ROAD TO THE CURE UPDATE NOVEMBER 2016 · Parkinson's Resource Organization

ROAD TO THE CURE UPDATE NOVEMBER 2016

Category: Road to the Cure

ICBI SMART Molecules (SMs) technology is poised to translate our most promising scientific breakthroughs into meaningful disease altering treatments capable of tackling the most complex and vexing medical challenges related to the diseases of the central nervous system (CNS). In spite of the rapid pace of our initial scientific advances, the recent budgetary constraints have slowed down the progress: manufacturing and the market penetration of our potential diagnostic and disease altering drugs for Parkinson’s and Alzheimer’s diseases. The process of drug development is complex filled with scientific, technical, financial, and regulatory challenges. The average cost (capitalized) to successfully develop a drug from start to finish (FDA Approval) in the US was estimated to be $2.6 Billion in 2014 (reference: Tuffs Center for the Drug Development, November, 2014). If one takes into account the time and resources wasted on failed drugs during the same time by the same group of scientists who successfully launched their drug in the market, the cost exceeds $4 Billion Last month we covered drug development through Filing for an Investigational New Drug (IND) Application with the FDA; this month we start with Clinical Trials:

Phase 1 Clinical Trial: INITIAL SAFETY TESTING IN A SMALL GROUP OF HEALTHY VOLUNTEERS

In Phase I trials the candidate drug is tested in people for the first time. These studies are usually conducted with a small number of healthy volunteers, generally 100 or less. The main goal of a Phase I trial is to assess the safety of the medicine when used in humans. Researchers look at the pharmacokinetics of a drug: How is it absorbed? How is it metabolized and eliminated from the body? They also study the drug’s pharmacodynamics: Does it cause side effects? These closely monitored trials are designed to help researchers determine what the safe dosing range is and if the candidate medicine should move on to the next stage of development. Average Time for Phase 1 Clinical Trials 9 years Average Pharma Cost up to this point $810M.

Phase II Clinical Trial: ASSESS SAFETY AND EFFICACY IN A SMALL GROUP OF PATIENTS

In Phase II trials we evaluate the candidate drug’s effectiveness in 100 to 500 patient volunteers with the disease or condition under study. Many Phase II trials study patients receiving the drug compared with patients receiving a different treatment, either an inactive substance (placebo), or a different drug that is usually considered the standard of care for the disease. Researchers also analyze optimal dose strength and schedules for using the drug and examine the possible short-term side effects (adverse events) and risks associated with the drug. If the drug continues to show promise, they prepare for the much larger Phase III trials. Average Time for Phase 1 Clinical Trials 10 years Average Pharma Cost up to this point $1.2Billion

Phase 3 Clinical Trial: DEMONSTRATE SAFETY AND EFFICACY IN A LARGE GROUP OF PATIENTS

Phase III trials generate statistically significant data about the safety, efficacy and the overall benefit-risk relationship of the investigational medicine. Phase III trials may enroll 1,000 to 5,000 patients or more across numerous clinical trials sites around the world. This phase of research is essential in determining whether the drug is safe and effective. It also provides the basis for labeling instructions to help ensure proper use of the drug (e.g., information on potential interactions with other medicines, specific dosing instructions, etc.) Phase III trials are both the costliest and longest trials, often encompassing hundreds of study sites at hospitals and centers both across the U.S. and around the world. Coordinating all the sites and the data coming from the clinical trial sites is a monumental task. Companies must coordinate closely with staff at each trial site, as well as with the IRB/EC that is monitoring the study and the FDA. Often, a clinical research organization (CRO) will work with a company to aid in recruitment and day-to-day operations of the trial. In addition, the company’s manufacturing scientists are working to ensure high quality production of the medicine for use in the trials, as well as planning for the full-scale production of the medicine after approval. Meanwhile, the company is working to assemble and prepare the complex application required for FDA approval. Average Time for Phase 3 Clinical Trials 12.5 years Average Pharma Cost up to this point $2.56 Billion.

FDA Review and Approval of Marketing ApplicationEVALUATION OF COMPLETE DATA SETS AND PROPOSED LABELING AND MANUFACTURING PLANS

After determining that the results of the clinical trials indicate the drug is both safe and effective, ICBI will submit a new biologics license application (BLA) to the FDA requesting approval to market the drug. This application will contain the results and data analysis from the entire clinical development program, as well as the earlier preclinical testing and proposals for manufacturing and labeling of the new medicine— which can run 100,000 pages or more. In order to accelerate the availability of medicines to patients with serious diseases or where there is an unmet medical need, the FDA implements expedited approaches to accelerate the development and review of new medicines, such as:  Fast Track: expedites the review of drugs that treat serious conditions and fill an unmet medical need;  Breakthrough Therapy: expedites the development and review of drugs that may demonstrate substantial improvement over available therapy;  Accelerated Approval: accelerates approval for drugs that address a serious condition or fill an unmet medical need, based on a surrogate or an intermediate clinical endpoint; Priority Review: accelerates FDA evaluation of drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. Scientists, physicians and statisticians at the FDA review the data from all of the studies on the compound and, after weighing the benefits and risks of the potential medicine, decide whether to grant approval. Occasionally the FDA will ask for additional research before granting approval or convene an independent expert advisory panel to consider data presented by the FDA and the company.

Note from Jo Rosen: I invite you to contact me if you want more information about supporting The Parkinson’s ROAD TO THE CURE. I would be proud to make the important introduction of you to the scientists at ICBI, and we continue to salute these scientists as they forge ahead!

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Updated: August 16, 2017