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BMTI - You are correct. I didn't catch that even though it was mentioned right on top of the PR. I assume this was cleared up during the conference call leading to the share price recovery. An investor aware of the requirement may have made a killing snapping up shares during the slide AH.
Exactly. Which is why I highlighted the ITT results.
BMTI - BioMimetic Therapeutics Announces Positive Top-Line Data from its Augment Bone Graft North American Pivotal Trial
Augment Bone Graft Demonstrates Non-Inferiority to Autograft
Company to Host Conference Call to Discuss Results on October 13
Check the ITT results
FRANKLIN, Tenn.--(BUSINESS WIRE)--BioMimetic Therapeutics, Inc. (NASDAQ: BMTI - News) today announced positive top-line results from its North American pivotal (Phase III) randomized controlled trial comparing its fully synthetic, off-the-shelf bone growth factor product, Augment Bone Graft (“Augment”), to autograft for use in hindfoot and ankle fusion surgery. The primary study goal was to establish non-inferiority of Augment compared to autograft. Autograft is the historical standard of care but has the limitation that it must be obtained and transplanted from another bone in the patient’s body, often requiring a second surgical procedure. These positive top-line results indicate that, with the use of Augment, patients can expect a comparable treatment outcome while being spared the pain and potential morbidity associated with traditional autograft bone harvesting and transplantation.
Study Results
For the primary endpoint, the percent of subjects achieving fusion as defined by 50% or greater bone bridging on CT scans at 24 weeks, patients treated with Augment experienced a similar fusion rate (61.2%) compared with those receiving autograft (62.0%), which met non-inferiority (p=0.037; n=397 patients). Since many patients had multiple joints treated, analysis was also performed on a per joint basis. Non-inferiority was also established on a per joint basis, with 66.5% of joints treated with Augment fused on CT scans compared to 62.6% of joints treated with autograft (p=<0.001; n=597 joints).
In the key clinical, secondary endpoints, the healing (union) rate was 83.1% for Augment compared to 83.9% for autograft at 24 weeks (p=0.008; n=397). The delayed/nonunion rates (lower rates are better) were 8.8% for Augment and 10.2% for autograft (p=0.008). The remaining patients were judged by the investigators to be progressing to healing but were not able to be definitively diagnosed. Infection rates also tended to be lower for Augment (7.3%) compared to autograft (9.5%; p=0.011). Pain at the autograft donor site was present in 95.6% of autograft patients, while Augment patients do not require a donor site. Substantial pain at the autograft donor site (at least 20mm on VAS pain scale) was present at six months in 12.4% of the patients treated with autograft and none of the Augment patients. These findings demonstrate that patients treated with Augment can expect clinical results as good as if they had been treated with autograft, while being spared the pain and potential for additional surgical and post-operative complications resulting from the extra surgical procedure often required to harvest the autograft.
Seventy-five percent (75%) of patients in both groups had one or more risk factors for poor healing. There were no significant differences in the frequency of serious adverse events between the Augment and autograft treated patients. Finally, analysis of human anti-PDGF antibodies indicated only 13% of Augment patients experienced antibody formation at any time point, which dropped to 3.9% at six months. Additionally, 3.5% of autograft patients also had anti-PDGF antibodies. Most importantly, none of the antibodies in either group was neutralizing.
The data above reflect the results of the 397 patient “modified intent-to-treat” (mITT) study population. Thirty-seven (37) patients were excluded from this analysis, 21 of which were randomized but never treated and 16 which had major protocol deviations which were prospectively identified (e.g. midfoot fusions even though these were a specific exclusion criteria). Thus, the mITT population represents over 90% of all randomized patients and over 95% of all treated patients.
On a strict intent-to-treat (ITT) population in which those patients who were randomized but never treated are counted as automatic failures, 24 week fusion rates on CT scans were 57.9% for patients randomized to Augment and 60.4% for patients randomized to autograft (p=0.065; n=434). On a per joint basis the CT fusion rate was 65.2% for Augment compared to 64.6% for autograft (p=0.004; n=631). Clinical union rate for the ITT population was 79.6% for the Augment group and 79.2% for the autograft group (p=0.004; n=434). The delayed/nonunion rate on the ITT population was 8.1% in the Augment group and 10.7% for the autograft group (p=0.015; n=434).
Medical Need
“We are excited that our pioneering work at BioMimetic on ways to improve orthopedic and dental tissue regeneration has again translated into a potential new treatment option for patients with significant debilitating injuries or diseases,” said Dr. Samuel Lynch, president and CEO of BioMimetic Therapeutics. “These top-line data demonstrate a consistent picture that Augment is at least as efficacious as autograft, while also having the benefit of sparing patients the pain and potential morbidity resulting from autograft bone harvesting and transplantation to the fusion site. We look forward to sharing these positive pivotal trial data with the FDA. Finally, I’d like to once again acknowledge the work of our investigators and thank them for their excellent execution of this complex and rigorous trial.”
“These top-line results are very exciting,” said Dr. Christopher DiGiovanni, principal investigator for the Augment trial and professor of orthopaedic surgery and chief of the division of foot and ankle surgery in the department of orthopaedic surgery at the Warren Alpert School of Medicine at Brown University, Rhode Island Hospital. “The data available thus far support the primary objective of the study which is to find a safe and effective alternative to autograft in foot and ankle fusion surgeries. Should Augment receive FDA approval based on the full data set, I believe it will be widely used by practicing foot and ankle surgeons anxious to spare patients all the pain, potential morbidity, and extra surgical and anesthesia time associated with traditional autograft bone harvest. I look forward to having Augment available for use in patients.”
