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| Factor in Success |
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In mid-March, Trendlines' staff members were present at the MIT Enterprise Forum's seminar on "Success Factors in Biotechnology — Drug Development from Concept to Prescriptions."
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The three speakers at the seminar — Dr. Avri Havron, VP and CTO of Clal Biotech Industries; Dr. Itzchak Angel, VP R&D of D-Pharm; and Dr. Michael Schickler, CEO of CureTech — gave excellent presentations that illustrated the possibilities (and challenges) involved in getting a drug all the way from an idea to market.
Dr. Havron explained that his presentation was excerpted from talks he gives to biotech entrepreneurs at universities in an attempt to make scientists aware of what lies ahead when they make a drug discovery in the lab.
In our work with biotech and other medical companies, we are well aware of the hazards facing young companies seeking to raise money to get their discovery through the regulatory process.
We will share a few of Dr. Havron's comments with you, and also add a few of our own.
| | Eureka! I've Validated |
Once a potential drug has been identified and isolated, it is absolutely critical to be able to show a reproducible benchtop process to support the following activities:
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| |  | Lead optimization
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Accepted and validated in vitro model
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Accepted and validated animal model
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| | | Reliable validated surrogate marker
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| | | Significant statistical analysis
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Dr. Havron emphasized the importance of cataloging your work from the very beginning in an organized manner: follow appropriate ISO standards, document everything very carefully, keep samples, and so on. He pointed out that even if you are far from needing to be concerned about ISO, the documentation and samples may be necessary for a potential strategic partner doing due diligence at some stage of your company's life.
| | Inside and Out
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Having isolated your drug, there are a number of extremely important factors that are vital to understand, including checking not only the effect that the drug will have on the patient, but also the effect the patient will have on the drug. He summarized these checks as follows:
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| | | Drug substance properties. Composition of the drug, temperatures and pH it can withstand, stability
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Pharmaceutical properties. The drug's medicinal properties
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Pharmacological properties. The properties and reactions of the drug
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| | | Pharmacokinetic properties. The body's absorption, distribution, metabolism, and excretion of the drug
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| | | Drug interaction potential. The effect it will have on other drugs
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| | | Toxicological properties. Whether it is poisonous or not
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Of course, at every step of the process used to produce the drug, it is critical to understand and evaluate how certain reactions occurred and why.
| | No Trespassing! |
Treatment Investigational New Drugs (IND) "...are used to make promising new drugs available to desperately ill patients as early in the drug development process as possible. The FDA will permit an investigational drug to be used under a treatment IND if there is preliminary evidence of drug efficacy and the drug is intended to treat a serious or life-threatening disease, or if there is no comparable alternative drug or therapy available to treat that stage of the disease in the intended patient population. In addition, these patients are not eligible to be in the definitive clinical trials, which must be well underway, if not almost finished.
An immediately life-threatening disease means a stage of a disease in which there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment. For example, advanced cases of AIDS, herpes simplex encephalitis, and subarachnoid hemorrhage are all considered to be immediately life-threatening diseases. Treatment INDs are made available to patients before general marketing begins, typically during Phase 3 studies. Treatment INDs also allow FDA to obtain additional data on the drug's safety and effectiveness.
From the Federal Register, 22 May 1987, CDER Handbook
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Another aspect that Dr. Havron emphasized as important to investors is a company's intellectual property (IP). IP goes beyond just patents. It also includes aspects that are known as "trade secrets" in other industries, tips about the process used to obtain your material, the company's source or types of raw material, and so on.
Another item that is sometimes considered part of a company's IP could be its regulatory strategy. There are several categories of approval that can be obtained at perhaps lower cost or faster time to market. If a company's product can be used for one of these indications, the chances of obtaining investment may be greatly enhanced.
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| | | A product that is indicated for an unmet medical need
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| | | A product eligible for orphan drug status of the FDA or the EC
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| | | Expedited approval process — drugs for treating AIDS for example may be passed through the approval process much faster because of the great demand for a solution (see box)
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| | | Compassionate use — a patient with advanced disease, no approved treatment options, or no appropriate clinical trial options
This access to a scientifically-tested drug outside of a clinical trial and prior to FDA approval is most commonly referred to as "compassionate use." For this purpose, you may think about trying to get a new, unapproved drug outside of the clinical trial. A letter from a patient requesting compassionate use will often increase the company's chances of getting investment.
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| | The Scent of Success |
Investors do not look favorably on products that have a low probability of successfully completing the registration process. Dr. Havron reviewed several technologies that had poor probability for approval:
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| | | In the past, transgenic animals, milk from BST-treated animals and GM plants were considered suspect and approvals held up.
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Currently there are difficulties with gene therapy products using retrovirus-derived vectors, which the big pharmaceutical are lobbying to overcome. Stem cell therapies are also being held up. Treatments such as products derived from transgenic animals and implants involving modified human cells which involve practices that are still very much unknown are not likely to be approved easily, especially if there are alternative solutions available.
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In the future, genetic profiling and personal medicine may prove to be problematic due to privacy issues, and, therefore, less attractive to investors.
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Overall, the speakers presented a great deal of valuable information about problems (outside those found in the lab) associated with developing biotech therapies and how to deal with some of them. Our overall impression, however, was that entrepreneurs should not be hesitant to seek advice from experts in the pharmaceuticals field and to invite them to be on their boards of directors; and following their advice could certainly help pave the path to success.
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The Trendletter team welcomes your comments.
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Naomi Alper
Senior Project Manager
The Trendlines Group
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