Absci, which engineers biologic drugs based on its own generative AI platform, has advanced its lead candidate into the clinic by dosing the first healthy volunteers. The trial is designed to assess the antibody’s ability to treat inflammatory bowel disease (IBD) by targeting tumor necrosis factor-like cytokine 1A (TL1A).
The randomized, placebo-controlled Phase I trial is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending doses of ABS-101 in healthy volunteers. The study is expected to enroll approximately 40 healthy adults and read out interim data in the second half of this year.
“We see this as a really exciting milestone for the company,” Sean McClain, Absci’s founder and CEO, told GEN Edge. “We’re really excited about being able to use both our de novo design model as well as our lead optimization model to design this molecule very rapidly, in a very cost-effective manner, and then being able to generate a really exciting TPP [target product profile] for IBD.”
The interim data is expected to shed light on three key measures, McClain explained.
“First, we’re looking at target engagement. We want to replicate the target engagement that we saw in NHPs [non-human primates], which is a very, very favorable profile. We want to show extended half-life. If we’re able to achieve greater than once-quarterly dosing, that would be the ideal scenario. And then, third would be looking at the immunogenicity profile and showing a low ADA [anti-drug antibody] response.”
McClain discussed ABS-101 and Absci’s pipeline in an interview that included Christian Stegmann, PhD, MBA, the company’s senior vice president of drug creation. Stegmann said Absci is conducting the Phase I trial (ACTRN12625000212459p) in Australia “for a number of reasons—first and foremost, because it allows us to be very quick.” Australia’s Clinical Trial Notification (CTN) and Clinical Trial Approval (CTA) processes allow companies to begin studies within weeks rather than months.
Over time, Stegmann said, Absci plans to disclose its full clinical development strategy, and expand it beyond Australia.
“We will expand this project into a global setting. As we expand the scope [this] means there will be U.S. sites. There will be sites across the globe,” Stegmann added.
Absci says its advance of ABS-101 into clinical trials shows that the candidate is delivering on the two key promises of AI, by delivering dramatically faster and less costly drug development.
“From starting on the program to getting into the clinic, we are a little over 24 months, and normally it takes five-plus years. We’ve shaved the time by more than half,” McClain said. “In terms of overall cost, large pharmas can spend anywhere from $50 million to $100 million to get an asset into the clinic, and we were able to get this asset into the clinic for roughly $15 million dollars. So, a dramatic saving on overall cost as well.”
Absci is among numerous drug developers looking to bring new therapies to market for IBD, an umbrella term for a group of conditions that cause swelling and inflammation of the tissues in the digestive tract. IBD or its conditions like Crohn’s disease (CD) and ulcerative colitis (UC) are among indications for three of last year’s top-10 best-selling drugs—AbbVie’s Humira® (adalimumab) and Skyrizi® (Risankizumab), as well as Johnson & Johnson’s Stelara® (ustekinumab). Humira lost U.S. patent exclusivity in 2023 and Stelara did likewise earlier this year, though Skyrizi’s U.S. composition of matter patents remain protected until 2033.
Among companies with IBD drugs in the clinic, Sanofi and Teva Pharmaceuticals in February presented positive data from the RELIEVE UCCD Phase IIb trial (NCT05499130) evaluating the companies’ candidate duvakitug (TEV’574/SAR447189) as a treatment for the two most common forms of IBD, moderate-to-severe UC and CD.
In the trial’s UC cohort, 36% of patients treated with the 450 mg dose of duvakitug, and 48% with the 900 mg dose achieved the study’s primary endpoint of clinical remission at week 14. The study’s placebo-adjusted rates were 16% (450 mg) and 27% (900 mg).
Also among companies with IBD drugs in the clinic is another AI-based drug developer, Insilico Medicine.
Insilico has applied AI to design ISM5411, a small molecule candidate that it says plans to stand out as the first to treat IBD by inhibiting the prolyl hydroxylase domain (PHD). ISM541 is an oral, PHD-specific inhibitor designed to treat IBD by inducing the expression of gut barrier protective genes. ISM5411 is designed to directly regulate the intestinal immune microenvironment, so that the drug simultaneously reduces the gut inflammation seen in IBD while also promoting repair of the intestinal mucosal barrier.
In a paper published last December in Nature Biotechnology, Insilico founder and CEO Alex Zhavoronkov, PhD, and 22 co-authors based at the company shared how the company used its Chemistry42 generative chemistry engine to design and optimize novel gut-restricted inhibitors of PHD1 and PHD2, protein targets implicated in biological processes that include aging and regeneration.
