News & Publications

• Collaboration leverages Atavistik Bio’s proprietary AMPS™ platform to identify and advance novel allosteric small molecules against multiple targets for cardiometabolic diseases
• Atavistik Bio to receive $70 million upfront with potential milestone payments up to $1.9B plus tiered royalties

Cambridge, Mass., September 24 2026 – Atavistik Bio, a clinical-stage biotechnology company advancing a pipeline of hematology therapeutics developed through a novel allosteric discovery platform, today announced that it has entered into a strategic research collaboration with Roche to discover and develop novel small molecule therapeutics against multiple targets for cardiovascular, renal, and metabolic (CVRM) diseases.

The collaboration will leverage Atavistik Bio’s proprietary AMPS™ drug discovery platform and expertise in allosteric drug discovery to identify novel functional binding pockets and advance selective small molecule therapeutics against targets for CVRM diseases. By uncovering cryptic, biologically relevant binding pockets, Atavistik Bio’s platform enables new approaches to develop differentiated small molecules against therapeutically important targets that have historically been challenging to modulate.

“We are pleased to partner with Roche, whose deep expertise in drug development makes them an ideal partner to extend the reach of our allosteric discovery platform,” said Bryan Stuart, Chief Executive Officer of Atavistik Bio. “This collaboration reflects the strength and broad applicability of our platform across therapeutic areas, further validating our approach to allosteric drug discovery. We are excited to extend the application of our platform into CVRM diseases by leveraging Roche’s deep expertise in the field, while we remain focused internally on advancing a robust pipeline of potentially best-in-class therapies for patients with rare hematologic diseases.”

Atavistik Bio will receive an upfront payment of $70 million and may be eligible to receive additional research, development, and commercial milestone payments of up to $1.9 billion, and tiered royalties on future net sales of any approved medicines resulting from the collaboration.

Under the terms of the agreement, Atavistik Bio will be responsible for discovery and research activities for the collaboration targets, with Roche responsible for further preclinical and clinical development, regulatory, and commercialization activities.

“Treating CVRM diseases remains a core strategic priority for Roche. By pairing Atavistik Bio’s novel approach to discovering allosteric small molecules with our expertise in disease biology and drug development, we aim to translate complex science into transformative, oral small-molecule therapeutics for patients who need them most” said Boris L. Zaïtra, Head of Corporate Business Development at Roche.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company advancing a pipeline of hematology therapeutics developed through a novel allosteric discovery approach. Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT) and advancing a JAK2^V617F mutant-selective inhibitor program for myeloproliferative neoplasms.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed therapies and backed by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Precision AQ
investors@atavistikbio.com

• Partnership with Cure HHT strengthens patient engagement and supports streamlined clinical trial execution
• Harmony-HHT Phase 1/2 trial activates first clinical site at Massachusetts General Hospital, marking an important milestone for ATV-1601

Cambridge, Mass., July 30, 2026 – Atavistik Bio, a clinical-stage biotechnology company developing the next generation of oral selective allosteric therapeutics for the treatment of rare hematological diseases, today announced it has joined the Cure HHT Clinical Trial Network (CTN) to support the clinical development of ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT).

Joining the Cure HHT CTN coincides with activation of the first clinical site in Atavistik Bio’s Harmony-HHT Phase 1/2 study at Massachusetts General Hospital (MGH), marking an important milestone in the advancement of the clinical program.

Through participation in the Cure HHT CTN, Atavistik Bio will work closely with Cure HHT to leverage the organization’s trial-qualified Centers of Excellence, established patient and clinician community, and extensive HHT-specific clinical and regulatory expertise to support execution of the Harmony-HHT study. The Cure HHT CTN is a first-of-its-kind partnership platform designed to accelerate development of new therapies for HHT.

“As our Harmony-HHT Phase 1/2 trial advances into the clinic, our partnership with Cure HHT strengthens our ability to engage and contribute to the HHT community in addition to efficiently executing the study,” said Susan Pandya, M.D., Chief Medical Officer of Atavistik Bio. “Beyond activating our first clinical site at the world-renowned HHT Center of Excellence, Massachusetts General Hospital, this collaboration connects us with a global network of HHT experts, clinical investigators, and patient advocates who share our commitment to accelerating the development of novel treatment options.”

Through the collaboration, Atavistik Bio and Cure HHT will work together on patient education, community outreach, clinical trial awareness initiatives, and engagement activities designed to help connect eligible individuals with the Harmony-HHT study.

