Financing provides for the development of Lumos Pharma's lead compound, a novel treatment for Creatine Transporter Deficiency

AUSTIN, Texas, Feb 4, 2014 – Lumos Pharma, an early stage biopharmaceutical development company, announced today that it has raised $14 million in Series A financing. The round was co-led by Santé Ventures and New Enterprise Associates (NEA). Lumos Pharma will use the financing to support the preclinical and clinical development of its lead compound, LUM-001. LUM-001 is a small molecule therapeutic for the rare disease Creatine Transporter Deficiency (CTD), an inborn error of metabolism. In preclinical studies LUM-001 has shown disease modifying potential as a treatment for CTD.

“We are extremely pleased to announce the closing of our Series A financing round from Santé Ventures and NEA,” says Lumos Pharma’s Founder, President, and CEO Rick Hawkins. “Santé and NEA’s deep knowledge of drug development in the rare disease space will be valuable assets to our company and to the development of our lead compound. We are absolutely committed to bringing a much needed therapeutic to the patients afflicted with CTD and their families. The patients remain our first priority.”

Lumos Pharma’s lead compound, LUM-001 has been granted orphan designation by the FDA. The project has garnered the attention and support of the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH). Lumos Pharma and NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program researchers are collaborating on the study and development of this potential treatment for CTD. Through its TRND program, NCATS provides in-kind support for rare disease drug development capabilities, expertise, and clinical and regulatory resources.

“We are very grateful for the ongoing collaboration with TRND staff, and together with our new financial partners, Lumos is in an excellent position to succeed,” says Mr. Hawkins. “TRND’s dedication and contribution toward the development of therapies for rare diseases is nothing short of astounding.”

Creatine Transporter Deficiency (CTD) is an inborn error of metabolism caused by a defect in the x-linked creatine transporter SLC6A8 gene, and falls within the general descriptive category of an Autism Spectrum Disorder. A defect in the SLC6A8 gene causes a defect in the transport of creatine across the blood-brain barrier, resulting in a near complete absence of cerebral creatine concentrations. The primary clinical manifestations of the disorder are x-linked mental retardation and global developmental delay, severe expressive speech and language delay, autistic behavior, and epilepsy. CTD is a completely unmet medical need. Patient registries are available.

Lumos Pharma is led by an experienced management team, including CEO Rick Hawkins, a 30-year industry veteran with a strong track record in the rare disease space, having successfully gained marketing approval for multiple orphan drugs (including Somavert®) and Chairman Jon Saxe, formerly of Hoffmann-LaRoche, Inc., and Protein Design Labs, Inc. Associated with the Series A financing, Kevin Lalande, Managing Director, Santé Ventures, and Ed Mathers, Partner, NEA, will join the Board of Directors.

“Rick and his team have made terrific progress in the development of this promising new therapeutic agent over the last two years since we first started following the company,” says Mr. Lalande. “Santé Ventures is pleased to help provide the equity required to advance this drug through clinical trials and closer to widespread commercial availability for this important patient population.”

“We are delighted to be working with Rick, the Lumos team and our partners at Santé Ventures on this important product. CTD has no current treatment options for the patients who suffer from this disease, and the work that Lumos has done gives hope for a future therapy,” says Ed Mathers, Partner at NEA.

Lumos Pharma intends to immediately initiate the preclinical and clinical development of its lead compound with the goal of receiving marketing authorization from regulatory bodies worldwide.

ABOUT LUMOS PHARMA

Lumos Pharma is focused on bringing novel therapies to patients afflicted with unmet medical needs in severe, rare, and genetic diseases. Lumos Pharma is led by an experienced management team with longstanding experience in the rare disease space. Lumos Pharma’s lead compound is supported by the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases program. Lumos Pharma is located in Austin, Texas. Please visit the company’s website at: www.lumos-pharma.com.

ABOUT SANTÉ VENTURES

Santé Ventures is a life sciences venture capital firm that invests exclusively in early-stage companies developing innovative new medical technologies or healthcare delivery models. The firm was founded in 2006 and has $260 million in capital under management in two funds. For more information, visit www.santeventures.com.

ABOUT NEW ENTERPRISE ASSOCIATES

New Enterprise Associates, Inc. (NEA) is a leading venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors and geographies. With approximately $13 billion in committed capital, NEA invests in information technology and healthcare companies at all stages in a company’s lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 175 portfolio company IPOs and more than 300 acquisitions. In the U.S., NEA has offices in Menlo Park, CA; New York, NY; Chicago, IL; Boston, MA; and the Washington, D.C. metropolitan area. In addition, New Enterprise Associates (India) Pvt. Ltd. has offices in Bangalore and Mumbai, India and New Enterprise Associates (Beijing), Ltd. has offices in Beijing and Shanghai, China. For additional information, visit www.nea.com.

For further information or inquires, please contact info@lumos-pharma.com