National Heart Lung and Blood Institute

The Office of Translational Alliances and Coordination (OTAC) at the National Heart, Lung, and Blood Institute supports the development of innovative biomedical products to address unmet medical needs in the heart, lung, blood and sleep fields.  OTAC is home to the NHLBI's small business programs (SBIR and STTR) and the NIH Centers for Accelerated Innovations.

The NHLBI's SBIR and STTR programs comprise one of the largest sources of early-stage capital for U.S. small business, acting as engines of innovation for developing and commercializing novel technologies and products that aid in the prevention, diagnosis, and treatment of heart, lung, blood, and sleep diseases and disorders. The NHLBI provides grant and contract funding opportunities and resources to support small businesses performing research and development on technologies related to the mission of NHLBI. With an annual budget of $85M, the NHLBI funds about 200 companies each year through the Institute's small business programs.

The NIH Centers for Accelerated Innovations accelerate translation of scientific discovery into commercial products that improve health for patients. This unique public-private partnership is changing the way discoveries with scientific and commercial potential are identified and developed.

Dr Kurt Marek
Dr Kurt Marek
LinkedIn logo Deputy Director OTAC 
Dr Gary Robinson
Dr Gary Robinson
LinkedIn logo Business Development Specialist 

New Leaf Venture Partners

New Leaf Venture Partners is a venture capital company formed in 25 with offices in New York and San Mateo California. The firm currently manages two funds with its most recent fund having closed at $45 million in 27. The firm is looking to make equity investments ranging from $1-$25 million over the lifetime of the investment. The firm looks for companies primarily in the United States and some select opportunities in Europe. The firm plans to make between 1-5 investments over the next year.
Mike Dybbs
Principal 

Novo Nordisk

Novo Nordisk was founded in 1923 and is based in Bagsvaerd Denmark with additional R&D sites in Seattle WA Beijing China and Bangalore India. The firm is actively seeking to in-license assets and also forms research collaborations and co-marketing and distribution partnerships with other biotech companies. Novo Nordisk is focused on developing protein and peptide therapeutics and protein delivery devices. The firm has also in-licensed antibodies. Indications of interest include metabolic disorders blood and immune disorders growth disorders and inflammatory disorders. Within the inflammation fieldthe firm is particularly targeting therapies based on cytokines and on molecules on the surface of cells involved in immune regulation. The firm is interested in certain orphan conditions. In addition to partnering and in-licensing Novo Nordisk has a corporate venture fund Novo AS. The firm also offers research grants and resources for studies into Novo Nordisk's products.
Charles Gray
Sourcing Director 
Tomas Landh
Director 

NYU Office of Therapeutics Alliances

The NYU Office of Industrial Liaison (OIL) promotes the commercial development of NYU technologies into products to benefit the public, while providing resources to the University to support its research, education, and patient care missions. NYU OIL also facilitates research collaborations between NYU researchers and industry on projects of mutual interest.

In 2013, NYU launched the Office of Therapeutics Alliances (OTA). OTA is a nimble, "virtual biotech" approach to advance novel therapeutic projects by playing on the strengths of NYU in dissecting disease pathways and those of external, professional capabilities in early stage R&D. OTA identifies NYU projects with potential for addressing unmet needs, delineates the path to therapeutic proof of concept and assembles internal and external resources tailored to each specific project’s needs to maximize the likelihood of successful partnerships with biopharma, new biotech startups or disease foundations. 

Nadim Shohdy
Director, Drug Discovery Partnerships 
Sunil Shah
Partner 
Prashant Shah
Partner 

Orphagen Pharmaceuticals United States

Orphagen’s focus is small molecule discovery at novel drug targets. We create programs leading to first-in-class drugs. Our goal is to partner these with development stage pharmaceutical companies.

We have been first mover in creating three discovery programs for novel targets, including one program that initiated Phase 1 clinical trials with a strategic partner (JT Pharma) in 2013 for autoimmune disease.

We work with novel drug targets from a very productive target class, the nuclear receptors.

Our work has so far been funded by $15 M in federal grants and partnership revenue. Orphagen is in the process of raising equity funding to accelerate current programs.