“We are thrilled to welcome Atavistik Bio to the Cure HHT Clinical Trial Network,” said Marianne Clancy, CEO, Cure HHT. “ATV-1601 represents an exciting investigational approach for HHT, and we look forward to partnering with the Atavistik Bio team to help connect the HHT community with the Harmony-HHT study and support the development of potential new therapies for people living with this disease.”

HHT is the second most prevalent inherited bleeding disorder, affecting more than 80,000 people in the US and 1.6 million people globally, with no approved therapies currently available. HHT is caused by loss-of-function mutations in ENG, ALK1 or SMAD4 genes which encode proteins that regulate the growth and branching of endothelial cells into blood vessels. Impaired function of these proteins results in hyperactivation of the AKT1 pathway resulting in malformed blood vessels called arteriovenous malformations (AVMs), which can rupture or cause abnormal blood flow, leading to chronic bleeding, anemia, organ damage, and for some, life-threatening complications. There are currently no approved therapies for HHT.

ATV-1601 is an investigational oral allosteric inhibitor that selectively targets AKT1, a key driver of the vasculopathy underlying HHT. By selectively inhibiting AKT1, ATV-1601 has the potential to provide a disease-modifying approach for HHT, with the potential to address all HHT-driver mutations (ENG, ALK1, and SMAD4) with a well-tolerated profile. In preclinical HHT models with ENG, ALK1, and SMAD4 mutations, ATV-1601 significantly reduced AVM formation, supporting its potential as a novel therapy applicable for all people living with HHT. ATV-1601 has received FDA Fast Track designation for the treatment of HHT.

The Harmony-HHT Phase 1/2 clinical trial (NCT07601425) is a proof-of-concept study designed to evaluate the safety and efficacy of ATV-1601 for individuals with moderate to severe HHT. Part 1 is a randomized, double-blind, multicenter, placebo-controlled study evaluating three oral dosing regimens of ATV-1601 over a 16-week treatment period. Eligible participants who complete Part 1 may enroll in an open-label extension (Part 2) to receive ATV-1601.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company advancing a pipeline of hematology therapeutics developed through a novel allosteric discovery approach. Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT) and advancing a JAK2^V617F mutant-selective inhibitor program for myeloproliferative neoplasms.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Precision AQ
investors@atavistikbio.com

• Mr. Dable brings deep expertise in clinical development, commercialization, and corporate strategy to support Atavistik Bio’s next phase of growth

Cambridge, Mass., July 29, 2026 – Atavistik Bio, a clinical-stage biotechnology company developing the next generation of selective allosteric therapeutics for the treatment of rare hematological diseases, today announced the appointment of Habib Dable to its Board of Directors. Mr. Dable joins the Board as the Company advances ATV-1601 for Hereditary Hemorrhagic Telangiectasia (HHT) and positions itself for its next phase of growth focused in rare hematology.

Mr. Dable brings more than 30 years of leadership experience across the global biopharmaceutical industry, including deep expertise in building and scaling blockbuster franchises and guiding companies through transformative growth.

“We are excited to welcome Habib to our Board of Directors,” said Bryan Stuart, Chief Executive Officer of Atavistik Bio. “As Atavistik Bio advances to its next phase of growth and our pipeline focused on addressing serious rare hematological diseases, Habib’s experience scaling innovative biotechnology companies will be invaluable. Habib brings a unique combination of operating, commercial, and strategic experience that is directly relevant to Atavistik Bio today. His proven track record of strategic execution is highly relevant as we advance our pipeline, achieve key development milestones and establish Atavistik Bio as a leader in rare hematology.”

“I am honored to join the Board of Directors at such an exciting time for the Company,” said Mr. Dable. “Atavistik Bio has built a strong scientific foundation, a highly experienced team, and a disciplined approach to advancing differentiated therapies for patients with significant unmet needs. I am particularly excited by the strength of Atavistik’s pipeline, including the promise of ATV-1601, which I believe represents a differentiated approach to addressing the underlying biology of HHT as well as its emerging JAK2^V617F mutant selective inhibitor program for myeloproliferative neoplasms. As Atavistik Bio approaches key milestones and enters a new phase as a clinical-stage rare hematology company, I look forward to partnering with management and the Board to advance its pipeline and help execute its long-term vision.”

