Corporate Overview This presentation contains forward-looking - - PowerPoint PPT Presentation
Corporate Overview This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts and referenced statistics, contained in this presentation,
This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts and referenced statistics, contained in this presentation, including statements regarding future company strategy, operations, financial position, revenues, projected costs, plans and objectives of management, will be forward-looking statements.
where all patients have a therapeutic option
) unmet needs of patients with rare diseases through the development of pharmaceuticals and medical devices, either independently
partnerships
) constantly be seeking new therapeutic innovations for patients with unmet needs
sourcing the next big breakthrough in rare disease treatments
Diseases (NORD) helps quantify the need for orphan drugs
affected by a rare disease. Some of these include:
National Organization for Rare Disorders, n.d., https://rarediseases.org/advocate/rareinsights/nord- rareinsights-rd-facts-2019-cover/ (Accessed 15 May 2020)ri0).
It’s estimated that 25-30 million Americans have a rare disease.
That’s 1 in 10 people More than 90%
an FDA approved treatment There are approximately
rare diseases
underlying genetic defects, which can be hard to identify1
that afflicted individuals might not be correctly diagnosed for many years, and there are instances
diagnosis2
years, which begins when the patent is issued
costly, which absorb much of the patent life:
pharma can commercialize a drug and therefore…
big pharma
the Orphan Drug Act of 1983 was a law passed to facilitate development
drugs for rare diseases
Drug is indeed “intended to treat a rare disease or condition.”
200,000 people in the country, or…
people in the country, but the FDA determines there is no reasonable expectation the company will exceed the costs of developing/producing the drug through sales
the early clinical testing stages
approval with the FDA
marketing exclusivity
patients sooner
exclusivity
clinical testing expense
✓ Affordable Care Act Pharmaceutical Fee ✓ Pediatric Research Equity Act Requirements (for non-oncology drugs) ✓ 340 (b) pricing
Mikulic, M. A. (n.d.). Orphan vs. non-orphan drugs pipeline sales forecast worldwide 2019-2024.
29 May 2020)
Pipeline sales forecast for orphan and non-orphan drugs worldwide from 2019 to 2024 (in billion U.S. dollars)*
Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020]. Note: Designations granted in a given year may include requests received from that year as well as previous years.
Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
roles/responsibilities: – Co-administered with Office of Pediatric Therapeutics – Grant special status (“RPD designation”) to products that meet eligibility criteria (prevalence less than 200,000; serious/life-threatening manifestations primarily affect those 18 years and under)
Voucher Program
marketing application for a different product
Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
therapeutic solutions can potentially be out-licensed. Some of these countries could include:
therapeutics principally based on collaborations and licenses with both academic and corporate partners
partnerships at all phases of development with:
✓Patient Advocacy Groups ✓Universities ✓Global Big Pharma ✓NIH ✓Charitable Foundations
development costs
guidelines for orphan drugs
(AKA non-diluted financing Asia/Europe)
transactions in the Orphan Drug Space
development field to establish robust board leadership
For the past two decades, Paul Michaels has established lasting relationships with key business leaders and stakeholders in Japan and the U.S., particularly in the Life Sciences field. He has completed transactions that influenced companies such as Celgene Corporation, Sumitomo Pharmaceuticals, Nobelpharma Co., Ltd, Teva Pharmaceuticals, ICOS, Neurocrine, Cell Therapeutics and Gilead, and more. Paul has also provided financial leadership for almost 18 years to Inabata & Co. Ltd., one of Japan’s largest trading companies with $4.5 billion in annual sales and successfully licensed multiple drugs from Gilead (formerly Nexstar Pharmaceuticals). Throughout his tenure, he structured and closed major transactions that brought high-value products to the Japanese pharmaceutical market in addition to other corporate responsibilities that included overall evaluation of corporate asset values. Paul subsequently founded Nobelpharma,
profitable orphan drug companies. As President of the U.S. division and a co- Founder, he secured $35 million in initial capital and negotiated the license for the first designated orphan drug product in the company’s pipeline from Teva Pharmaceuticals. As an adult onset Type 1 diabetic, Paul knows firsthand how a patient with a rare disease can struggle to get the right diagnosis and treatment. Only 40,000 adults a year are diagnosed with Type 1 diabetes. Paul is actively involved in the Juvenile Diabetes Research Foundation (JDRF), a global organization funding research for Type 1 diabetes.
