Arkayli Biopharma Inc. Receives Study May Proceed Response to IND from FDA and SBIR Grant from NIH to Advance Clinical Development of ARK001 for a Rare Pediatric Disease

• Study May Proceed (SMP) letter is based on IND filing to conduct a Phase 1b Trial of ARK001 in Infants with Infantile Hemangioma (IH)
• NIH SBIR grant of $2.98 Million acknowledges the high unmet need for a non-systemic treatment for IH
• Appointment of new CEO, Katie MacFarlane, PharmD, to lead the company transition into clinical development
• The company is launching a Series A financing round

MADISON, Wis., Oct. 6, 2026 /PRNewswire/ -- Arkayli BioPharma Inc. ("Arkayli"), a biopharmaceutical company dedicated to developing innovative therapies for rare dermatologic conditions, today announced that the U.S. Food and Drug Administration (FDA) has responded to its Investigational New Drug (IND) application for its lead investigational product candidate, ARK001, with a Study May Proceed (SMP) letter. In addition, Arkayli received a Notice of Award (NoA) from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) for a Small Business Innovative Research (SBIR) grant totaling $2.98 Million (grant number 1R44 HL184627-01).

The company is preparing to initiate a Phase 1B clinical trial evaluating the safety of ARK001 in infants diagnosed with proliferating infantile hemangioma (IH). In addition, exploratory measures of effect will be assessed as part of the study. Arkayli believes that ARK001 has the potential for improved treatment of IH, through the topical, non-systemic treatment of this disfiguring skin disease. ARK001 is intended to provide a treatment option that can be used early in the disease by pediatricians, dermatologists, and parents and caregivers.

The Arkayli Board of Directors has appointed a new Chief Executive Officer, Katie MacFarlane, PharmD. Katie has over 35 years of experience in pharmaceutical drug development and commercialization, including products for a range of dermatologic conditions. To further support the activities to advance its pipeline, Arkayli is actively raising a Series A financing round.

"The Study May Proceed response to our IND from the FDA represents a key regulatory milestone for Arkayli and a significant step forward for babies and families affected by infantile hemangioma," said Katie MacFarlane, CEO of Arkayli Biopharma. "ARK001 is designed to address critical unmet needs in this vulnerable patient population, potentially offering a topical approach to treatment of this rare pediatric disease. With the NIH grant, we are focused on preparing for the Phase 1b trial, and we also are looking to accelerate our pipeline development through a Series A financing round."

"I am proud of what our team has accomplished thus far" said Thomas Rossi, PhD, Chairman of the Board of Arkayli Biopharma, Inc., "completing the preclinical research, formulation, and manufacturing preparation to support the IND filing. We look forward to entering the clinic with ARK001."

About Arkayli Biopharma, Inc.

Arkayli Biopharma Inc. is a biopharmaceutical company founded in 2022 with the mission to develop novel medications with precision drug delivery systems designed to enhance efficacy and maximize safety of treatments for children and adults with vascular anomalies. Our lead product candidate, ARK001, has the potential to be the first FDA-approved non-systemic topical treatment for infantile hemangioma (IH).

About ARK001

ARK001 is Arkayli BioPharma's lead investigational asset in development for the treatment of infantile hemangioma. Designed with a proprietary formulation for application to infant skin, ARK001 is intended to provide a topical therapy for early intervention for infants requiring treatment of proliferating superficial infantile hemangiomas.

About Infantile Hemangioma

Infantile hemangioma (IH) is the most common benign vascular tumor of infancy, affecting approximately 4% to 5% of newborn babies, or approximately 160,000 infants per year. While IH can resolve spontaneously without severe complications, over 50% of IHs result in permanent sequelae if left untreated, such as scarring. The majority of IH appear on the face, head, or neck, where scarring may require plastic surgery or laser therapy as the child matures and can contribute to psychosocial distress for children and their parents. The ideal time for intervention in IH is in the first 1-2 months of life, when proliferation is most rapid and damage to the dermis of the skin occurs that results in permanent scarring or disfiguration. However, currently, it takes an average of over 5 months for an infant with IH to be seen by a hemangioma specialist. There is a high unmet market need for a safe, effective, topically applied therapy that can be used early in diagnosis and prescribed by pediatricians and dermatologists.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve inherent risks and uncertainties, including statements regarding the timing and success of the proposed Phase 1b clinical trial, the ability to secure Series A financing, and regulatory pathways. Actual results may differ materially from those projected. Arkayli undertakes no obligation to update these statements in light of new information or future events.

SOURCE Arkayli Biopharma, Inc.