“PHD is not a super novel target but it is novel for IBD. Our company wanted to go into IBD as it is a very large patient population and unmet medical need,” Zhavoronkov told GEN Edge soon after the paper was published. “Most other companies focus on the inflammatory cytokine pathways, immune cell trafficking mechanisms, or intracellular signaling nodes like JAK. Most of these just treat or ameliorate the symptoms. We wanted to produce a lasting disease-modifying effect.”
“The more drugs we have towards chronic conditions such as IBD which have a high amount of medical need, the better. The more folks that are looking to leverage AI in IBD and other I&I [immunology and inflammation] conditions, I think the more beneficial it ultimately is for patients,” says McClain.
ABS-101 is one of four disclosed programs in Absci’s pipeline. Also in that pipeline are:
ABS-201 is expected to begin Phase I trials in early 2026, McClain said.
With Absci stepping up clinical development of its pipeline candidates, the company at deadline was seeking a Head of Clinical Operations who will oversee the planning and execution of clinical development programs.
Absci announced the dosing of its first clinical trial participants with ABS-101 on May 13, the same day it reported first quarter results. The company saw its Q1 net loss climb nearly 20% year-over-year, to $26.346 million from its $21.975 million net loss in the first three months of 2024. The company’s quarterly revenue, all derived from partner programs, rose 31% to $1.179 million in Q1 2025 from $898,000 a year earlier.
Absci finished the first quarter with $133.983 million in cash, cash equivalents, and short-term investments as of March 31, a sum the company says is enough of a “runway” to fund its operations into the first half of 2027. That cash position is up 19% from $112.425 million as of December 31, 2024—but down 17% from the $161.543 million the company reported March 31, 2024.
The post Absci Advances Lead AI-Designed Candidate for IBD into the Clinic appeared first on GEN - Genetic Engineering and Biotechnology News.
The randomized, placebo-controlled Phase I trial is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending doses of ABS-101 in healthy volunteers. The study is expected to enroll approximately 40 healthy adults and read out interim data in the second half of this year.
“We see this as a really exciting milestone for the company,” Sean McClain, Absci’s founder and CEO, told GEN Edge. “We’re really excited about being able to use both our de novo design model as well as our lead optimization model to design this molecule very rapidly, in a very cost-effective manner, and then being able to generate a really exciting TPP [target product profile] for IBD.”
The interim data is expected to shed light on three key measures, McClain explained.
“First, we’re looking at target engagement. We want to replicate the target engagement that we saw in NHPs [non-human primates], which is a very, very favorable profile. We want to show extended half-life. If we’re able to achieve greater than once-quarterly dosing, that would be the ideal scenario. And then, third would be looking at the immunogenicity profile and showing a low ADA [anti-drug antibody] response.”
McClain discussed ABS-101 and Absci’s pipeline in an interview that included Christian Stegmann, PhD, MBA, the company’s senior vice president of drug creation. Stegmann said Absci is conducting the Phase I trial (ACTRN12625000212459p) in Australia “for a number of reasons—first and foremost, because it allows us to be very quick.” Australia’s Clinical Trial Notification (CTN) and Clinical Trial Approval (CTA) processes allow companies to begin studies within weeks rather than months.
Beyond Australia
Over time, Stegmann said, Absci plans to disclose its full clinical development strategy, and expand it beyond Australia.
“We will expand this project into a global setting. As we expand the scope [this] means there will be U.S. sites. There will be sites across the globe,” Stegmann added.
Absci says its advance of ABS-101 into clinical trials shows that the candidate is delivering on the two key promises of AI, by delivering dramatically faster and less costly drug development.
“From starting on the program to getting into the clinic, we are a little over 24 months, and normally it takes five-plus years. We’ve shaved the time by more than half,” McClain said. “In terms of overall cost, large pharmas can spend anywhere from $50 million to $100 million to get an asset into the clinic, and we were able to get this asset into the clinic for roughly $15 million dollars. So, a dramatic saving on overall cost as well.”
Absci is among numerous drug developers looking to bring new therapies to market for IBD, an umbrella term for a group of conditions that cause swelling and inflammation of the tissues in the digestive tract. IBD or its conditions like Crohn’s disease (CD) and ulcerative colitis (UC) are among indications for three of last year’s top-10 best-selling drugs—AbbVie’s Humira® (adalimumab) and Skyrizi® (Risankizumab), as well as Johnson & Johnson’s Stelara® (ustekinumab). Humira lost U.S. patent exclusivity in 2023 and Stelara did likewise earlier this year, though Skyrizi’s U.S. composition of matter patents remain protected until 2033.