Paul M. Michaels Interim CEO & Director
School-HBX Program
executives across industries, bringing expertise in strategy and performance in the pharmaceuticals and medical-products sector. He also consults with healthcare companies to develop global strategies and operating models and is an experienced leader with a demonstrated history of success working as an entrepreneur in the health and wellness
management, content development, video production, online marketing, sales, and patient education. Combined with an expansive network of contacts and an exclusive key opinion leader database throughout North America, Europe, Middle East, Asia, and South America, he brings consummate value to the team.
Samaritans and One Sight. He recently retired after practicing over 35 years in the medical field. He participated in four Externships including the Feinbloom Low Vision Center in Philadelphia, focusing on Age Related Macular Degeneration (AMD) and the Goldschleger Eye Institute in Tel Hashomer Hospital in Israel, focusing on diseases of the eye including Glaucoma.
Chief Strategy Officer
Minor in Economics – Yeshiva University
Optometry – Pennsylvania College
LaSalle College
A communications and marketing leader, Pam Bisikirski guided the brand development of multiple healthcare optical centers including America’s Best Contacts & Eyeglasses and Eyeglass World while working at National Vision, Inc, (EYE) a $1.7 billion company. During her 20-year tenure at National Vision she managed the public relations for the organization as well as multi-million-dollar partnerships and endorsement programs. Pam has also led communications as a consultant in the healthcare and retail spaces and is experienced with both start-ups and rapidly expanding companies. Pam sat on the Board of Directors of Prevent Blindness Georgia for 6 years, the maximum allowed by the charitable foundation that is dedicated to preserving sight. She continues to support the organization today.
Pam Bisikirski
Director of Communications
State University of New York at Geneseo
MICHAEL GRACE, PH. D.
Michael Grace has 30 years of executive and technical experience in both research and development of protein therapeutics and peptides within the biotechnology industry at Schering-Plough, Bristol Myers Squibb, NPS Pharma and Advaxis Inc. He has contributed to or personally led the development to commercial approval of 9 drugs by FDA and EMA including PEG-IntronTM, OrenciaTM, NulojixTM, YervoyTM, GattexTM and NatparaTM. His experience encompasses all aspects of drug development from discovery, process development, analytical development, quality control and regulatory. He is well-published within his field of expertise and has presented world-wide
RONALD BORDENS, PH.D.
Ronald Bordens is an accomplished executive scientist with over 40 years of successful experience in biotechnology and pharmaceutical industries for companies focused on research and development. He possesses a unique skill set including but not limited to people leadership, scientific expertise, and quality assurance and control. His pharmaceutical industry experience includes drug discovery, drug development and research technology with a focus on bioanalytical research, biomarkers, and leadership development from his 26-year career at Schering-Plough Research.
RICHARD GARR, ESQ.
Additionally, as a co-founder and General Counsel at Neuralstem, Mr. Garr had responsibility and oversight for all operations of a world- leading regenerative medicine company including worldwide business development, joint ventures, and licensing. and created the Stem cell industry’s first development agreements for Korea, Indonesia, Singapore, Malaysia, and Vietnam. In 2016. Mr. Garr became a full- time advocate for patient’s rights to access experimental treatments in development both in the U.S. and Europe. In 2018, he founded Access Hope CRO, the only CRO dedicated exclusively to facilitating safe, ethical, and legal expanded patient access to potentially life- saving investigational treatments under the Trickett Wendler, Frank Mongiello, Jordan McLinn and Mathew Bellina Right to Try Act of
Furthermore, he was a founder and is a current Board member of the First Star Foundation, a children’s charity focused on abused children’s issues; a founder of The Starlight Foundation Mid-Atlantic chapter, which focuses on helping seriously ill children; and a past Honorary Chairman of the Brain Tumor Society.