Joint fusion is the standard surgical treatment for chronic pain in the foot and ankle. This pain is often caused by arthritis, joint instability and congenital defects. The current gold standard for fusion involves the use of autograft, which is bone taken from another location in the patient’s body. While effective, the use of autograft creates significant morbidities at a previously asymptomatic harvest site. Some of these morbidities include severe pain, serious infection, numbness and increased anesthesia time. Autograft also incurs a financial burden when operating room time, instrument cost, anesthesia cost and surgeon charges are considered. Therefore it is of great value to the surgical community to find a safe and effective off-the-shelf alternative to autograft.
Study Design
The Augment North American pivotal trial is a prospective, randomized, blinded, controlled study providing a head-to-head comparison of Augment to autograft for use in hindfoot and ankle fusion surgery. Treatment was randomized 2:1, Augment to autograft, and the randomization was stratified for two variables, hindfoot vs. ankle procedures and risk factor vs. no risk factor. Risk factors included diabetes, smoking or recent history of smoking and obesity (BMI> 30kg/m2).
Thirty-seven sites in the United States and Canada participated in the study, enrolling a total of 434 patients. Patient enrollment was completed at the end of December 2008. The results announced today are through 24 weeks after treatment surgery which is the time of assessment of the primary endpoint, but all study patients will be followed through a final 52 week visit. The study is designed to demonstrate non-inferiority between Augment Bone Graft and autograft.
All p values reported in this release are derived from non-inferiority analyses. Analysis of the complete data set will continue for some time. A substantial amount of data in addition to the top-line data announced today will be included in the Company’s PMA submission, which is expected to be filed with the FDA within the next three months.
Conference Call and Webcast
BioMimetic will be hosting a conference call and webcast on October 13, 2009 at 6:30 EDT to discuss the study results. A live webcast of the conference call will be available on the Investor Relations section of BioMimetic’s website at www.biomimetics.com. The webcast will be archived on the website for at least 30 days.
The conference call may be accessed on October 13, 2009 by dialing 888-679-8018 (passcode 32982191). The international dial in number is 617-213-4845, and the same passcode applies. Participants should dial in 10 minutes prior to the call if they have not pre-registered.
About Augment Bone Graft
Augment consists of a combination of a sterile solution of 0.3mg/ml purified recombinant human platelet-derived growth factor BB (rhPDGF-BB) and a synthetic tricalcium phosphate (TCP) bone matrix. The rhPDGF-BB functions to recruit the bone healing cells and new blood vessels to the site of injury through processes termed chemotaxis, mitogenesis and angiogensis, while the TCP acts as a scaffold or lattice for the deposition of new bone. A similar product successfully developed by BioMimetic (GEM 21S) has already been FDA approved for bone and periodontal regeneration in the jawbone.
About BioMimetic Therapeutics
BioMimetic Therapeutics is a biotechnology company utilizing purified recombinant human platelet-derived growth factor (rhPDGF-BB) in combination with tissue specific matrices as its primary technology platform for promotion of tissue healing and regeneration. rhPDGF-BB is a synthetic copy of one of the body's principal agents to stimulate and direct healing and regeneration. The mechanism of action of this platform technology suggests it may be effective in a broad array of musculoskeletal applications, including the repair of bone, ligament, tendon and cartilage. Through the commercialization of this technology, BioMimetic seeks to become the leading company in the field of orthopedic regenerative medicine. BioMimetic received marketing approval from the FDA for its first product, GEM 21S®, as a grafting material for bone and periodontal regeneration following completion of human clinical trials, which demonstrated the safety and efficacy of the rhPDGF-BB platform technology. Additionally, BioMimetic Therapeutics has completed and ongoing clinical trials with its product candidates Augment and Augment Injectable in multiple orthopedic bone healing indications including the treatment of foot and ankle fusions and the stimulation of healing of fractures of the wrist.
http://finance.yahoo.com/news/BioMimetic-Therapeutics-bw-3880144727.html?x=0&.v=1
They are? I wasn’t aware of any survey of WFMI’s customers’ political inclinations
People make all kinds of claims on social networking sites. I’m surprised that you would take such claims at face value.
WFMI -
WFMI reached its highest price in almost two years today (up 367% from the Nov 2008 low). John Mackey’s op-ed piece on healthcare does not appear to be a concern for investors. (Nor should it be a concern, for the reasons mentioned in #msg-40553109.)
VNDA -
$200M up-front and $265M in potential milestones is not $465M of NPV
VNDA - Vanda only has one other drug in its pipeline. I don't know why they didn't just sell themselves for say 500 million which would have been close to a 60% premium. Instead they sign a deal for $465 million. I think investors may have been happier with the former choice.
VNDA - Wow. I agreed with most of the posters that thought this was way overvalued. Guess I was wrong.
DEPO and Solvay -
I'm not trying to argue with you, just pointing out it's a risk.
DEPO and Solvay - Well the reaction I had was the same reaction the CEO of DEPO had when an analyst asked the same question you did in the Q&A session after the trial results were presented, which is surprise given that the results were positive. Have a listen. It happens right at the beginning of the Q & A.
http://investor.depomedinc.com/phoenix.zhtml?c=97276&p=irol-EventDetails&EventId=2474151&WebCastId=918107&StreamId=1374809
I guess there's a risk for any biotech company when it partners with someone that the partner will void the contract. That risk decreases substantially when the program for the partnership is going well. I think given the current marketcap of DEPO, the amount of cash it has, the rest of its pipeline and the fact that it has two marketed products, I'm not too concerned about that risk even though the indication is not that novel .
DEPO and Solvay -
Solvay is getting acquired by Abbott. How to know Abbott will stick with the program?