Among companies with IBD drugs in the clinic, Sanofi and Teva Pharmaceuticals in February presented positive data from the RELIEVE UCCD Phase IIb trial (NCT05499130) evaluating the companies’ candidate duvakitug (TEV’574/SAR447189) as a treatment for the two most common forms of IBD, moderate-to-severe UC and CD.
In the trial’s UC cohort, 36% of patients treated with the 450 mg dose of duvakitug, and 48% with the 900 mg dose achieved the study’s primary endpoint of clinical remission at week 14. The study’s placebo-adjusted rates were 16% (450 mg) and 27% (900 mg).
Standout plans
Also among companies with IBD drugs in the clinic is another AI-based drug developer, Insilico Medicine.
Insilico has applied AI to design ISM5411, a small molecule candidate that it says plans to stand out as the first to treat IBD by inhibiting the prolyl hydroxylase domain (PHD). ISM541 is an oral, PHD-specific inhibitor designed to treat IBD by inducing the expression of gut barrier protective genes. ISM5411 is designed to directly regulate the intestinal immune microenvironment, so that the drug simultaneously reduces the gut inflammation seen in IBD while also promoting repair of the intestinal mucosal barrier.
In a paper published last December in Nature Biotechnology, Insilico founder and CEO Alex Zhavoronkov, PhD, and 22 co-authors based at the company shared how the company used its Chemistry42 generative chemistry engine to design and optimize novel gut-restricted inhibitors of PHD1 and PHD2, protein targets implicated in biological processes that include aging and regeneration.
“PHD is not a super novel target but it is novel for IBD. Our company wanted to go into IBD as it is a very large patient population and unmet medical need,” Zhavoronkov told GEN Edge soon after the paper was published. “Most other companies focus on the inflammatory cytokine pathways, immune cell trafficking mechanisms, or intracellular signaling nodes like JAK. Most of these just treat or ameliorate the symptoms. We wanted to produce a lasting disease-modifying effect.”
“The more drugs we have towards chronic conditions such as IBD which have a high amount of medical need, the better. The more folks that are looking to leverage AI in IBD and other I&I [immunology and inflammation] conditions, I think the more beneficial it ultimately is for patients,” says McClain.
Pipeline programs
ABS-101 is one of four disclosed programs in Absci’s pipeline. Also in that pipeline are:
- ABS-201, an anti-prolactin receptor (PRLR) antibody designed to treat androgenetic alopecia, also known as male and female pattern hair loss. ABS-201 is in the investigational new drug (IND)-enabling phase.
- ABS-301, an immuno-oncology candidate now in lead identification phase. Absci has said the fully human antibody is designed to bind to an undisclosed novel target discovered through Absci’s Reverse Immunology platform, with the aim of limiting immune infiltration and turning tumors immunologically “cold.”
- ABS-501, a HER2-targeting oncology program now in candidate identification phase. Absci says it is exploring a breast cancer indication as an alternative to the Daiichi Sankyo/AstraZeneca co-marketed therapy Enhertu® (fam-trastuzumab deruxtecan-nxki) or following treatment with the drug. ABS-501 was shown in a mouse xenograft model to suppress growth of trastuzumab-sensitive and resistant HER2+ breast tumors.
ABS-201 is expected to begin Phase I trials in early 2026, McClain said.
With Absci stepping up clinical development of its pipeline candidates, the company at deadline was seeking a Head of Clinical Operations who will oversee the planning and execution of clinical development programs.
Absci announced the dosing of its first clinical trial participants with ABS-101 on May 13, the same day it reported first quarter results. The company saw its Q1 net loss climb nearly 20% year-over-year, to $26.346 million from its $21.975 million net loss in the first three months of 2024. The company’s quarterly revenue, all derived from partner programs, rose 31% to $1.179 million in Q1 2025 from $898,000 a year earlier.
Absci finished the first quarter with $133.983 million in cash, cash equivalents, and short-term investments as of March 31, a sum the company says is enough of a “runway” to fund its operations into the first half of 2027. That cash position is up 19% from $112.425 million as of December 31, 2024—but down 17% from the $161.543 million the company reported March 31, 2024.
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