Replies to post #51 on Biotech Values

[rph_in_wi]

In regards to an SPA, I had never heard of an up front "binding agreement" to approve a drug. Is this truely what you ment? My understanding was that the SPA was used by companies to make sure that the sponsor/FDA were all on the same page for what would be required for approval. Due to the length of time it takes to complete these studies, the FDA has been known to change their minds in mid stream based on new science or other factors. Granted my experience was from 1996, so new agreements between drug companies and the FDA have surely come about, but I have seen sure approvals for "me-too" drugs being delayed because of trivial concerns.
Interesting stuff though. Thanks for the education,
RPh

[mskatiescarletohara]

Special Protocol Assessment....

It's my impression too that more and more small-cap and mid-cap biotechs are implementing this strategy for several reasons.

The FDA changes doctors, opinions, review boards frequently, sometimes when protocols are agreed upon, new blood on these committees two years later can say..."Well, wait, we'd like to see this data now.." IMCL, MAXM, GNTA, TRMS, and who know's how many others have been caught in this trap after the pivotals are enrolled and the NDA goes before the review panel. It winds up costing the patients and the company tons of money, their arguments, data and money spent gets lost in the political administrative quagmire. The last three years have been hell on pharma regarding the FDA, finally McClellan is in, and the bureacracy has a leader who appears to be focused.

Biotechs are learning the SPA is more of an insurance contract with the FDA these days, and frankly, I don't blame them for pursuing this strategy. Especially when millions of dollars are on the table for enrolling pivotals. Nothing wrong with having your protocol agreed upon in black and white with the FDA. The FDA needs to be accountable for their mishaps, and so does pharma.

I just wish the SEC would make the FDA letters to pharma public! I get tired of playing guessing games with FDA/pharma relationship. I think IMCL's debacle rattled a lot of nerves.

katie...

[DewDiligence]

Feedback from the FDA: this is how the SPA process is supposed to work:

[IMHO: any company that proceeds to a pivotal trial *without* securing an FDA is either out to lunch or not acting in shareholders’ interests.]

http://biz.yahoo.com/bw/040217/175677_1.html

>>
Boston Life Sciences Receives Further Guidance From FDA on Its Special Protocol Assessment for Phase III Altropane Study

Company in Discussions With FDA to Obtain Formal SPA Agreement Without Necessitating Additional Full Protocol Submission

BOSTON--(BUSINESS WIRE)--Feb. 17, 2004-- Boston Life Sciences, Inc. (NASDAQ: BLSI - News) announced that the Company has received the FDA's response to its latest special protocol assessment (SPA) submission for Altropane for the indication of differentiating Parkinsonian from non-Parkinsonian syndromes in patients with tremors. The FDA requested further modification to the statistical presentation section of the protocol relating to statistical methodologies. The Agency also provided statistical guidance regarding the circumstances under which this single Phase III study would be sufficient to achieve approvability for this indication, and the Company is further seeking clarification on this point.
…
"The requested changes to the statistical plan have been made, and a conference call with the FDA to resolve any remaining ambiguities has been scheduled," stated Marc Lanser, MD, President of BLSI. "Based on the feedback from the Agency, we currently believe that an additional full protocol submission will not be necessary in order to secure an SPA Agreement with the Agency, but no absolute assurances can be made until final agreement is reached with the Agency. In any case, we will continue to respond positively and quickly to requested modifications to the SPA protocol, since we believe that concluding a formal SPA agreement is important to the timely success of Altropane and to the Company," added Dr. Lanser.
<<

[ciciagt]

Hi Dew..catching up on some posts that were started with my question on where is a decent PARS board. My question to you is, what will be your impression/recommendation/investment of GENR if it (or partner) does NOT seek and obtain Special Protocol Assessment as they further the trials with Squalamine? Are you saying the only way you are interested in investing in a biotech is only when they have received SPA? Thanks in advance for any reply. ciciagt

[DewDiligence]

Another SPA:

[This company was so proud of the accomplishment they couldn’t even wait until Monday morning to make the announcement.]

http://biz.yahoo.com/prnews/040523/nysu030_1.html

>>
FDA Approves Isolagen's Request for Special Protocol Assessment

Sunday May 23, 9:00 pm ET

HOUSTON, May 23 /PRNewswire-FirstCall/ -- Isolagen, Inc. (Amex: ILE - News) today announced that the U.S. Food and Drug Administration, or FDA, has approved Isolagen's request for a Special Protocol Assessment, or SPA, relating to the design of two pivotal Phase III clinical trials to be conducted by Isolagen in support of registration of the Isolagen Process for the treatment of nasolabial folds and glabellar lines. The company believes that the FDA's action will significantly reduce the risks associated with conducting its pivotal Phase III clinical trials to provide evidence of efficacy and safety sufficient for license application.

An SPA is an understanding that the FDA concludes with a sponsor company to resolve issues with regard to Phase III clinical trial design, in contemplation of the subsequent filing by that sponsor of an application for a license. The grant by the FDA of a request for an SPA is not commonplace. [I beg to differ here –for the companies I follow, SPA’s have become *very* commonplace.] In the SPA process, the FDA reviews the design and size of a proposed Phase III program and provides comments regarding the adequacy of the clinical trial design to support a claim of efficacy in an approvable Biologic License Application, or BLA. The FDA's comments are binding on its review decision, except in limited circumstances, such as when a substantial scientific issue essential to determining the safety and efficacy of a product candidate is identified after the Phase III program commences. There can be no assurance that the Company's efforts to obtain a BLA will be successful, however, trials conducted on an SPA are frequently associated with successful license application because many of the design concerns that arise in connection with a clinical trial are resolved in advance by the sponsor and the FDA.

About Isolagen, Inc.

Isolagen specializes in the development and commercialization of autologous cellular therapies for soft and hard tissue regeneration. The company's product candidates are based on its proprietary Isolagen Process. Based on the accumulated experience of the company through its retrospective study, clinical trials and treatment of patients in the United Kingdom, the company believes that the Isolagen Process utilizes the patient's own cells to create safe and effective therapies to treat the underlying cause of the patient's condition. Autologous cellular therapy is the process whereby a patient's own cells are extracted, allowed to multiply and then injected into the patient. Isolagen's product candidates are designed to be minimally invasive and non-surgical.
<<

[biomanbaba]

Thanks DEW....i missed that one and needed to read it