DEPO - Seems undervalued and I snapped up some shares in the AM. I see that for their pain drug they are due about 370 M in milestone and sales payments from Solvay and it also looks like they are funded for the next couple of years with 80 M in cash. There was a note from an analyst valuing it between 7 and 8 which is a 350 to 400 M marketcap.
They will most certainly need additional trials for Serada and according to the presentation they are ready to do them if necessary. The current set of trials apparently cost around 15 M and they said they have the funds for that assuming similar cost trials are required.
DEPO - Mixed results for hot flash trial
Depomed Reports Results From Two Phase 3 Clinical Trials Evaluating Non-Hormonal Therapy for Menopausal Hot Flashes
Press Release
Source: Depomed, Inc.
On 7:00 am EDT, Monday October 12, 2009
MENLO PARK, Calif.--(BUSINESS WIRE)--Depomed, Inc. (NASDAQ:DEPO - News) announced today top-line results from the BREEZE 1 and 2 Phase 3 clinical trials evaluating the safety and efficacy of SeradaTM, an investigational non-hormonal extended release formulation of gabapentin for the treatment of menopausal hot flashes.
In the higher dose treatment arm of the two doses evaluated, the 1800mg dose achieved positive results at 4 weeks. All four co-primary endpoints of the 1800mg dose at 4 weeks demonstrated significant reductions in frequency and severity in both clinical trials (p-values ranged from 0.0001 to 0.004). Of the other four co-primary endpoints of the 1800mg dose at 12 weeks, one endpoint was positive (p=0.0026) while the other three endpoints did not achieve statistical significance.
In the lower dose treatment arm, the 1200mg dose at 4 weeks achieved statistical significance in three of the four co-primary endpoints. Frequency was significantly reduced in both clinical trials (p-values of 0.0024 and 0.0117) at four weeks. Severity was significantly reduced in only one trial (p-value 0.0016). Of the other four co-primary endpoints of the 1200mg dose at 12 weeks, one endpoint was positive (p=0.0024) while the other three endpoints did not achieve statistical significance.
The primary endpoints in the studies were a statistically significant reduction in the frequency and severity of menopausal hot flashes relative to placebo after 4 weeks and 12 weeks of stable treatment. Both patients’ and clinicians’ impression of overall improvement in the higher dose treatment arm was highly statistically significant relative to placebo in both studies.
Additional efficacy and safety details will be provided on Depomed’s investor conference call scheduled for today, October 12, 2009.
“We remain very enthusiastic about Serada and our menopausal hot flash program. There is a large unmet need for a non-hormonal hot flash therapy, and we believe Serada has the potential to address that need,” said Carl Pelzel, Depomed’s president and chief executive officer. “We look forward to meeting with the FDA later this quarter to discuss these results, the path to approval and any additional clinical work that may be required.”
“We are pleased to see that the 1800mg dose clearly demonstrated Serada’s efficacy at 4 weeks while the lower 1200mg dose at 4 weeks achieved three of the four co-primary endpoints. While the drug effect in the studies was what we expected to see, there was an unexpectedly high placebo effect, particularly in the latter part of one of the studies,” said Dr. Michael Sweeney, M.D., Depomed’s vice president, Research and Development. “We need to extensively analyze the data to better understand all of the implications in order to refine our approach to any additional development.”
Study Design
In each BREEZE study, patients were randomized into three treatment arms: placebo; Serada 1200mg dosed once daily; or Serada 1800mg twice daily (dosed 600mg in the morning and 1200mg in the evening). BREEZE 1 and 2 combined enrolled 1,100 patients. The four co-primary efficacy endpoints in both studies were the reductions in the mean frequency of moderate to severe hot flashes, and the average severity of hot flashes, measured after four weeks and 12 weeks of stable treatment. Patients in the BREEZE 2 study received twelve weeks of treatment, while patients in the BREEZE 1 study were treated for six months in order to assess safety and persistence of efficacy.
Safety
Serada was generally well tolerated in both BREEZE trials. The most common adverse events were dizziness and somnolence. The incidence of dizziness in the active arms was between 17% and 24% (compared to 3% for placebo). Somnolence ranged from 7% to 19% in the active arms (compared to 2% or 3% in the placebo arms).
About Menopausal Hot Flashes
Hot flashes, which affect 32 million women in the U.S. annually, are characterized by a sudden, temporary onset of body warmth, flushing and sweating. Hot flashes are disruptive and impact women’s overall quality of life, affecting their mood and their ability to sleep. In fact, insomnia typically worsens with the severity of hot flashes. According to the North American Menopause Society, hot flashes are the most common menopause-related discomfort. Research suggests hot flashes occur when the body’s internal thermoregulatory mechanism (located in the hypothalamus) becomes irregular, narrowing the body’s thermoneutral zone. Thus, even small fluctuations in body temperature can cause menopausal women to experience perfuse sweating or severe chills that would not affect a person with a properly functioning thermoregulatory mechanism.
About Serada
Serada is an extended-release formulation of gabapentin for the treatment of menopausal hot flashes using Depomed’s proprietary Acuform® drug delivery technology. By combining gabapentin with Acuform technology, Serada is absorbed slowly into the upper gastrointestinal tract over several hours rather than immediately. Immediate release formulations of gabapentin have been approved by the FDA to treat neuropathic pain and epilepsy.
Investor Conference Call
Depomed will host a conference call today, October 12, 2009, beginning at 9:00 a.m. ET, 6:00 a.m. PT to discuss the results of the trials in further detail. The conference call will be available via a live webcast on the investor relations section of Depomed’s website at http://www.depomed.com. Please access the website 15 minutes prior to the start of the call to download and install any necessary audio software. An archived webcast replay will be available on the Company’s website for three months.
