Updated FDA Decision Calendar: Five Decisions Before Year-End 10 comments
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The accompanying table (click to enlarge) includes an updated FDA calendar of 65 expected decision dates included in the ETF Innovators (ETFI) New Drug Regulatory Catalyst Index, with six companies listed in red with overdue decisions and the possibility for five decisions during the coming week. Click here for my previous FDA calendar articles. Below is a review of recent decisions + a preview of the coming week's activity:
1.) Allergan (AGN) received FDA approval on Friday for Latisse (bimatoprost solution 0.03%) as a cosmetic medicine treatment which represents the first and only FDA-approved product to enhance eyelashes (making them darker, longer, and thicker). Allergan estimates peak sales for Latisse of $500M, compared to trailing 12-month sales of $4.4B, and the stock rose by 2% during trading on Friday.
2.) EPIX Pharma (EPIX) is up over 213% in the past five days since receiving FDA approval for a blood vessel imaging agent, Vasovist (gadofosveset trisodium), to evaluate peripheral vascular disease in adults.
3.) Genzyme (GENZ) is still waiting on a FDA decision for its Synvisc-One PMA, with the reformulated product designed to provide pain relief for osteoarthritis of the knee for up to six months from a single dose in patients who do not respond to conventional treatments. While the FDA questioned the small sample size and short 26-week follow-up period of the European study, the agency noted that Synvisc-One appears equivalent to the existing product, Synvisc, in terms of safety and component materials.
4.) King Pharma (KG) and Alpharma (ALO) – King Pharma still has another shot at approval of an abuse-resistant opiate pain drug (since receiving a complete response for Remoxy) before year-end since it is in the process of acquiring Alpharma, which has a pending decision for Embeda. An advisory panel endorsed Embeda as an improvement to existing opiate pain drugs which can easily be abused to circumvent their controlled-release formulations, but the panel conceded the drug is not abuse-proof.
5.) Northfield Labs (NFLD) should hear back from the FDA on whether the agency will accept its BLA filing for its blood substitute PolyHeme by 12/29 and if a priority review of six months will be granted. If the depressed share prices and low market caps for NFLD and another blood substitute company, Biopure (BPUR), are not enough to scare you away from investing – a meta-analysis published in JAMA concluded the following:
. . . the authors found a 30% increase in the risk of death and nearly a 3-fold increase in risk of myocardial infarction when all hemoglobin-based oxygen carrier (HBOC) trials were pooled. . .
I would be very surprised if the FDA even accepts the BLA for review given the available data for blood substitutes summarized in the JAMA study – for those speculators and traders looking for the next EPIX, I would look elsewhere.
6.) Savient Pharma (SVNT) should hear back from the FDA on whether the agency will accept its BLA filing for gout drug Puricase by 12/31 and if a priority review of six months will be granted. SVNT has lost about three-quarters of its market value in the past year on cardiovascular concerns for Puricase and the CEO resigned in late November.
7.) Labopharm (DDSS) has an expected PDUFA date of Friday 1/2 for its once-daily formulation of the widely used pain drug tramadol. DDSS is up by nearly 150% in the past month and is up by about 8% in the past year. The company's once-daily tramadol is already marketed outside of the U.S. and the drug is typically dosed about four times daily for the standard products.
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This article has 10 comments:
And very happy Holidays,
Tony
As a second point, we would not have airplanes now if someone wasn't willing to take a chance on the early, very questionably safe planes. I have not heard that PolyHeme is particularly dangerous. It seems likely it is more dangerous than I had thought. However, one does expect progress over time. Using generalizations to bad mouth PolyHeme in particular seems less than fair to NFLD. If you have information specific to PolyHeme, I would be very interested to hear it.
It does seem that there will eventually be a blood substitute. Perhaps PolyHeme will be the first. However, saying "space flight to the moon or to Mars" is impossible is simply recalcitrant thinking. Whether or not you are eventually correct about NFLD as an investment, specific information about the specific company and the specific product is always preferred. To completely deny a company's invention by generalized inuendo (even from JAMA) is unthinkable. Again if you have specific information about NFLD's product, please present it.
Seven hundred fourteen patients were enrolled at 29 urban Level I trauma centers (79% men; mean age 37.1 years). Injury mechanism was blunt trauma in 48%, and median transport time was 26 minutes. There was no significant difference between day 30 mortality in the as-randomized (13.4% PolyHeme versus 9.6% control) or per-protocol (11.1% PolyHeme versus 9.3% control) cohorts. Allogeneic blood use was lower in the PolyHeme group (68% versus 50% in the first 12 hours). The incidence of multiple organ failure was similar (7.4% PolyHeme versus 5.5% control). Adverse events (93% versus 88%; p=0.04) and serious adverse events (40% versus 35%; p=0.12), as anticipated, were frequent in the PolyHeme and control groups, respectively. Although myocardial infarction was reported by the investigators more frequently in the PolyHeme group (3% PolyHeme versus 1% control), a blinded committee of experts reviewed records of all enrolled patients and found no discernable difference between groups.
As you can see from the last comment, the committee of experts found no discernable difference between the controlgroup and the PolyHeme test group. This also brings up another salient point. EMT's could give PolyHeme blood substitute to patients with blood loss, without typing being necessary. This might save a lot of lives. They could carry a supply of PolyHeme, but they could not easily carry an adequate supply of different blood types.
www.thestreet.com/stor...