Orphagen’s lead internal program is for retinitis pigmentosa, the major form of hereditary blindness. Closely following are antagonists to SF-1, a promising target for treatment of Cushing’s syndrome, a life-threatening endocrine disorder, and two cancers with an endocrine connection: prostate cancer and adrenocortical cancer. New target screening may lead to first-in-class programs for glioblastoma, sickle cell anemia, and cancer immunotherapy.

Website:
www.orphagen.com
Year Founded
2000
Biotech Subsector
Biotech Phase of Development
Technology Overview
Orphagen targets unexplored or “orphan” nuclear receptors (NRs), members of a receptor family that includes targets for major breast and prostate cancer drugs and for anti-inflammatory glucocorticoids. Approximately half of the 48 known NRs are targets for drugs on the market or new compounds approaching FDA approval. These marketed drugs generate in excess of $10 billion in annual U.S. sales alone. The remaining nuclear receptors, the orphans, have potential for design of first-in-class drugs. Orphagen has proprietary methodology, experience and knowhow for identifying drug-like small molecules that turn these receptors on and off. Orphagen’s work with these orphans is based on close relationships with academic laboratories.
Alliance & Collaborations
In 2003-6, Orphagen developed a first-in-class drug discovery program for ROR-gamma, a new target for autoimmune diseases such as psoriasis, rheumatoid arthritis and steroid-resistant asthma. Orphagen licensed its discovery-phase ROR-gamma program to JT Pharma in 2008. In 2013, JT Pharma initiated the first Phase 1 clinical trial for this target. BMS, Merck, Pfizer, Janssen, and Amgen also started biotech partnerships for the same target, but not until 2 to 5 years after JT-Pharma and Orphagen. Orphagen has demonstrated ability as a first mover for novel drug targets.
Supporting Metrics or Evidence
Approximately 50% of all industry partnership deals are done early, at a preclinical stage. Upfront payments for first-in-class drugs can reach $10-15 M and have grown substantially in the last 15 years. Combined later milestones are greater: $100 M to $300 M. First-in-class programs, such as Orphagen’s, are likely candidates for accelerated approval by the FDA, which markedly increases their potential for appreciated value. Orphagen plans to partner its discovery and development programs one by one as they mature. Partnership revenue will be used to expand R&D in Orphagen’s discovery pipeline and, when sufficient, to return capital to investors.
Current Financing Needs
$3 M to accelerate the Orphagen program for retinitis pigmentosa and to support a backup program (SF-1). Orphagen continues to receive significant non-dilutive funding from its ongoing partnership, from grants, and from sale of non-proprietary assay services.
Current Timeline
Orphagen expects to have advanced preclinical proof-of-principle data for retinitis pigmentosa and to execute a value-generating partnership for this indication in 2016. This is Orphagen’s most advanced internal program. Additional lead stage partnerships are anticipated in 2017 and beyond. Orphagen is also exploring revenue-generating partnerships in 2015-2016 for new target screening.
Current Investors
Friends and family, only. Orphagen has a single class of stock, Common stock.
IP Status
Orphagen has filed broad use patents for all ligands to two of its major targets where it has been the first to identify small molecule agonists or antagonists. Its screening technology and lead compounds, which are new chemical entities, are maintained as trade secrets.
Recent Milestones
2010-14 Phase 1 SBIRs for: (i) prostate cancer; (ii) endometriosis; (iii) glioblastoma; (iv) retinitis pigmentosa (RP); and (v) Cushing’s syndrome. 2013 File broad use provisional patent application on novel target for RP. 2013 JT Pharma starts Phase 1 clinical trial based on Orphagen strategic partnership. 2014 Structural biology collaboration for SF-1 with pharmaceutical partner. 2014 Initial proof-of-principle for treatment of RP in an animal model of disease. Identification of target co-crystal structure.
Management Team Highlights
Scott Thacher, Ph.D. Founder, CEO and CSO. Investigator for 15 SBIR grants to Orphagen. Concluded Orphagen’s licensing deal with Japan Tobacco. Previous: Investigator at Allergan. Co-founder: Io Therapeutics. Judy Blakemore. Director Business Development. Former interim COO at Onyx. Business development and transactional advisor for small to mid-size biotechnology companies since 1993. Paul Crowe, Ph.D. Director of Biology, Former Senior Director Pharmacology at Neurocrine. Ruo Steensma, Ph.D. Director of Chemistry, Former Director at Structural Genomix.
Dr Scott Thacher
LinkedIn logo CEO 
BIO

