Innovation Competition (Boston, MA)

The Phoenix Project

Therapeutics

Jim Jakway is the principal architect of this concept. He completed post docs in cellular immunology at the NIH and UC San Francisco. He then spent 20+ years in the inflammation drug discovery groups at the Schering Plough Research Institute and Abbott Labs. He is an expert in the cellular biology/pharmacology of inflammatory diseases. He has led multiple drug discovery project teams, taking projects from conception to the clinic.

A compound was taken to Phase IIb clinical trials for a major chronic inflammatory condition by a big pharma company. The trial was recently abandoned for undisclosed reasons. Based on the mechanism of action of the compound, I believe that the compound would be of use for treatment of an unrelated acute inflammatory condition. This condition currently has no good treatment options and results in significant morbidity, mortality and financial costs.

This condition develops in a fraction of a well defined patient population. The size of that population consists of about 400,000 patients annually in the US. While it is not possible to predict which patients will develop the condition, the condition develops at a predictable time in these patients. The mechanism of action predicts that the compound must be given before the condition develops. Thus, it is envisioned that the entire patient population would be treated. Treatment would involve a short course of therapy (1 - 5 dosings).

This solution takes a compound with an extensive clinical trials history (now abandoned) and redirects it to a new indication for which there is no effective treatment. The compound, if successful, would be a first-in-class solution for the indication. Because of the extensive clinical trials history, it should be possible to go straight to a Phase II trial with the compound (PIIb?). The solution envisions treating the entire susceptible population. Thus, patients that would not develop the condition would be dosed. However, the compound has been shown to be quite safe in humans, especially with short term dosing. This would in essence be an insurance policy against developing the condition. With appropriate pricing, the overall averaged cost to the entire susceptible population would be lowered significantly by this approach.

This will initially be a one-and-done approach: One specific compound in one indication. If it works, great. If it doesn’t work, the party is over. The philosophy of the team will be to travel fast and light. This will be mostly a virtual company, with most of the work being done by CROs. The envisioned clinical trial would be quick and relatively simple: Dose patients with a short course of therapy and observe them for 7 – 10 days for development of the condition (frequency and severity). The most likely end game, assuming success, will be to sell the asset to another company with the manufacturing and sales capabilities to take it to market.

This compound would be a first-in-class therapy for prevention of the target condition. No therapy exists for prevention of the condition. (Other compounds with different mechanisms of action have failed in trials.) Current therapy is limited to supportive therapy after the condition develops.

Pre-funding

The first milestone will be to actually form a company to go forward with the opportunity. (Currently, it is just me and my idea.) The major milestone for this opportunity will be to license the compound (and associated preclinical and clinical data) from the current big pharma owner. The challenge will be to do so at a reasonable cost relative to the potential market. (The company has stated a willingness to out-license programs that they are no longer pursuing.) Failure to obtain a license will be the end of the opportunity

I need feedback from the community as to whether this is a valid approach.