Also, the death rate for PolyHeme was 37.5% higher than the control arm in the Phase 3 trial when all 712 patients are analyzed and 18.7% higher when the 126 protocol violators are removed, which is in-line with the numbers reported in the JAMA meta analysis
712 patients: 349 got PolyHeme, 363 standard of care --> 46 patients died from PolyHeme arm (13.2% rate) vs. 35 died in control arm (9.6% rate) missed statistical gaol of non-inferiority
Excluding 126 protocol violators (18% of the patients in the study) --> leaves 586 patients to analyze resulting in 10.8% death rate for PolyHeme vs. 9.1% in control arm, which results in achieving the non-inferiority goal for statistical analysis for PolyHeme
Among the 126 pateients excluded for protocol violations --> 23% death rate for PolyHeme (17/70) vs. 12.5% for control arm (7/56)
NFLD got a nice pop on acceptance + priority review yesterday so you were right to be long. However, the FDA typically prefers intent to treat analysis which better reflects real world conditions, especially in the case of emergency medical treatment such as the PolyHeme trial so I don't think they will accept the exclusion of 126 protocol violators to achieve non-inferiority.
I have not traded or invested in NFLD long or short, but it looks like most people are taking their profits today with the stock down about 20% at this point. Finally, I hope that a safe blood substitute can be developed at some point to address the needs you outlined, but I don't think PolyHeme is the answer.
The important data is NOT a direct comparison of how Polyheme compares to blood as a substitute for blood, but how close to being like blood for when there is a need for blood but no blood is available. In the prior study it was found that people who were going to die from bleeding out, had about a 70% survival rate with minimal adverse events v. getting saline. It really is simple deductive reasoning that if I am going to die from bleeding out because we can't get me a transfusion in short order and am offered a 70% chance at survival using Polyheme, I'm using the Polyheme.
This product will receive a limited approval, the stock will rise and the company will be bought by a large well funded company that can develop and market the product further. Of course, part of that deal will be Dr. Gould to get a great paying job, but that's fine, we'll see a price north of $6 (extrapolate near term profit potential just on U.S. remote location trauma usage) in 2009.
If you were long going into the BLA filing/acceptance you already made a lot of money - I have no position long or short in the past and no plans for a position either way in the future - only watch NFLD as part of my FDA calendar of pending new drug decisions
On Jan 05 11:14 AM Ulysses Benjamin Dover wrote:
> Mikey, once again your analysis is faulty. Like Feuerstein you miss
> the central issue of what Polyheme is. It is NOT supposed to be a
> substitute for blood when blood is available (not this version anyway,
> butt someday), it is for when somebody is bleeding out and there
> is no blood available. There is a substantial need for such a product,
> hence why the FDA walked this product through a controversial clinical
> trial and now has granted fast track. To boot, the military wants
> this product.
>
> The important data is NOT a direct comparison of how Polyheme compares
> to blood as a substitute for blood, but how close to being like blood
> for when there is a need for blood but no blood is available. In
> the prior study it was found that people who were going to die from
> bleeding out, had about a 70% survival rate with minimal adverse
> events v. getting saline. It really is simple deductive reasoning
> that if I am going to die from bleeding out because we can't get
> me a transfusion in short order and am offered a 70% chance at survival
> using Polyheme, I'm using the Polyheme.
>
> This product will receive a limited approval, the stock will rise
> and the company will be bought by a large well funded company that
> can develop and market the product further. Of course, part of that
> deal will be Dr. Gould to get a great paying job, but that's fine,
> we'll see a price north of $6 (extrapolate near term profit potential
> just on U.S. remote location trauma usage) in 2009.
The point I am trying to make is as follows: If i was seriously looking at the issues of safety and efficacy, I would NOT go by what Natanson, or Feurstein says. I would talk to the doctors who have actually used Polyheme or Hemopure or other blood substitues in their practice...
"An internal Navy report blasts the Food and Drug Administration for blocking clinical trials of a blood substitute manufactured by Biopure Corp., a struggling biotech company based in Cambridge.
The Navy ultimately hopes to use the experimental product, called Hemopure, to treat military personnel wounded in battle, where traditional blood transfusions aren't readily available. But for the past four years, the FDA has consistently rejected Biopure and the Navy's efforts to test the product in clinical trials, citing safety worries and other concerns.
Now the Navy has commissioned a strongly worded report, obtained by the Globe, that concluded the FDA reviews were "faulty."
"The reviews had consistent patterns of erroneous, misleading, and anecdotal statements, reporting bias, changing requirements, no 'sense of urgency,' " as well as conflicts of interest.
FDA spokeswoman Karen Riley said the agency recognizes the importance of developing products to help both military and civilian trauma patients, but must also make sure the trials do not "involve unreasonable risks to patients. There have been significant safety concerns raised about this class of products," Riley said, noting some studies linked the products to toxic effects, including heart and kidney damage."
--- End copy
Unreasonable risks? If I am dying from massive blood loss, then the risk of dying from or being injured by Hemopure is a risk that I am willing to take. Our military men and women deserve the right to have the option of receiving Hemopure in lieu of death, if needed.
For the FDA to stand in the way of Hemopure - a potentially life-saving product - is insane.