Avila Therapeutics™, Inc., a biotechnology company developing novel targeted covalent drugs, announced that it has successfully completed two Phase 1a clinical studies for AVL-292, its orally-available, selective inhibitor of Bruton's tyrosine kinase (Btk). Avila presented summary results from the first-in-human study, AVL-292-001, at the Keystone Symposium on Molecular and Cellular Biology: Evolving Approaches to Early-Stage Drug Discovery.

The study AVL-292-001 was a double-blind, placebo-controlled, single ascending dose study in healthy volunteers, and in this study AVL-292 demonstrated favorable safety, tolerability, and pharmacokinetics. In addition, the trial used Avila's unique covalent probe technology to assess the quantitative relationship among dose level, systemic exposure, and occupancy of the target by AVL- 292. This combination of analyses provides a powerful and rigorous understanding of AVL-292 action at the molecular level and serves as a rational guide to future clinical development. Clinical development of AVL-292 has been supported, in part, through an alliance with The Leukemia & Lymphoma Society (LLS).

"Over 50 healthy volunteers participated in these two trials, and based on these early and encouraging results, we are actively advancing the clinical development of AVL-292, initially for the treatment of B cell cancers, with a Phase 1b study expected to initiate in mid-2011," said Katrine Bosley, Chief Executive Officer of Avila. "The successful completion of this first clinical study is an important milestone for Avila and our covalent drug platform, as we demonstrate positive data and progress with our covalent drug candidate, AVL-292."

"LLS's goal in establishing our collaboration with Avila a year ago was to help accelerate the development of AVL-292 as a treatment for hematological malignancies. We've been impressed by the speed with which Avila has been able to advance the Btk covalent drug program to this point in development," said Louis DeGennaro, Ph.D., LLS Chief Mission Officer. "Patients with B-cell cancers need new treatment options and it is our sincere hope that AVL-292 will be successful in future clinical studies."

In an oral presentation at Keystone titled "Bruton's Tyrosine Kinase from Bench to Bedside: Covalently Silencing B Cells with AVL-292", Juswinder Singh, Ph.D., Co-Founder and Chief Scientific Officer at Avila Therapeutics, presented data from the first clinical trial of AVL-292.

Highlights of the data presented include:

- AVL-292 was shown to be generally safe and well tolerated

- AVL-292 was administered to subjects in all five planned dose cohorts

- AVL-292 was well-absorbed with good systemic exposure and demonstrated a dose-proportional pharmacokinetic profile across all dose levels (ranging from 0.5 to 7.0 mg/kg) with low inter-subject variability

- AVL-292 achieved statistically significant levels of target occupancy at all dose levels, with >80% Btk target site occupancy achieved at doses as low as 1.0 mg/kg

About AVL-292 and Bruton's Tyrosine Kinase (Btk)

AVL-292 is a novel, orally available, covalent drug that targets Bruton's tyrosine kinase (Btk). Inhibition of Btk is a promising new approach to treatment of diseases that are driven by B cells, including certain cancers and autoimmune diseases. AVL-292 selectively and covalently binds to Btk to inactivate and silence its activity. This unique mechanism of action confers greater target selectivity and a longer duration of action than is typical of conventional small molecule drugs. In preclinical studies, AVL-292 selectively and potently inhibited Btk and B cell receptor signaling in vitro and was efficacious in a variety of animal disease models. AVL-292 is in clinical development and has successfully completed two Phase 1a clinical studies to date.

Btk plays a crucial role in the activation of normal B lymphocytes and contributes to the proliferation and survival of B cells in certain cancers. AVL-292, with its ability to irreversibly inhibit and silence Btk, may represent a new best-in-class therapeutic option for the treatment of B cell-related hematological cancers, including non-Hodgkin lymphoma (NHL) and B cell chronic lymphocytic leukemia (B-CLL), as well as autoimmune diseases such as rheumatoid arthritis where B cells are known to contribute to the disease pathology.

Source:
Avila Therapeutics™, Inc.

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