Anelixis Therapeutics Announces the Initiation of a Phase 1 Study of AT-1501, an anti CD40L Antibody

Immune Tolerizing Treatment Could Benefit ALS As Well As Other Neurodegenerative and Autoimmune Disease and Transplant Patients

November 28, 2018 10:00 am EST

CAMBRIDGE, Mass. — Anelixis Therapeutics C Corp, a biotechnology company focused on the development of anti-CD40 ligand (CD40L) antibodies to address serious unmet medical needs in patients with neurodegenerative disease including amyotrophic lateral sclerosis (ALS, or Lou Gehrig disease) and autoimmune disease, and people requiring an organ or cell-based transplant, today announced that  it has initiated a Phase 1 clinical trial of Anelixis’ AT-1501 safety-engineered anti-CD40 ligand monoclonal antibody, following allowance of an Investigational New Drug (IND) application. by the U.S. Food and Drug Administration (FDA)  Anelixis’ Phase 1 clinical trial is evaluating the safety and pharmacokinetics of AT-1501 in healthy volunteers and will also enroll patients  with ALS at the Massachusetts General Hospital (Boston, MA).

Anelixis Therapeutics entered into an exclusive licensing agreement with the ALS Therapy Development Institute (ALS TDI) in 2015 to develop AT-1501 and other anti CD40L antibodies as a potential treatment for ALS and other disease indications. Research results previously published by ALS TDI have implicated an autoimmune mechanism in the disease progression of patients with ALS, which is blocked by the immune modulatory effects of anti-CD40 ligand monoclonal antibody therapy.  Current treatments for ALS and other neurodegenerative disorders such as Alzheimer’s disease provide only limited benefit and generally do not halt disease progression.  The FDA granted orphan drug designation for the investigational antibody, AT-1501, for ALS in April 2018. Substantial preclinical data also highlights the relevance of blocking CD40L in autoimmune disease and in solid organ and cell based transplantation models.

“The advancement of AT-1501 into clinical development is a major milestone for Anelixis, but more importantly for people living with ALS and other devastating neurodegenerative or autoimmune diseases, including for example Type I Diabetes patients no longer able to monitor their glycemic index and requiring transplant surgery, said Steven Perrin, PhD., President and Chief Executive Officer of Anelixis Therapeutics. “The preclinical data for blocking CD40L is very compelling in preclinical models of ALS and in kidney transplantation and in pancreatic islet cell transplantation to treat Type 1 diabetes. This is an exciting opportunity for Anelixis to translate these preclinical studies into a potentially significant health benefit for many thousands of people with devastating diseases.”

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, 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, and demonstrated safety and tolerability in multiple animal species in preparation for clinical development activities.

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. The rates of incidence and the prevalence are approximately the same globally. Fewer than 10% of ALS cases are genetically inherited — often termed familial ALS (FALS). More than twenty different genes have been associated with familial ALS with the majority of families having mutations in C9ORF72, SOD1, TDP43, or FUS1. 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 Anelixis Therapeutics

Anelixis Therapeutics is a clinical development company focused on developing effective treatments for serious unmet medical needs for patients with autoimmune diseases, neurodegenerative disease, and people requiring an organ or cell based transplant. 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.