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).

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 

Pharmatek United States

Pharmatek is a contract development & manufacturing company providing dosage form development & cGMP manufacturing of oral, injectable & topical products. Founded in 1999, our services focus on the rapid advancement of small molecule & peptide drug candidates from the bench to the clinic & include:

·         Formulation & Analytical Development

·         cGMP Manufacturing

·         Clinical Packaging, Labeling & Worldwide Distribution

 

Our experience includes first-in-man strategies, solutions for poorly soluble compounds, controlled release formulations & separate facilities for the handling of cytotoxic & potent compounds.  Pharmatek’s drug delivery technologies include:

·         Solid Dispersions

·         Particle Size Reduction

·         Lipid Delivery

·         Complexation

·         Lyophilization

·         Suspensions & Emulsions

Pharmatek’s 68,000 sq. ft. facility includes 9 class 100,000 cGMP manufacturing suites, formulation & analytical laboratories, & ICH stability storage.  Pharmatek has over 150 clients globally, ranging from virtual to large pharmaceutical companies. Having manufactured product for clinical trials in the North America, Europe & Asia; Pharmatek has successfully completed several large pharma quality, EH&S & QP audits.

Mr Tim Scott
Mr Tim Scott
LinkedIn logo 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 

Plug and Play Ventures 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.


Neda Amidi
Investment Associate 

Prolog Ventures United States

Prolog Ventures is a venture capital firm based out of St. Louis Missouri that was founded in 2001. The firm is currently making investments out of its vintage 2013 4th fund of approximately $100 million. The firm is looking to make equity investments in companies ranging from $500,000 to $3 million. The firm will invest in companies across the United States and plans to make approximately 3-4 investments over the next 6-9 months.

Brian Clevinger
Managing Director 

Recursion Pharmaceuticals United States

The Problem: Pharmaceutical development has traditionally focused on intense study of an explicit molecular target related to a specific disease of interest. This strategy is costly and inefficient.

 

The Solution: We have developed technology that can be scaled to quickly, precisely, reliably, and simultaneously model thousands of genetic diseases in human cells and evaluate the effect of thousands of individual drugs on those disease models. We've built a computational platform that recognizes structural changes in millions of diseased cells and then identifies drugs that return those diseased cells to a healthy state.

 

Proof of Concept: We have already used an early version of this platform to discover a potential treatment for one genetic disease. We have IP for this drug, and have already been approached about licensing.  We are scaling our platform now to enable us to achieve our goal of discovering and partnering to bring to market treatments for at least 100 genetic diseases in 10 years.

Dr Chris Gibson
LinkedIn logo CEO, Recursion Pharmaceuticals 

RiverVest Venture Partners United States

RiverVest Venture Partners is a venture capital firm that was founded in 2000 and is based in St. Louis, Missouri with additional office in Cleveland, Ohio. The firm currently has managed over $208 million of total assets. RiverVest has raised two investment funds with total committed capital of $165 million since inception. The recent fund closed at $75 million. RiverVest typically makes equity investments into global companies, and has no specific geographic preferences. The firm will consider making investments at all the stages, and focuses on investments in seed, early-stage and select later-stage companies. The typical investment size is around $6 million, though the firm can invest more or less, depending on the opportunity. 

RiverVest is extremely opportunistic when it comes to investments in the life sciences space; with that being said RiverVests specified sectors and sub sectors of interest may or may not be an area in which RiverVest is currently looking to allocate capital to. The firm invests in companies in the biotech therapeutics and diagnostics, as well the medical technology space. 

Some of the firms investments have included companies developing biotech therapeutics and diagnostics targeting diseases of the blood and blood forming organs, neoplasms, cancer, and oncology, skin and subcutaneous tissue, cardiovascular diseases, diseases of the ear, and infectious diseases. The firm has also invested in companies developing anti-body based therapeutics. The firm has also invested in firms developing medical technologies such as therapeutic radiation devices, active implantable devices, non active implantable devices, hospital hardware, and imaging devices.

Year Founded
2000
Investor Type
Medtech Phase of Development
Capital Structure Preference
Investment Stage Preference
Karen Spilizewski
VP