Anelixis Therapeutics Announces a Financing Led by BVF Partners L.P. to Fund Phase 2 Development of AT-1501

Financing to Support Clinical Development of AT-1501 in Amyotrophic Lateral Sclerosis (ALS), Transplant Rejection and Autoimmune Diseases

Company Also Announces Successful Completion of Phase 1 Study of AT-1501 in Healthy Volunteers and ALS Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Anelixis Therapeutics Inc. Anelixis Therapeutics, Inc., “Anelixis” is a clinical stage development company with a portfolio of antibodies blocking CD40 Ligand (CD40L) activity being developed as potential treatments for amyotrophic lateral sclerosis (ALS), organ and cellular transplantation, and autoimmune diseases. The financing announced today was led by Biotechnology Value Fund, L.P. and other affiliates of BVF Partners L.P. (“BVF”), a San Francisco-based private investment firm which invests exclusively in biotechnology companies. Additional financial terms were not disclosed.

Anelixis is developing its lead anti-CD40L antibody, AT-1501, as a potential treatment to slow the progression of ALS, for prevention of allograft rejection after organ and cellular transplantation, and as a therapy for autoimmune diseases that impair kidney function.

Anelixis also announced completion of a Phase 1 safety and pharmacokinetic study of AT-1501, involving both healthy volunteers and participants with ALS. AT-1501 was well tolerated at all doses tested and the pharmacokinetic properties of AT-1501 were as predicted for an IgG1 antibody. Anelixis expects that these results will enable further clinical studies in ALS and multiple other indications. Details of the study will be presented at an upcoming scientific meeting.

“With significant support from BVF, Anelixis is poised to launch an aggressive clinical development program for evaluating the safety and efficacy of AT-1501 for ALS, islet cell transplant, and other autoimmune diseases. BVF’s support and commitment, along with that of our founding investors, is a substantial endorsement of our approach,” said Steven Perrin, Ph.D., Founder and CEO of Anelixis. “ALS, allograft rejection and autoimmune diseases represent devastating unmet medical needs and we are eager to explore the potential value of AT-1501 as rapidly and diligently as possible. Advancement of AT-1501 into a robust clinical research program could offer tremendous potential opportunity for patients, including those in the ALS and diabetes communities.”

In connection with the financing, Arman Gupta, Principal at BVF, was named to the Anelixis Board of Directors.

Arman Gupta commented: “We are very excited by our investment in Anelixis. AT-1501 has the potential to provide meaningful benefits to patients across a range of autoimmune and neurodegenerative diseases. It is a privilege to help advance this compelling and differentiated program.”

There is a significant body of preclinical and clinical evidence for blocking activation of the CD40/CD40L pathway to induce allograft tolerance, decrease immune mediated toxicities to multiple cell types and organs in the context of autoimmune diseases, and reduce neuroinflammation in neurodegenerative diseases. AT-1501 was designed to mitigate the safety issues observed with previous anti-CD40/CD40L antibodies.

AT-1501 has received orphan drug designation from the U.S. Food and Drug Administration as a potential treatment in slowing the progression of ALS.

About AT-1501

AT-1501 is an engineered, humanized anti CD40L antibody that lacks FC effector function and blocks costimulatory signaling between lymphocytes and antigen presenting cells. Blocking CD40L signaling in preclinical models of organ transplant induces long term tolerance in multiple species and ameliorates disease progression in preclinical models of autoimmunity and preclinical models of neurodegeneration including ALS. Since its founding in 2015, Anelixis Therapeutics has optimized and validated a cGMP manufacturing process for AT-1501, confirmed exceptional activity in preclinical disease models, demonstrated safety and tolerability in multiple animal species, and has completed phase 1 studies in healthy volunteers and adults with ALS.

About ALS

Amyotrophic Lateral Sclerosis is a progressive neurodegenerative disease that is uniformly fatal, following a median survival of 3-5 years after diagnosis. Patients are most often diagnosed in their fourth or fifth decade of life, whereupon quality of life is dramatically impacted for patients and their families. ALS is an orphan disease with an incidence of approximately 5,000 newly diagnosed cases each year in the United States resulting in a prevalence of about 30,000 cases. Fewer than 10% of ALS cases are genetically inherited — often termed familial ALS (FALS). The remaining 90% of ALS cases are sporadic (SALS) with the etiology or cause of the disease unknown. The mechanism of action of AT-1501 is anticipated to be relevant to all types of ALS.

About Type 1 Diabetes

Type 1 Diabetes (T1D) is a T cell-mediated autoimmune disease associated with progressive loss of insulin producing beta cells in the pancreas, decreased insulin production, and uncontrolled hyperglycemia. Loss of beta cells is mediated by autoantibodies against beta cell antigens and the infiltration of pro-inflammatory lymphocytes, dendritic cells and macrophages. There is no cure for T1D with current therapeutic strategies designed to manage insulin levels and maintain normal glucose levels via either exogenous insulin supplementation or replacement of insulin producing islet cells. An estimated 40,000 new cases of T1D are diagnosed annually in the US, and a prevalence of 1.2 million people. Of these, an estimated 200,000 people are less than 18 years of age. In the last decade, the incidence of T1D has risen by 21 percent, with a projected prevalence of five million Americans with T1D by 2050.

About Anelixis Therapeutics

Anelixis Therapeutics, Inc. is a clinical stage, privately held biotechnology company developing treatments for patients with neurodegenerative disease, people requiring an organ or cell based transplant, as well as people with an autoimmune disease. For more information, please visit the company’s website at www.AnelixisTherapeutics.com.

Anelixis Therapeutics’ Forward Looking Statements

This press release contains forward-looking statements, all of which are qualified in their entirety by this cautionary statement. Any statements contained herein that do not describe historical facts are forward-looking statements that are based on management’s expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcomes, timing and performance to differ materially from those expressed or implied by such statements. These factors, risks and uncertainties include, but are not limited to: the costs and uncertainties associated with our research efforts and other discovery activities; the inherent uncertainties associated with the conduct, timing and results of preclinical and clinical studies of our product candidates; and the adequacy of our capital resources and availability of additional funding. Except as otherwise noted, these forward-looking statements speak only as of the date of this press release, and we undertake no obligation to update or revise any of such statements to reflect events or circumstances occurring after this press release. We caution readers not to place undue reliance on the forward-looking statements contained in this press release.

The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for the uses being investigated. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

Contacts

Anelixis Therapeutics

Steven Perrin

[email protected]