Scott Thacher, Ph.D., CEO and Director, founded Orphagen in 2001.  He has 30 years of experience in life sciences research and pharmaceutical R&D and led Orphagen to its first partnership in 2008. Prior to founding Orphagen, Scott directed programs in acne, psoriasis, hyperlipidemia, and diabetes at Allergan. Scott was previously on the biochemistry faculty at the Texas A&M College of Medicine (1986-1993) and was a Staff Fellow at the NIH. He holds a Ph.D. in biophysics from Harvard University and a B.S. in physics (Stanford).

Pappas Ventures

Pappas Ventures is a Venture Capital company based in Durham North Carolina founded in 1994. The firm manages 4 funds for a total of approximately $35 million in assets under management. The firms is currently investing out of its $1 million 4th fund focused exclusively on the life sciences. The firm looks to provide up to $5 million of preferred equity capital in the initial round and up to $1 million over the life of the investment. The firm is very flexible in terms of period to exit but generally looks to exit in around 5 years. The firm plans to make 2-3 investments over the next 6-9 months and will consider companies throughout North America.
Jayson Punwani
Associate 

PBM Capital Group

PBM Capital is a hybrid family office and operating company based in Charlottesville Virginia with an additional office in New York City. The firm is funded and led by the successful life science entrepreneur Paul Manning who started and sold several companies in his career. The firm can make investments ranging from approximately $5 to $1 million into companies and due to its funding structure has no requirements for holding period or capital structure. The firm makes investments in the forms of equity controlling interest in-licensing MBO/LBO growth capital and is also willing to co-invest. The firm is looking for companies located around the globe and makes around 5-15 investments in a given year.
Jayson Rieger
SVP of Business Development & Portfolio Management 

Pfizer Venture Investments United States

Pfizer Venture Investments (PVI) is the corporate venture capital arm of Pfizer and was founded in 2004. PVI has an annual investments budget of $50 million and invests up to $10M per investing round. The firm focuses mainly on U.S. startups but has global reach. PVI attempts to allocate 80% of its funding to U.S. based companies and utilizes the remaining 20% for international ventures. PVI provides equity funding for private companies in need of seed, growth, or venture financing. Remaining opportunistic, PVI focuses entirely on high growth prospects in all sectors and all phases of development. The ideal candidate has a potential for high growth and returns. Additionally, PVI will seek to in-license products and buyout companies if the opportunity arises.

Barbara Dalton
Vice President 

Plug and Play Tech Center United States

Plug and Play Ventures (a successor fund to Amidzad Partners) is a private/family investment vehicle based in Silicon Valley, CA. The fund is a structured organization for making angel investments in pre-seed or seed rounds. Investments are in the form of equity; in the next 6 months Plug and Play Ventures expects to make about 5-10 seed investments of $50,000-100,000 and an additional 40 pre-seed investments of $25,000, and is hoping to increase their allocations in the healthcare sector. Plug and Play also provides a three-month accelerator program. The firm will consider investing in companies worldwide.

Plug and Play Ventures is interested in investing in medical technology, and invests in subsectors in which the firm can apply expertise; typically this means products that have a significant software component. Healthcare IT, biosensors, wearables and health monitoring devices are of interest. While the firm is open to investing in any indication, Plug and Play is particularly interested in diabetes & blood glucose monitoring, personal fitness, and mental/behavioral disorders. In the healthcare IT sector, Plug and Play Ventures is interested in both consumer applications and enterprise software, but is not interested in diagnostic software such as genomic, proteomic, or molecular diagnostic algorithms; however, optimization and data analysis software for hospitals and diagnostic laboratories is of interest.

Phillip Vincent
Corporate Partnerships Manager