About Depomed
Depomed, Inc. is a specialty pharmaceutical company with one product candidate through Phase 3 clinical development, another in Phase 3 clinical development, two approved products on the market and other product candidates in its early stage pipeline. Product candidate DM-1796 has completed Phase 3 clinical development and has been licensed to Solvay Pharmaceuticals. A New Drug Applications for DM-1796 is expected to be filed with the FDA in the first quarter of 2010. Product candidate SeradaTM is in Phase 3 clinical development for menopausal hot flashes. GLUMETZA® (metformin hydrochloride extended release tablets) is approved for use in adults with type 2 diabetes and promoted by Santarus, Inc. in the United States. Depomed formulates its products and product candidates with its proven, proprietary Acuform® drug delivery technology, which is designed to improve existing oral medications, allowing for extended, controlled release of medications to the upper gastrointestinal tract. Benefits of Acuform-enhanced pharmaceuticals include the convenience of once-daily administration, improved treatment tolerability and enhanced compliance and efficacy. Additional information about Depomed may be found on its website, www.depomed.com.
http://finance.yahoo.com/news/Depomed-Reports-Results-From-bw-3699568438.html?x=0&.v=1
SPPI - Currently has a marketcap of 250 million with approximately 150 million in cash, two approved drugs, a reasonable pipeline and a few positive triggers within the next few months. Thinking of wading in tomorrow. I know that its not the most popular stock on this board. Anyone with a contrarian view and thinks the stock has a lot further to go down?
ARYX -
never any shame in taking some good profits
If you are reasonably confident a significant deal is going to get done for budiodarone at some point, whether that be in one month or by 1Q10, then why sell now? The stock still looks pretty cheap to me at this point even after the nice rebound after the tecarfarin debacle as it's still at a sub-$100 million market cap.
ACOR - Even with a yes vote on efficacy, with a black box warning, would the potential market drop significantly? Their presentation slides state that about 65-85% percent of the more than a million people with MS worldwide have walking impairment. I wonder by how much a blackbox would drop the potential patients for the drug and whether that has been factored into the current price.
ARYX - I think its a bit concerning that they need to play poker with potential partners. The data appears to be compelling enough that any deep pocketed partner should have plunked down the 50-75 million for the partnership by now. I wonder whether the delay is because the partners want to hash out the details for the potential PIII trial first with the FDA. In any case I lightened my holdings by about 50% last week assuming a delay till Q1 of 10. If I had to guess, given my knack for timing, this means that ARYX will probably sign a partnership next week :(
ARYX - ARYx Therapeutics Secures $35 Million Committed Equity Financing Facility
Looks like they are finding it hard to convince a partner
FREMONT, Calif.--(BUSINESS WIRE)--ARYx Therapeutics, Inc. (NASDAQ:ARYX - News), a biopharmaceutical company, today announced that it has secured a committed equity financing facility under which it may sell up to $35 million of shares of its common stock to Commerce Court Small Cap Value Fund, Ltd. (“Commerce Court”) over a 24 month period. The maximum number of shares that can be sold by ARYx is specified in the agreement. ARYx is not obligated to utilize any of the $35 million facility and remains free to enter into and consummate other equity and debt financing transactions.
http://finance.yahoo.com/news/ARYx-Therapeutics-Secures-35-bw-3601928290.html?x=0&.v=1
RPRX up over 150 % in premarket - Talk about a pump. And now they'll have money for the lawsuits
PDP market - You could be right. According to BVF though its about 30 to 40 % of PD market. Check slide 16
http://www.biovail.com/local/files/Resources/August%202009.pdf
The annual cost of the drug would have been about $3500 -$5000 so depending on how much market share they get sales can reach well above a billion.
Pimavanserin (ACAD) -
My only question is why?
LOL I guess now an exception can be made for articles from biomedreports
ALTH and ADAM F - Adam has been a staunch supporter of ALTH. I wonder what he will write about ALTH raising money since he has been claiming that it is a likely candidate to partner or even be bought out. In the past he has been skeptical about companies that were unable to find a partner.
Allos Therapeutics Announces Proposed Public Offering of Common Stock
WESTMINSTER, Colo.--(BUSINESS WIRE)--Allos Therapeutics, Inc. (Nasdaq: ALTH - News) today announced that it is offering to sell, subject to market and other conditions, 11,000,000 primary shares of its common stock pursuant to an effective shelf registration statement in an underwritten public offering. Allos Therapeutics also intends to grant the underwriters a 30-day option to purchase up to an aggregate of 1,650,000 additional primary shares of common stock to cover over-allotments, if any. All of the shares in the offering are to be sold by Allos Therapeutics, with proceeds to be used to support the commercialization of FOLOTYN™ (pralatrexate injection), preclinical research and clinical development of FOLOTYN, and general corporate purposes. J.P. Morgan Securities Inc. and Citigroup Global Markets, Inc. are acting as joint book-running managers of the proposed offering. Leerink Swann LLC is acting as co-lead manager of the offering. JMP Securities LLC is acting as co-manager of the offering.
http://finance.yahoo.com/news/Allos-Therapeutics-Announces-bw-3005021453.html?x=0&.v=1
orex - looks like they've got another fat burning pill in the pipe
Orexigen(R) Therapeutics Schedules September 30, 2009 Teleconference and Webcast to Discuss Phase 2b Trial Results From Second Obesity Drug Candidate, Empatic(TM)
SAN DIEGO, Sept. 29 /PRNewswire-FirstCall/ -- Orexigen® Therapeutics, Inc. (Nasdaq: OREX - News), a biopharmaceutical company focused on the treatment of obesity, plans to announce the results of a Phase 2b trial for Empatic(TM) on Wednesday, September 30, 2009 before the markets open. The announcement will be followed by a live webcast and conference call at 8:30 a.m. Eastern time.