Mr. Dable previously served as the President and Chief Executive Officer at Acceleron Pharma where he led the company through its evolution into a leading rare disease company, including the successful launch of Reblozyl® and eventual sale to Merck in 2021. Prior to that, Mr. Dable held roles of increasing leadership responsibility at Bayer AG for over 20 years, including President of U.S. Pharmaceuticals and Global Head of Specialty Medicine. He is currently an advisor at RA Capital Management and serves on the boards of several biotech companies including Tyra Biosciences, SpyGlass Pharma, Relay Therapeutics, and PepGen and is a former board member of Blueprint Medicines, Day One Biopharmaceuticals, Millendo Therapeutics, Aerovate Therapeutics, and Albireo Pharma. He holds a B.B.A. and M.B.A. from the University of New Brunswick.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company advancing a pipeline of hematology therapeutics developed through a novel allosteric discovery approach. Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT) and advancing a JAK2^V617F mutant-selective inhibitor program for myeloproliferative neoplasms.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Precision AQ
investors@atavistikbio.com

 

• FDA cleared the Harmony-HHT Phase 1/2 study design, enabling Atavistik Bio to leverage existing clinical data with ATV-1601 and advance directly into a randomized study with individuals who have moderate to severe HHT
• Fast Track Designation supports the development of ATV-1601 for HHT, the second most prevalent inherited bleeding disorder, affecting more than 80,000 people in the US and 1.6 million people globally
• ATV-1601 is an oral, selective allosteric AKT1 inhibitor with disease-modifying potential for all driver mutations of HHT, which currently has no approved therapies for people with this severe and debilitating chronic disease

Cambridge, Mass., June 9, 2026  – Atavistik Bio, a biotechnology company discovering the next generation of selective allosteric therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for the Harmony-HHT study, a Phase 1/2 trial evaluating ATV-1601 in individuals with moderate to severe Hereditary Hemorrhagic Telangiectasia (HHT). In addition to the IND clearance, Atavistik Bio also announced that ATV-1601 has been granted Fast Track Designation by the FDA.

The FDA’s clearance of the Harmony-HHT Phase 1/2 study design allows Atavistik Bio to leverage existing clinical data with ATV-1601 and proceed directly into a randomized study. This approach creates the opportunity to accelerate development timelines while rapidly generating clinical proof-of-concept data.

“We recognize the significant burden HHT places on individuals and families, as recurrent bleeding, chronic anemia, and progressive complications can affect daily life and well-being,” said Susan Pandya, M.D., Chief Medical Officer at Atavistik Bio. “Treatment options are largely limited to supportive care and invasive interventions. ATV-1601 has the potential to provide a disease-modifying approach for people living with HHT. FDA clearance of our study design allows us to move directly into a randomized trial and, together with Fast Track Designation and existing ATV-1601 clinical experience, positions us to efficiently advance development for the HHT community.”

HHT is the second most prevalent inherited bleeding disorder, affecting more than 80,000 people in the US and 1.6 million people globally, with no approved therapies currently available. HHT is caused by loss-of-function mutations in ENG, ALK1 or SMAD4 genes which encode proteins that regulate the growth and branching of endothelial cells into blood vessels. Impaired function of these proteins results in hyperactivation of the AKT1 pathway resulting in malformed blood vessels called arteriovenous malformations (AVMs), which can rupture or cause abnormal blood flow, leading to chronic bleeding, anemia, organ damage, and for some, life-threatening complications.

ATV-1601 is an investigational oral allosteric inhibitor that selectively targets AKT1, a key driver of the vasculopathy underlying HHT. By selectively inhibiting AKT1, ATV-1601 has the potential to provide a disease-modifying approach for HHT, with the potential to address all HHT-driver mutations (ENG, ALK1, and SMAD4) with a well-tolerated profile. In preclinical HHT models with ENG, ALK1, and SMAD4 mutations, ATV-1601 significantly reduced AVM formation, supporting its potential as a novel therapy applicable for all people living with HHT.

