming clinical data in androgenic alopecia and endometriosis. Founder and CEO Sean McClain said the company is “entering this really exciting new era” in which molecules designed using its generative AI are moving into the clinic and approaching proof-of-concept readouts.
AI platform update: Origin-1 model released
McClain said Absci released a new model, Origin-1, alongside a manuscript describing the approach. He characterized Origin-1 as focused on de novo design of antibodies for what the company calls “zero prior epitopes,” meaning epitopes that do not have a structurally defined protein-protein interface and lack known structural binders.
In the Q&A, management compared Absci’s approach to Recursion, describing both as using a wet-lab-in-the-loop framework but noting Absci’s focus is on antibody design. McClain said Origin-1 is “all protein-based or antibody-based,” but added the company sees expansion potential into bispecifics, noting that Absci already has bispecifics in its pipeline.
Development timelines and cost claims
McClain contrasted Absci’s approach with traditional drug development, saying it typically takes about five and a half years and $50 million to $100 million to get a drug into the clinic. He said Absci’s work on ABS-101 and ABS-201 demonstrates the company can reach the clinic in roughly two years with a total investment of about $15 million.
He framed cost and speed as important but not sufficient on their own, stating Absci aims to use AI to pursue diseases with high unmet need and poor standard of care, rather than “me-too therapies.”
ABS-201: prolactin receptor antibody for androgenic alopecia
McClain highlighted ABS-201, an antibody targeting the prolactin receptor, as a program aimed at both androgenic alopecia (AGA) and endometriosis. For AGA, he described the opportunity as large, stating 80 million people in the U.S. suffer from the condition and that existing options have limited efficacy and durability. He also noted that AGA studies can be “very cost-effective and fast” to run, which he said supports a path toward rapid approval.
McClain explained the proposed mechanism by stating prolactin production in the scalp is driven by a promoter independent of systemic pituitary production, leading over time to a buildup of prolactin in the scalp and activation of the STAT5 pathway. He said this process is associated with a decrease in progenitor cell lines and miniaturization of hair follicles, and that blocking the prolactin receptor can help restore the machinery needed to reverse miniaturization.
He also described preclinical work that included a stump-tailed macaque model of naturally occurring baldness. According to McClain, after 28 weeks of treatment with an anti-prolactin receptor antibody, animals went “from a bald head to a full head of hair” by around six months, with hair repigmentation. He further stated that post-treatment durability persisted for up to four years in that model.
McClain also referenced ex vivo work with human scalp biopsies in collaboration with Professor Ralf Paus. He said ABS-201 drove follicles into an active growth (anagen) state, while prolactin drove follicles into catagen, and that ABS-201 plus prolactin “rescued” follicles to remain in anagen. He also said the company observed increased progenitor cell markers (CD34 and CD200), prevention of apoptosis in K15 stem cells, increased growth factors (IGF-1 and FGF-7), and decreased catagen drivers (TGF-β), which he said supports prolactin receptor as a “master regulatory switch” for hair follicles.
Clinical trial design and upcoming readouts
Absci said it is running a Phase I/II-A study in androgenic alopecia that evaluates safety, tolerability, and efficacy. McClain said the single ascending dose (SAD) portion in healthy volunteers has started and the company has filled the first two cohorts. The company expects to complete the SAD portion in the first half of this year and then transition to a 26-week multiple ascending dose (MAD) portion evaluating safety, tolerability, and efficacy.
McClain said the trial is designed for 227 participants (male and female). He listed primary efficacy endpoints as:
- Target area hair count
- Hair width
- Hair darkness
He said the company expects a safety readout in the first half of the year and a 13-week interim efficacy readout in the second half of the year, with study completion expected early next year.
In Q&A, McClain said human ex vivo data increased confidence in translatability and highlighted the importance of achieving greater than 90% receptor occupancy, which he said was observed in the macaque study. Management also said the molecule has been engineered with a longer half-life to support dosing convenience and that PK data from SAD will help guide future development planning. The company said MAD participants are not allowed to use background finasteride or minoxidil and that there is a four-month washout period for those previously on those therapies.
Endometriosis plans and other corporate updates
For endometriosis, McClain said the condition affects 1 in 10 women and that standard of care is poor with no disease-modifying therapies currently on the market. He noted that the prolactin receptor mechanism has Phase II proof-of-concept data from the HMI-115 study, which he said de-risks the mechanism clinically. He described the rationale as local prolactin driving lesion growth and prolactin receptors on sensory neurons contributing to pain, with prolactin receptor blockade intended to address both lesion growth and pain.
McClain said Absci plans to start an endometriosis trial in Q4 of this year with a Phase II proof-of-concept readout a year later. In Q&A, management said the SAD safety readout would help de-risk both programs but suggested there may not be significant efficacy read-through from AGA to endometriosis.
McClain also provided corporate metrics, stating Absci has 140 employees, three clinical stage programs, a wet-lab-in-the-loop facility in Vancouver, and “over 10 partners.” He said the company ended the year with $143 million on the balance sheet and expects runway into the first half of 2028. The company also discussed a technology partnership with AMD, including a $20 million investment, aimed at scaling compute for protein design.
About Absci (NASDAQ:ABSI)
Absci Corporation (NASDAQ: ABSI) is a biotechnology company that applies machine learning, synthetic biology and automation to accelerate the discovery and development of protein-based therapeutics. The company’s Integrated Drug Creation® (IDC®) platform is designed to identify and produce novel antibody and enzyme candidates at speeds and scales that traditional biopharma discovery methods cannot match. Absci works with pharmaceutical and biotechnology partners to generate, screen and optimize protein molecules for a wide range of therapeutic applications.
The core of Absci’s offering is its end-to-end discovery engine, which combines proprietary algorithms, high-throughput laboratory automation and a deep learning framework.