Orexigen management will host the call and webcast to discuss the results of this clinical trial and answer questions. The live call may be accessed by phone by calling (800).884.5695 (domestic) or (617).786.2960 (international), participant code 64389662. The webcast can be accessed live on the investor relations section of the Orexigen web site at www.orexigen.com, and will be archived for 14 days following the call.
http://finance.yahoo.com/news/OrexigenR-Therapeutics-prnews-632635738.html?x=0&.v=1
ARYX - There is a yahoo post that states that according to an analyst a deal is possible before Q1 of 2010 which seems to indicate that the deal has been pushed off. Anyone else following aryx know whether thats true? If so they will likely raise cash soon since there is no other event on the calendar that is likely to raise value within the next few months. Any thoughts?
PARD (mcbio) - I think all your points are excellent and perfectly valid. Regarding lack of a controlled trial and the possibility of the control arm doing better than expected, there is that possibility and that is exactly why I used the "assuming BSC survival is 14 weeks as expected". I think the difference between this trial and the numerous other trials where the control arm on SOC fared better than expected is that the primary endpoint is overall survival. There is no placebo effect as well. The trial is an open label trial monitored by a DMC so patients know whether they are getting pico or not. Based on the literature that I've read, the average survival period for patients with relapsed SCLC is around 12 weeks. Some state survival could be as low as 8 weeks. Since the control arm does not receive any type of chemo or any other treatment that will rid of the cancer cells I think there is very little chance that overall survival will exceed the average.
The phase II trials of picoplatin had a mixture of refractory, resistant and sensitive patients but the majority were not sensitive. The phase III trial includes a lot more sensitive patients and since those type are more responsive to platinum therapy, the assumption is that survival on the Pico arm will be a lot better than in the P II trials. The occurrence of the 320th event certainly points that way. The sensitive patients in the PII trials had an overall survival period of 36 weeks compared to the refractory ones who had only 26 weeks.
I am more concerned about other factors that I may not have thought about. I think the lack of a partner until now is both concerning and encouraging. Management has not diluted yet and financially the company is not sound so they need money. So it is encouraging that dilution has not occurred. On the other hand if the outcome is so obvious I also wonder why a partner hasn't swooped in yet. Management wants the best deal possible so they seem to be willing to wait, or so they say.
PARD - I agree that an entry point at this price can be considered risky. Cash on hand is only about 45 million so the share price will likely drop to around 1.5 if results are not stat sig. I was lucky enough to get some when the share price dropped to around $4 a few weeks after ASCO though I have some shares in the 6-7 range as well. I will not be buying any more and instead am focusing on hedging myself but if there are others who got in earlier or at the same time i did I would like to know what they think.
However even for people looking to get in now, odds are stacked in PARD's favor. The treatment arm patients will receive the drug picoplatin plus best supportive care (BSC) while the second arm will only receive BSC, which means they will not get any type of chemo. The standard survival period for patients on BSC is 13-14 weeks based on historical data from past clinical trials. Picoplatin in previous ph II trials demonstrated a range of 27-36 weeks for patients who were refractory and platinum resistant or sensitive. To achieve success pico just needs to demonstrate around 19 weeks survival assuming BSC survival is 14 weeks as expected. There is no reason to believe patients on BSC will survive longer than 14 weeks especially because they will receive no chemo. Based on what management said, to achieve 19 weeks the 320th event, which was the trigger for the data to be collected, should have occurred sometime between july and august and instead occurred in mid september which likely means that the Pico arms survival period was around 26-28 weeks.
As for the marketcap, 2nd line SCLC is about 400 -600 million worldwide. Once 2nd line is obtained there is the potential for off label 1st line because pico has a pretty clean safety profile which potentially doubles the market. The drug also showed efficacy in CRC and CRPC which are much larger markets. According to management there are 4 other indications they want to look at. Other than SCLC other indications are several years away.
Pico is an orphan drug and has fast track so this drug could be selling sometime next year. So if the trial is successful I expect at least a market cap of 500 million and maybe more than 750 million if the survival exceeds 27 weeks. PARD will likely partner or even sell itself for the right price which could mean a much higher marketcap.
PARD - Event 320 for their PIII 2nd line SCLC trial occurred a few weeks ago, somewhat later than management expected which is actually a positive. The trial is powered to show a 33% improvement in overall survival meaning that the arm treated with the drug, picoplatin + BSC, has to show around 19 weeks survival assuming the other arm, on BSC, shows around 14 weeks. Based on the occurrence of event 320 it appears that the pico + BSC arm will come somewhere around 26-28 weeks.
I've been holding since ASCO but wanted to know if anyone else is on PARD and what they think the chances are of success in the trial. The company seems extremely undervalued at this point though it only has about 45 mil in cash. What impresses me is that management has not diluted yet despite the shortage in cash and is steadfastly waiting for trial results to either strike a partnership deal or dilute.
scam indicator - Solvency ratios under financials
RIGL (mcbio) - What are your thoughts on current valuation though? Cash was about 80 million in june and the recent raise is about 95 million giving it about 150 million as of 9/09 assuming about 20-30 million was burned for the current Q. The current marketcap is about 415 M.
At what price would you get in?
ALTH - Question on insurance for anyone who has seen it before. Management claimed that the price tag for the drug was going to be a steep 70-120 K. How have insurance companies handled such high priced drugs when they are only given accelerated approval?
Allos Therapeutics' FOLOTYN First and Only FDA-Approved Therapy for Relapsed or Refractory Peripheral T-cell Lymphoma
Press Release
Source: Allos Therapeutics, Inc.
On Friday September 25, 2009, 7:00 am EDT
Companies: Allos Therapeutics, Inc.