The Harmony-HHT study is a Phase 1/2 trial (NCT07601425) proof-of-concept study designed to evaluate the safety and efficacy of ATV-1601 for individuals with moderate to severe HHT. Part 1 is a randomized, double-blind, multicenter, placebo-controlled study evaluating three oral dosing regimens of ATV-1601 over a 16-week treatment period. Eligible participants who complete Part 1 may enroll in an open-label extension (Part 2) to receive ATV-1601.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company developing novel allosteric therapeutics, with an internal pipeline focused on rare hematology.  Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT) and advancing a JAK2^V617F mutant-selective inhibitor program for myeloproliferative neoplasms.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

• Series B funds will enable clinical development of ATV-1601, an oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT), and the advancement of its JAK2 V617F mutant-selective inhibitor program for myeloproliferative neoplasms (MPNs) through clinical proof of concept
• RA Capital Management joins Atavistik Bio’s syndicate, which also includes Nextech Invest, The Column Group, Lux Capital and Regeneron Ventures

Cambridge, Mass., March 5, 2026 – Atavistik Bio, a biotechnology company discovering the next generation of selective allosteric therapeutics, today announced the closing of a $40 million extension to its Series B financing from new investor RA Capital Management, bringing the total round proceeds to $160 million. RA Capital joins other top-tier investors who participated in the Series B round, including Nextech Invest, The Column Group, Lux Capital and Regeneron Ventures, as previously announced in December 2025. Atavistik Bio will use the proceeds from the upsized Series B financing to fund clinical development of ATV-1601, a potential best-in-class AKT1-selective oral inhibitor for HHT, and its JAK2 V617F mutant-selective inhibitor program for MPNs through clinical proof of concept.

“We’re excited to welcome RA Capital as an investor in Atavistik Bio. Our Series B round, backed by a highly respected syndicate, reflects strong belief in our selective allosteric programs and our focus to advance meaningful therapies for patients with severe diseases,” said Bryan Stuart, Chief Executive Officer at Atavistik Bio. “This additional funding further strengthens our ability to accelerate the development of best-in-class therapies designed to deliver superior efficacy, improved tolerability profiles, and potentially transformative outcomes for patients.”

“We are pleased to support Atavistik Bio in this next phase of growth as the company is at the forefront of developing transformative therapies for HHT and MPNs,” said Nandita Shangari, PhD, Managing Director at RA Capital Management. “Atavistik Bio’s highly experienced team and a pipeline of high-quality programs targeting significant unmet need, position the company to create meaningful value.”

About Hereditary Hemorrhagic Telangiectasia (HHT), AKT1 Inhibition and ATV-1601

HHT is a severe inherited bleeding disorder that affects more than 1.6 million people globally, with no approved therapies currently available. This condition often leads to frequent bleeding episodes and vascular shunts, resulting in chronic anemia, multiorgan damage, and life-threatening complications. AKT1 hyperactivation is a hallmark of HHT and has been shown to drive the vascular pathology of HHT. Selectively inhibiting AKT1, the primary AKT isoform and driver of abnormal endothelial growth implicated in HHT, offers a novel and potentially disease-modifying therapeutic approach for this difficult disease. Although there has been substantial investment in pan-AKT inhibitors, their use is limited by AKT2-driven toxicities, most notably hyperglycemia, which impact tolerability and restrict their use for chronic dosing. ATV-1601 is an oral allosteric inhibitor that selectively inhibits AKT1.

ATV-1601 was evaluated in a Phase 1 oncology study, and demonstrated a favorable safety profile, validating its differentiated selectivity profile that addresses key limitations of pan-AKT inhibitors. Development efforts will now focus on advancing ATV-1601 in HHT.

About Myeloproliferative Neoplasms (MPNs) and Selective Targeting of the JAK2 V617F Mutation

MPNs are a group of rare chronic blood cancers for which current treatment options are limited. The JAK2 V617F mutation is the most common driver mutation in patients living with MPNs, affecting approximately 95% of patients with polycythemia vera, 60% of patients with essential thrombocythemia, and 55% of patients with myelofibrosis. Approved pan-JAK inhibitors, such as ruxolitinib, provide symptom relief, but non-selectively inhibit both mutant and wild-type JAK2. This limits the ability to reduce JAK2 V617F mutant allele burden and can disrupt normal blood cell production regulated by wild-type JAK2, contributing to adverse events and treatment discontinuation. Selectively targeting the JAK2 V617F mutation has the potential to reduce mutant allele burden, preserve normal bone marrow function, and have a disease modifying impact that will substantially improve long term outcomes for patients with MPNs.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company accelerating the discovery and development of transformative selective allosteric therapeutics to address serious unmet patient needs. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

• Proceeds will support the advancement of Atavistik Bio’s oral allosteric AKT1-selective inhibitor for Hereditary Hemorrhagic Telangiectasia (HHT) and the JAK2 V617F mutant-selective inhibitor program for myeloproliferative neoplasms (MPNs) through clinical proof of concept
• Atavistik Bio anticipates initiating its clinical trial in HHT in the first half of 2026

