Rare disease specialist Atyr Pharma Inc. has completed a $76 million series E financing that it will use to further develop and advance its pipeline, led by an ongoing phase Ib/II trial of Resolaris (ATYR-1940) for patients with facioscapulohumeral muscular dystrophy (FSHD).

New investors Sofinnova Ventures and an undisclosed large institutional investor led the financing, while additional new investors taking part in the round include funds and accounts advised by T. Rowe Price Associates Inc.; Federated Investors Inc.; Deerfield; Rock Springs Capital Management; EcoR1 Capital; Sphera Global Healthcare and two additional undisclosed institutional investors. Current venture capital investors and a current public investment fund also took part in the raise.

In conjunction with the financing, the company is also adding Sofinnova general partner Srinivas Akkaraju to its board, building on his roster of current board seats at Intercept Pharmaceuticals Inc., Seattle Genetics Inc. and Versartis Inc.—all public companies. In a statement announcing the new round, Atyr chairman and CEO John Mendlein said Akkaraju was joining the company at an "important juncture," but the company said it was unable to provide further information immediately.

Akkaraju joins board members including Atyr founder and serial entrepreneur Paul Schimmel, as well as industry pioneers Ed Penhoet and James Blair.

The company has been bringing on additional talents to deepen its capabilities, adding veteran immunobiology researcher Fred Ramsdell as vice president of immunology in November and John McKew, former acting scientific director of the Division of Preclinical Innovation at the National Center for Advancing Translational Sciences in October.

Atyr is developing therapies based on physiocrines, a class of endogenous proteins that help re-balance the immune system for use in treating autoimmune diseases. The company contended that physiocrines, which are extracellular signaling regions of transfer ribonucleic acid (tRNA) synthetases, can promote restoration of stressed or diseased tissue to a healthier state.

From what it has shared to date, the company has a pipeline of at least three active programs, including Resolaris, Resokine and ATYR-1920, according to Thomson Reuters Cortellis Clinical Trials Intelligence. A fourth program, ATYR-0030, has no recent development reported.

The company's ongoing phase I/II trial of Resolaris is a double-blind, placebo-controlled, multiple-ascending dose trial in up to 44 adults with FSHD, a rare and severe genetic myopathy for which there are currently no approved treatments. The myopathy has an estimated prevalence of one in 20,000 people, according to the National Institutes of Health. However the Facioscapulohumeral Muscular Dystrophy Society, a patient organization that supports research and education about the condition, points to a Dutch study published in the Sept. 16, 2014, edition of Neurology that suggested the condition's prevalence may be much higher and is one of the most prevalent neuromuscular disorders.

In February, Atyr reported that it had secured an orphan drug designation for Resolaris from the EC. The company has said it believes Resolaris will provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.

Resokine, another physiocrine-based compound for the potential I.V. treatment of rare diseases including vascular and immune diseases rolled into a phase I study in February 2014. The double-blind, placebo-controlled study tested the potential therapy in 32 healthy volunteers in the EU in four dose cohorts. Data from the study supported the basic premise of Resokine I.V. as an immuno-modulator rather than a classic interleukin or interferon with systemic immuno-stimulating properties upon administration, the company said.

A preclinical physiocrine-based immunomodulator, ATYR-1920 (Homeokine), has also drawn the company's attention for the potential treatment of inflammatory bowel disease, immune disorders and rare genetic diseases, as has ATYR-0030 (Tmax), a resected variant (resectin) of human aminoacyl tRNA synthetase, but the company not reported further development of the program since 2011, according to Thomson Reuters Cortellis Clinical Trials Intelligence.

The San Diego-based biotech has raised about $172 million to date, including this latest round, according to BioWorld Today data. It landed an oversubscribed $49 million series D round in July 2013 funded by Alta Partners, Cardinal Partners, Doman Associates and Polaris Partners. Prior to that, it closed a $23 million series C round in 2010. (See BioWorld Today, July 29, 2013.)