WESTMINSTER, Colo.--(BUSINESS WIRE)--Allos Therapeutics, Inc. (Nasdaq:ALTH - News) today announced that last night the U.S. Food and Drug Administration (FDA) granted accelerated approval for FOLOTYNTM (pralatrexate injection) for use as a single agent for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). FOLOTYN is the first and only drug approved by the FDA for this indication and represents a new treatment option for patients with relapsed or refractory PTCL. This indication is based on overall response rate. Clinical benefit such as improvement in progression free survival or overall survival has not been demonstrated. Allos expects to make FOLOTYN available to patients in the U.S. in October.
“Individuals with peripheral T-cell lymphoma have a very poor prognosis and almost always relapse or become refractory to initial therapy.1 As a result, there is an urgent need for new therapies to treat patients with this challenging disease. FOLOTYN has demonstrated its efficacy and safety in the PROPEL clinical trial, and I believe it will be a welcome addition for physicians who treat patients with relapsed or refractory PTCL,” stated Owen A. O'Connor, MD, PhD, principal investigator in the PROPEL study of FOLOTYN; deputy director for Clinical Research and Cancer Treatment, NYU Cancer Institute; chief, Division of Hematologic Malignancies and Medical Oncology; professor of Medicine and Pharmacology at the NYU Langone Medical Center.
PTCL comprises a biologically diverse group of aggressive blood cancers that has a poor prognosis.2 The Company’s New Drug Application (NDA) for FOLOTYN was based on data from the PROPEL trial. The Company believes PROPEL is the largest prospective, multicenter, international trial ever conducted in patients with relapsed or refractory PTCL.
“We are enthusiastic about providing this new therapy to patients with relapsed or refractory PTCL,” said Paul L. Berns, president and chief executive officer at Allos Therapeutics, Inc. “The approval of FOLOTYN is a transformative event for Allos representing our first U.S. indication. We thank the many patients and clinical investigators who participated in the PROPEL study. Moving forward, we plan to continue advancing the FOLOTYN clinical development program.”
“Aggressive peripheral T-cell lymphomas have been a largely ignored group of diseases,” said James O. Armitage, MD, The Joe Shapiro Professor of Medicine, Department of Internal Medicine, University of Nebraska Medical Center. “It is exciting to have the first FDA-approved therapy for relapsed or refractory peripheral T-cell lymphoma.”
Allos is dedicated to patient access and has established a patient assistance program named ASAP (Allos Support for Assisting Patients) to provide reimbursement support. Commencing in October, more information regarding ASAP will be available by calling the Hotline at 1-877-ASAP102 (272-7102), Monday to Friday, 8 a.m. to 7 p.m. Central Time or by visiting www.getASAPinfo.com.
“The approval of FOLOTYN brings a new treatment option to patients afflicted with peripheral T-cell lymphoma,” said Peter L. Saltonstall, president and chief executive officer of the National Organization for Rare Disorders (NORD). “We at NORD are excited about this approval and will continue our efforts to focus national attention on rare diseases and on the fact that most rare diseases have no FDA-approved treatment at this time.”
In connection with the accelerated approval, Allos has agreed to undertake additional clinical studies to further verify and describe the clinical benefit of FOLOTYN in patients with T-cell lymphoma.
FOLOTYN was discovered by Sloan-Kettering Institute for Cancer Research, SRI International and Southern Research Institute and developed by Allos Therapeutics.
http://finance.yahoo.com/news/Allos-Therapeutics-FOLOTYN-bw-392090082.html?x=0&.v=1
FDA To Reassess Approval Of Regen Device >RGBO
By Alicia Mundy and Jared A. Favole
WASHINGTON (Dow Jones)--The U.S. Food and Drug Administration is reconsidering the approval of ReGen Biologics Inc.'s (RGBO) knee device after an investigation showed that unprecedented industry and Congressional involvement influenced the agency's decision and even reached the commissioner's office.
The FDA released a report Thursday saying top FDA officials repeatedly deviated from agency procedures in overriding FDA scientists who rejected the device's approval twice amid concerns about its safety and effectiveness. The director of the FDA's device division who granted approval for the device, Daniel Schultz, has since left the agency.
Joshua Sharfstein, the deputy director of the FDA, said the report shows there were "definite threats" to the integrity of the FDA's medical device review process. The report could have far-reaching effects for not only ReGen, but the entire $200 billion device industry. Sharfstein said the review focused on "underlying weaknesses" in the FDA's rapid-approval process for certain medical devices, also known as the 510-K program.
The FDA yesterday announced that it was reviewing that program and reached out to an influential scientific group, the Institute of Medicine, to also investigate it.
Menaflex was cleared through the rapid-approval process. One of the main questions the FDA wants to answer in the next few months is whether industry and Congressional influence led the agency to improperly allow the device to go through that process.
Sharfstein said, however, the report isn't a reason for patients who have had the device, Menaflex, implanted in their knee to panic because the report focused on the FDA decision, not the science behind that decision.
He said the FDA will now review the entire decision over the next few months and that may result in the product being withdrawn from the market.
ReGen Chief Executive Gerald E. Bisbee said in a statement the report didn't evaluate the scientific evidence of the device, and noted the product is still available. The company has said that it is safe and helps knee-surgery patients recover more quickly.
The questions about the approval of ReGen's knee device were first reported by The Wall Street Journal in March.
The report shows Congressional and industry influence reached as high as the commissioner's office, where, in a rare move for the head of an agency, former FDA Commissioner Andrew von Eschenbach pressured other FDA officials to act quickly on the device. Von Eschenbach didn't make himself available to comment for the FDA report and wasn't immediately available to comment.