Cambridge, Mass., December 18, 2025 – Atavistik Bio, a biotechnology company discovering the next generation of precision allosteric therapeutics, today announced that it has raised a $120 million Series B financing to support the advancement of selective allosteric small molecule therapeutics for the treatment of HHT and MPNs. The financing was led by Nextech Invest and The Column Group, with participation from existing investor Lux Capital and new investor Regeneron Ventures. Atavistik Bio will use the proceeds from the Series B financing to advance its oral allosteric AKT1-selective inhibitor for HHT and its JAK2 V617F mutant-selective inhibitor program for MPNs through clinical proof of concept.

“We’re thrilled to announce our Series B financing, and grateful for the support of both our existing and new investors. With this capital, we are well-positioned to advance both our HHT and MPN programs through key clinical proof-of-concept milestones, with the HHT program expected to enter the clinic in the first half of 2026,” said Bryan Stuart, Chief Executive Officer at Atavistik Bio. “Our precision allostery approach holds tremendous potential to deliver best-in-class therapeutics with superior efficacy and tolerability profiles. Our team is working with tremendous passion and momentum to translate that promise into meaningful outcomes for patients.”

HHT is a severe bleeding disorder that affects more than 1.6 million people globally, with no approved therapies currently available. This condition often leads to frequent bleeding episodes, resulting in chronic anemia, organ damage, and a reduced lifespan. AKT1 hyperactivation is a hallmark of HHT and has been shown to drive the vascular pathology of HHT. Selectively inhibiting AKT1, the primary AKT isoform and driver of abnormal endothelial growth implicated in HHT, offers a novel and potentially disease-modifying therapeutic approach for this difficult disease. Although there has been substantial investment in pan-AKT inhibitors, their use is limited by AKT2-driven toxicities, most notably hyperglycemia, which impact tolerability and restrict their use for chronic dosing. Atavistik Bio has developed an oral allosteric inhibitor that selectively inhibits AKT1, overcoming the shortcomings of pan-AKT inhibitors and offering improved therapeutic potential and tolerability.

MPNs are a group of rare chronic blood cancers for which current treatment options are limited. The JAK2 V617F mutation is the most common driver mutation in patients living with MPNs, affecting approximately 95% of patients with polycythemia vera, 60% of patients with essential thrombocythemia, and 55% of patients with myelofibrosis. Approved pan-JAK inhibitors, such as ruxolitinib, provide symptom relief, but non-selectively inhibit both mutant and wild-type JAK2. This limits the ability to reduce JAK2 V617F mutant allele burden and can disrupt normal blood cell production regulated by wild-type JAK2, contributing to adverse events and treatment discontinuation. Selectively targeting the JAK2 V617F mutation has the potential to reduce mutant allele burden, preserve normal bone marrow function, and have a disease modifying impact that will substantially improve long term outcomes for patients with MPNs.

“We’ve been continually impressed by the exceptional quality of the compounds discovered from Atavistik Bio’s proprietary AMPS™ platform and by the team’s ability to consistently execute against critical milestones,” said John A. Josey, PhD, Atavistik Board Chair. “Atavistik Bio’s HHT and MPN programs would represent enormous advancements in the treatment of these respective diseases. We look forward to seeing Atavistik Bio move these programs into the clinic and bring them closer to patients in need.”

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company accelerating the discovery and development of transformative precision allosteric therapeutics to address serious unmet patient needs. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, and Regeneron Ventures. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

 

• Atavistik Bio to present the discovery and characterization of novel allosteric JAK2 V617F selective inhibitors that suppress mutant-dependent signaling and proliferation while sparing wild-type JAK2
• Atavistik Bio’s JAK2 V617F mutant-selective inhibitors have the potential to address the significant unmet needs of patients with myeloproliferative neoplasms by providing disease-modifying efficacy while avoiding off-target hematological effects associated with wild-type JAK inhibition

Cambridge, Mass., December 4, 2025 – Atavistik Bio, a biotechnology company discovering the next generation of precision allosteric therapeutics, today announced that it will unveil the discovery of its novel, potent, and selective allosteric inhibitors of JAK2 V617F for the treatment of myeloproliferative neoplasms (MPNs) and present compelling, supportive preclinical data for the program at the 67th American Society of Hematology (ASH) Annual Meeting and Exhibition to be held in Orlando December 6-9, 2025.