A high-ranking FDA official described pressure from Capitol Hill as "the most extreme he had seen, and the agency's acquiescence to the Company's demands for access to the Commissioner and other officials in the Commissioner's office as unprecedented," according to the report.
Congressional pressure was so intense that it "initiated a chaotic new phase" during the agency's review of the device, the report says.
Four Democrats from the New Jersey delegation contacted the FDA to encourage it to speed of its review of the ReGen device. ReGen is based in New Jersey. They are Sens. Frank Lautenberg and Robert Menendez, and Reps. Steve Rothman and Frank Pallone Jr. Pallone is chair of the House Energy and Commerce Committee's health subcommittee.
The politicians have said through spokespeople that their involvement was merely to help a constituent get fair treatment at the FDA.
The FDA report says over the device's 17-year approval history "multiple departures from processes, procedures, and practices occurred."
Peter Lurie of the consumer group Public Citizen, which opposed Menaflex's approval last year, said the report "confirms our suspicion that the approval process here was completely corrupted, and we believe a review of the science will result in its being withdrawn."
-By Jared A. Favole, Dow Jones Newswires; 202-862-9207; jared.favole@dowjones.com
http://online.wsj.com/article/BT-CO-20090924-714314.html
Chelsea Therapeutics Reports Preliminary Phase III Data of Droxidopa for Treatment of Symptomatic Neurogenic Orthostatic Hypotension
Press Release
Source: Chelsea Therapeutics
On Thursday September 24, 2009, 9:08 am EDT
Companies: Chelsea Therapeutics International Ltd.
* Study 302 Did Not Meet Statistical Significance on Primary Endpoint
* Key Secondary Endpoints Strongly Support Symptomatic Benefit
* Design of Second Study May Offer Significant Advantages
* Company Conducting Additional Data Analyses
CHARLOTTE, N.C., Sept. 24, 2009 (GLOBE NEWSWIRE) -- Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP - News) announced top-line results from Study 302, the first of two Phase III trials of Droxidopa for the treatment of symptomatic neurogenic orthostatic hypotension (NOH). While Study 302 demonstrated that Droxidopa showed a strong symptomatic benefit during the open-label dose titration and run-in phase of the trial, a preliminary review of the data indicates it did not demonstrate a statistically significant improvement relative to placebo, as measured by the mean score of Item 1 (dizziness or light-headedness) of the Orthostatic Hypotension Symptom Assessment (OHSA) during the double-blind phase of trial, the study's primary endpoint. Droxidopa was safe and well tolerated, with no significant related adverse events reported.
"While the outcome on Item 1 of the OHSA scale did not meet the company's expectations, our preliminary look at each of the secondary symptomatic outcome measures was encouraging and supportive of the therapeutic benefit of Droxidopa in neurogenic orthostatic hypotension," commented Dr. Simon Pedder, Chelsea's President and CEO. "Further, we anticipate a more comprehensive review of the data will help determine the relative impact of a higher than anticipated placebo response and what, if any, additional factors may have contributed to these unexpected results. Key features to the design of this study included an initial 7-day open-label drug treatment period following dose titration and prior to a 14-day randomized withdrawal treatment period. While we intended to stabilize patients immediately prior to withdrawal, the observed decline in BP during this period appears to have had a negative effect on the study's ability to discern treatment effect. In addition, the benefits of Droxidopa, as measured by both BP and item 1 of the OHSA scale, appeared to persist to some extent despite absence of therapy, raising potential questions regarding the suitability of this type of trial design for an NOH study. We remain hopeful that the results of Study 301, which is a standard induction design study in which patients are washed out between titration and the blinded study, may provide a better opportunity to clearly demonstrate the efficacy of Droxidopa in this indication."
Study 302 was an enriched, double-blind, placebo controlled withdrawal-design study in which all patients underwent an initial open-label dose titration. Patients demonstrating both a symptomatic benefit and blood pressure improvement following titration continued on open-label Droxidopa for a 1-week run-in period prior to being randomized on a 1:1 basis to continue on active drug or be withdrawn to placebo. The 101 patients enrolled in the blinded study had a mean score on Item 1 of the OHSA scale of 2.1 at randomization. At the end of the 14-day blinded treatment period, patients in the placebo arm had an average OHSA score of 4.0, or a mean change (increase) of 1.9 units from randomization. Patients in the Droxidopa arm of the trial had a mean score of 3.5 at the end of the two week treatment period, reflecting a mean change from randomization of 1.3 units resulting in a 0.6 unit difference (p=0.51) between arms.
Dose Titration 1-Week on Drug Blinded Study
Change Change Change
from Change from from
BL to from Rndm BL to
End of End of BL to End of End of End of
(BL) Titrtn Titrtn Rndm Rndm Study Study Study
------ ------ ------ ------ ------ ------ ------ ------
Droxidopa
(n=50)
Item 1
OHSA 6.6 1.5 -5.1 2.1 -4.4 3.5 1.3 -3.1
Standing
SBP 87 109.1 22.6 106.3 19.4 98.8 -10 12.5
Supine
SBP 130.1 144.8 15 143.3 13.2 138.7 -4.6 8.6
Placebo
(n=51)
Item 1
OHSA 6.3 1.5 -4.9 2.1 -4.2 4 1.9 -2.3
Standing
SBP 88 112.4 25.5 101.1 12 96 -5.2 8.2
Supine
SBP 133 142 8.7 138.5 5.7 132.1 -7.7 -0.9
Significantly, the company's preliminary analysis revealed that nearly every secondary symptomatic endpoint in the trial was either supportive or strongly supportive of Droxidopa's therapeutic benefit, including standing short time, standing long-time -- and importantly the composite orthostatic hypotension activities of daily living composite score all of which were statistically significantly in favor of droxidopa. Clinical and patient global assessments of severe orthostatic hypotension at end of study were also strongly in favor of Droxidopa.