The JAK2 V617F mutation is the most common driver mutation in patients living with MPNs, affecting approximately 95% of patients with polycythemia vera (PV), 60% of patients with essential thrombocythemia (ET) and 55% of patients with myelofibrosis (MF). Selectively targeting the JAK2 V617F mutation has the potential to reduce mutant allele burden, modify disease progression, and significantly enhance treatment outcomes for MPN patients. Approved pan-JAK inhibitors, such as ruxolitinib, target the active site in the JH1 kinase domain of JAK2. Though they have demonstrated clinical benefit and symptomatic relief, pan-JAK inhibitors non-selectively inhibit both mutant and wild-type JAK2. This limits the ability to significantly reduce JAK2 V617F mutant allele burden that is correlated with disease progression. Additionally, inhibition of wild-type JAK2 disrupts normal hematopoiesis, leading to adverse events, and contributes to treatment discontinuation. These limitations underscore the need for mutant-selective, disease-modifying therapies. A highly selective allosteric JAK2 V617F mutant-inhibitor that spares wild-type JAK2 has the potential to provide disease-modifying efficacy, enable molecular remission by reducing mutant JAK2 allele burden and improve hematologic tolerability.

At ASH, Atavistik Bio will present the identification of JAK2 V617F mutant-selective inhibitors that bind the allosteric pseudokinase JH2 domain, which harbors the V617F mutation. These compounds were developed using the company’s proprietary AMPS™ platform and structure-based drug design and demonstrate picomolar affinity for the JAK2 V617F JH2 domain and >100-fold selectivity over related JAK family kinases (TYK2, JAK1). In preclinical models, these compounds show potent inhibition of JAK2 V617F-dependent signaling and proliferation in multiple human hematopoietic cancer cell lines harboring the mutations while sparing wild-type JAK2 cells. Additionally, these compounds demonstrated more than ten-fold selective inhibition of cytokine-independent JAK2 V617F signaling over cytokine-dependent wild-type JAK2 signaling in an isogenic cell model in which the JAK2 V617F mutation was edited to wild-type. In a mouse model bearing JAK2 V617F-expressing SET2 tumors, Atavistik JAK2 V617F selective inhibitors demonstrated strong target engagement, evidenced by reduced phosho-STAT5 levels. Compounds with favorable PK properties, good tolerability, and limited off target activity are being advanced for further development.

“We are pleased to share the discovery of our novel JAK2 V617F mutant-selective allosteric inhibitor program and compelling preclinical data at the upcoming ASH meeting,” said Marion Dorsch, Ph.D., President and Chief Scientific Officer, Atavistik Bio. “Unlike pan-JAK inhibitors that broadly target JAK2 and can only manage symptoms of myeloproliferative neoplasms, our highly selective allosteric JAK2 V617F mutant-selective inhibitors have the potential to deliver durable responses and mitigate off-target hematological effects, offering a powerful disease-modifying benefit and addressing the unmet needs of this patient population.”

Atavistik Bio Poster Presentation Details

Poster Title: Discovery of JAK2V617F mutant specific allosteric inhibitors for the treatment of myeloproliferative neoplasms
Session Name: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster I
Session Date and Time: Saturday, December 6, 2025, 5:30 PM – 7:30 PM ET
Location: Orange County Convention Center – West Halls B3-B4
Poster #: 1977

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company accelerating the discovery and development of transformative precision allosteric therapeutics to address serious unmet patient needs. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery. Atavistik Bio is currently conducting a Phase 1 clinical trial of ATV-1601, an allosteric selective inhibitor for solid tumors.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, and Lux Capital. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

• Dr. Pandya brings 20 years of drug development expertise in precision medicine in both oncology and hematology, with previous senior leadership roles at Servier Pharmaceuticals and Agios Pharmaceuticals
• Her appointment follows Atavistik Bio’s recent initiation of a Phase 1 clinical trial evaluating ATV-1601, an allosteric selective inhibitor for AKT1 E17K-driven solid tumors
• She will also play a critical role in advancing the company’s preclinical pipeline programs toward the clinic

Cambridge, Mass., September 15, 2025 – Atavistik Bio, a biotechnology company discovering the next generation of precision allosteric therapeutics, today announced the appointment of Susan Pandya, M.D., as Chief Medical Officer (CMO). Dr. Pandya brings 20 years of drug development experience including successful regulatory approvals. She has a track record of building and leading highly effective teams and delivering novel precision medicines for unmet patient needs. As CMO, Dr. Pandya will lead all clinical development strategy and execution for Atavistik Bio, including the ongoing Phase 1 clinical trial of its lead program, ATV-1601, an allosteric selective inhibitor for AKT1 E17K-driven solid tumors. She will also play a central role in advancing the company’s preclinical pipeline into efficient and well-designed clinical trials.