Preliminary Safety Results
As anticipated, Droxidopa proved to be safe and well tolerated at all dose levels, with no significant adverse events or treatment related withdrawals in the Droxidopa arm.
The most common adverse events noted in the clinical trial were: supine hypertension at 12% for Droxidopa and 6% for placebo; falls at 2% for Droxidopa and 12% for placebo; and headache at 2% for Droxidopa and 8% for placebo.
"There is a long history of successfully treating neurogenic orthostatic hypotension with Droxidopa -- in the clinic, in the Japanese market, and here in the US through a long-standing compassionate use program," says Dr. Horacio Kaufmann, Professor of Neurology and Medicine at NYU School of Medicine, and investigator for this trial. "Having studied Droxidopa extensively, I firmly believe that this agent has a significant and clinically meaningful therapeutic benefit. I am looking forward to continuing to work with Chelsea to confirm these findings and bring Droxidopa to the US market. I have no doubts that patients suffering from symptomatic NOH in the US should have Droxidopa available."
http://finance.yahoo.com/news/Chelsea-Therapeutics-Reports-pz-585498803.html?x=0&.v=1
Chelsea's hypotension drug likely to meet trial goal
* What: Data from late-stage neurology drug
* When: Late September
* Analysts expect the trial to meet main goal
By Anand Basu
BANGALORE, Sept 23 (Reuters) - Chelsea Therapeutics' (CHTP.O) experimental neurological disorder drug, which is already approved in Japan, is expected to show high efficacy and a clean safety profile in a U.S. late-stage trial.
Data from the first of two late-stage trials for Chelsea's drug, Droxidopa, is expected in late September and the stock has risen more than 3 times over the last six months in anticipation of positive news.
Droxidopa, which aims to treat symptomatic neurogenic orthostatic hypotension (NOH) -- a neurological disorder that causes blood pressure to drop when the afflicted person assumes a standing position -- has a fast track status from U.S. health regulators.
"The drug is already approved in Japan for over 10 years, so the drug works, the question is execution risk in clinical trial," Wedbush Morgan Securities analyst Liana Moussatos said. If approved, Droxidopa could easily replace the current standard-of-care midodrine for the treatment of NOH, based on its better safety profile.
Currently the use of midodrine is limited due to concerns of supine hypertension -- an excessive increase in blood pressure, especially when lying down.
"The side effect profile for Droxidopa is far superior to midodrine, and it is also more effective. For a safety point of view this is about as clean as drug you can get," Roth Capital Partners analyst Andrew Vaino said.
About 70 percent of patients using midodrine encounter hypertension, but in Droxidopa that portion drops to about 1 percent, he said.
Vaino, who has a "buy" rating and $7 price target on Chelsea shares, expects the company to launch the drug in the first quarter of 2011. Wedbush's Moussatos, who expects the U.S. Food and Drug Administration to approve the drug in the fourth quarter of 2010, sees peak Droxidopa sales of about $275 million in the United States.
Some investors have voiced concerns of a potential placebo effect -- a genuine improvement in health driven by psychological expectations of a benefit and not due to the physiological effect of a given treatment -- but Moussatos is unperturbed.
During the initial open label titration phase all patients received the drug, but after 14 days half of them got the drug daily while the rest got a dummy drug.
"Investors are concerned that 14 days without Droxidopa treatment may not be long enough for the higher dose to wash out and NOH symptoms to return," said Moussatos, who has an "outperform" rating and $13 price target on Chelsea shares.
But the analyst believes that symptoms usually re-appear within 2-3 days after a patient stops taking the drug -- leaving plenty of time for the symptoms to re-emerge and differentiate the placebo patients from the drug patients. (Reporting by Anand Basu in Bangalore; Editing by Anthony Kurian)
http://www.reuters.com/article/marketsNews/idCNBNG53301320090924?rpc=44
RIGL -There was a Barrons article on the 18th about RIGL being a takeover target. May be the reason.
http://online.barrons.com/article/SB125322204396020853.html
ARIA -
You have this concept backwards—the completion of the interim look without the trial being stopped is slightly bearish. The best-case scenario in which the drug was exhibiting overwhelmingly strong efficacy has been removed from the universe of possible outcomes, which outweighs the corresponding removal of the worst-case scenario in which the drug was actually harming patients.
ARNA - Lorcaserin is intended as a monotherapy and will probably be submitted to the FDA as one so the lack of a study with phentermine should not be an issue. For marketing purposes ARNA may probably want to conduct a trial with phentermine.
Obesity drugs - ARNA looks like its approvable because of its safety profile but marketing the drug will be very difficult since its not clear which patient will benefit from the drug. Even with exercise the difference from placebo is only 4% and the ARNA presentation slides did not specify which patients benefited the most.
Regarding Qnexa as has already been pointed out by others on the board, if physicians prescribe topiramate with phentermine it will be a cheaper alternative than Qnexa. So there's risk in investing in Qnexa.
Regarding Contrave, some of the side effects seem serious and the first contrave trial did not achieve either of the FDA's benchmarks.
http://ir.orexigen.com/phoenix.zhtml?c=207034&p=irol-newsArticle&ID=1256571&highlight=
IMO none of the three drugs, lorcaserin,qnexa and contrave, will be blockbusters and the door is still open for a drug for obesity. Each of the drugs has a drawback that makes it risky for a bigger company to partner and a potential partner may hold off until the FDA Or maybe the government should just increase the tax on sugar and fatty foods and offer tax breaks on healthier foods.
RIGL - Agree that company is a good prospect. Though I imagine a 30% dilution is going to cause some downward pressure on the stock. I was surprised that ARRY's RA failure did not give it a boost.