“We are very excited to welcome Susan to the Atavistik Bio team as we transition to a clinical stage organization,” said Bryan Stuart, Chief Executive Officer at Atavistik Bio. “Susan’s expertise in guiding oncology programs through early development, global registration studies, and marketing approval will be instrumental as we progress the ongoing clinical study of our lead precision allosteric candidate, ATV-1601, while continuing to rapidly advance our other pipeline programs toward the clinic.”

“Atavistik Bio’s commitment to discovering and developing both best-in-class and first-in-class allosteric therapeutics for significantly underserved patient populations represents an important advancement in the field of precision oncology medicine,” said Dr. Pandya. “I am thrilled to join this exceptional team at such a pivotal time for the company, and I look forward to helping advance this exciting portfolio of novel precision allosteric therapeutics for patients in dire need of new therapeutic options.”
Dr. Pandya most recently served as Vice President, Clinical Development and Global Head of late-stage oncology at Servier Pharmaceuticals where she led all late-stage clinical development programs in oncology and hematology and played an integral role in corporate business development and portfolio strategy. Before joining Servier, Dr. Pandya led the clinical development of the isocitrate dehydrogenase (IDH) inhibitor portfolio at Agios Pharmaceuticals, acquired by Servier in 2021 and oversaw multiple global drug approvals across a range of oncology indications. Earlier in her career, she focused on early-phase development at Acceleron Pharma.

Dr. Pandya earned her M.D. from Tufts University School of Medicine and completed her residency in internal medicine and fellowship in hematology/oncology and held a clinical practice at Beth Israel Deaconess Medical Center. She also serves on the Board of Directors of MOMA Therapeutics.

About Atavistik Bio

Atavistik Bio is a clinical stage biotechnology company accelerating the discovery and development of transformative precision allosteric therapeutics to address serious unmet patient needs. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery. Atavistik Bio is currently conducting a Phase 1 clinical trial of ATV-1601, an allosteric selective inhibitor for solid tumors.

Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, and Lux Capital. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

 

• Dr. William Sellers is a leading cancer researcher who currently serves as a Core Institute member and Director of the cancer program at the Broad Institute and a professor of medicine at Dana-Farber Cancer Institute and Harvard Medical School
• He brings deep expertise and experience in cancer biology and cancer therapeutics to Atavistik Bio as the company advances its pipeline of precision allosteric small molecule therapeutics

Cambridge, Mass., March 31, 2025 – Atavistik Bio, a biotechnology company discovering the next generation of precision allosteric therapeutics inspired by the body’s natural regulators, today announced that it has appointed renowned cancer scientist William Sellers, M.D. to its Scientific Advisory Board. Dr. Sellers joins Atavistik Bio as the company rapidly advances a pipeline of next generation allosteric small molecules for high-value clinically validated targets that have the potential to address significant unmet needs.

“We’re thrilled to welcome Dr. Bill Sellers to our SAB at this pivotal moment for the company as we advance our pipeline and transition to the clinic with ATV-1601, a selective inhibitor targeting AKT1 E17K driven cancers,” said Marion Dorsch, Ph.D., President and Chief Scientific Officer, Atavistik Bio. “As an accomplished industry and academic leader, Bill’s many contributions have led to a deeper understanding of cancer biology and cancer genomics that, in turn, has yielded transformative therapeutic advancements for patients. With his extensive experience in oncology, Bill will provide invaluable guidance as we work to develop innovative therapies and advance our pipeline of next-generation precision allosteric therapeutics for patients.”

“Precision small molecule medicines have transformed the cancer treatment landscape, resulting in better outcomes for many patients. Despite this notable progress, as we continually unfold the relentless intricacies of cancer biology, we have only scratched the surface of what’s possible,” said Dr. Sellers. “I am impressed with Atavistik Bio’s pipeline and novel approach to discover next-generation precision small molecule medicines that leverage the power of allostery, and I look forward to helping further the team’s pursuit of high-value targets aimed at addressing serious unmet needs.”

Dr. Sellers currently serves as a core institute member and director of the cancer program at the Broad Institute and a professor of medicine at Dana-Farber Cancer Institute and Harvard Medical School. At the Broad Institute, Dr. Sellers directs a research group focused on translating genomic discoveries into new therapeutics.

Previously, Dr. Sellers directed cancer drug discovery and early cancer clinical development at the Novartis Institutes for Biomedical Research. Prior to joining Novartis, Dr. Sellers was an associate professor of medicine at Dana-Farber Cancer Institute and Harvard Medical School and an associate member of the Broad Institute.

Dr. Sellers has spent his academic career at the intersection of cancer biology and cancer genomics, investigating the basic mechanisms of tumor development. He collaborated with his Dana-Farber and Broad colleague Matthew Meyerson to lead the Broad’s first major foray into cancer genome sequencing. Their work, as well as work by other groups including investigators at Massachusetts General Hospital, led to the identification of EGFR mutations in lung cancer – work that paved the way for EGFR-inhibiting drugs becoming standard-of-care for patients.

Among other achievements in his career, Dr. Sellers was the founder of Civetta Therapeutics, a co-founder of Delphia Therapeutics and currently serves on the Scientific Advisory Boards of Ideaya Bioscience and Epidarex Capital. Additionally, Dr. Sellers was a previous member of the National Cancer Advisory Board.

About Atavistik Bio

Atavistik Bio is a biotechnology company accelerating the discovery and development of transformative precision allosteric therapeutics to address serious unmet patient needs. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery. ATV-1601, a selective allosteric inhibitor for solid tumors, is anticipated to enter the clinic in early 2025. Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, and Lux Capital. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com

• Collaboration will leverage Atavistik Bio’s proprietary AMPS™ platform to rapidly identify and validate novel allosteric binders against two undisclosed, Pfizer-designated targets
• Atavistik Bio has rapidly identified novel, functional, allosteric binding pockets across a broad range of intractable target classes
• Collaboration with Pfizer will enable Atavistik Bio to extend the reach of its platform to potentially address a broader range of oncology indications, while simultaneously progressing its internal pipeline of precision oncology small molecule therapies

Cambridge, Mass., January 2, 2025 – Atavistik Bio, a biotechnology company committed to discovering the next generation of precision allosteric therapeutics inspired by the body’s natural regulators, today announced that it has entered into a research collaboration with Pfizer (NYSE: PFE) to accelerate the discovery of novel precision allosteric therapeutics to address significant unmet medical needs.

Under the terms of the collaboration, Atavistik Bio will leverage its proprietary AMPS™ platform to identify novel allosteric binders against two undisclosed, targets selected by Pfizer. At the completion of the research period, Pfizer will have the option to license the programs. The financial terms of the collaboration are undisclosed.

“Allostery holds the key to targeting disease-causing proteins and delivering highly selective, better tolerated, and more effective therapies. We’re excited to collaborate with Pfizer to harness the tremendous promise of allostery to advance the opportunity for therapeutic innovations for patients in need,” said Bryan Stuart, CEO of Atavistik Bio. “Our internal pipeline has been enabled by our ability to identify novel, functional allosteric binding pockets that allow us to rapidly progress novel chemical matter. We look forward to applying the efficiency and productivity of our platform to our collaboration with Pfizer as we simultaneously advance our internal pipeline of precision oncology small molecule therapies.”

About Atavistik’s Platform

Atavistik Bio’s proprietary AMPS™ platform is a highly integrated discovery engine that combines proprietary computational and experimental techniques to rapidly unlock functional cryptic pockets across a broad range of target classes. By leveraging insights from these novel allosteric chemical starting points, the platform enables the design of small molecules against historically challenging targets. Our highly integrated team and automated data infrastructure makes rapid iteration and progression of chemistry possible, while the versatility of its platform allows the company to accelerate the development of groundbreaking medicines across any therapeutic area.

About Atavistik Bio

Atavistik Bio is a biotechnology company accelerating the discovery and development of transformative precision allosteric therapeutics to address serious unmet patient needs, with a focus on oncology. Since its inception, Atavistik Bio has rapidly established an emerging pipeline of allosteric therapeutics with the potential to achieve superior efficacy and tolerability profiles by leveraging the power of allostery. ATV-1601, a selective allosteric inhibitor for solid tumors, is anticipated to enter the clinic in early 2025. Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, and Lux Capital. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.

Media Contact:

Liz Melone
Melone Communications, LLC
liz@melonecomm.com