Sunday, October 19, 2014

Amicus Therapeutics (FOLD) Announces Additional Positive Phase 3 Data Late Sunday Night

Amicus (FOLD) announced additional positive phase 3 results from its Phase 3 trial in Fabry disease using its lead compound Migalastat late Sunday night. I've provided an insert of the release below and expect shares to move up Monday morning due to the additional positive data. Across All Subgroups, Patients Treated with Migalastat Compare Favorably to Natural History on Kidney Function (eGFR) Additional GL-3 Data Further Validate Assay for Identifying Patients with Amenable Mutations CRANBURY, N.J., and SAN DIEGO, Oct. 19, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced additional positive data from a Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") in Fabry disease patients with amenable mutations. In a poster at the American Society of Human Genetics (ASHG) Annual Meeting, Daniel G. Bichet, M.D., M.Sc., Professor, Department of Physiology, University of Montreal, presented results from patients in Study 011 including those who continued on migalastat in an open-label extension (Study 041). Assessment of kidney function by various measures of glomerular filtration rate (GFR) for patients receiving migalastat in Study 011 for at least 18 months and continuing migalastat treatment in Study 041 showed continued stability of kidney function for an average of 32 months. Decline in kidney function is a key cause of morbidity and mortality in patients with Fabry disease. Measured (iohexol) GFR (mGFR) showed stability over 18-24 months in Study 011 but was not collected in Study 041; mGFR was previously reported with topline Study 011 results. Mean Annualized Change in GFR (ml/min/m2/yr) (SEM) Over an Average of 32 Months with Migalastat in 011 and 041 Estimated GFR (eGFR) (CKD-EPI) (n=41) -0.20 (0.60) eGFR (MDRD) (n=41) +0.63 (0.08) Stratifying patients for gender and baseline proteinuria demonstrated that patients treated with migalastat experienced less decline in kidney function than untreated patients from a previously published natural history study1. Dr. Daniel Bichet, Full Professor and Section Head, Renal Function & Transport Physiology, University of Montreal, said, "Baseline proteinuria levels are among the most predictive indicators of disease prognosis and kidney function decline in Fabry patients. The data presented today show that when comparing patients with similar levels of proteinuria, patients treated with migalastat are more stable in their kidney function versus untreated patients. These results are very encouraging for migalastat as a treatment for Fabry patients with amenable mutations." Data from a subgroup analysis comparing the change in GL-3 substrate levels between amenable patients and non-amenable patients based on the GLP HEK cell assay provided additional validation of the sensitivity of the GLP HEK assay for identifying patients who will respond to migalastat monotherapy. Overall, patients with amenable mutations had declining levels of GL-3 when treated for six months with migalastat. In contrast, patients with non-amenable mutations had no change or increasing levels of GL-3 after six months of migalastat treatment. "We are pleased to present these additional Phase 3 results in a scientific forum. With 32 months of data, Fabry patients treated with migalastat exhibit long-term stability in their kidney function. These results contrast with the decline in kidney function reported in natural history studies. Decline in kidney function is one of the primary causes of morbidity and mortality in Fabry patients," stated Dr. Jay A. Barth, Chief Medical Officer of Amicus Therapeutics, Inc. "The additional data on substrate reduction show that we can accurately identify patients who may benefit from migalastat. We look forward to meeting with regulatory agencies starting this quarter as we work to make migalastat available for all amenable Fabry patients as quickly as possible."

Thursday, October 9, 2014

Targacept (TRGT) provides update on partnership

Targacept (TRGT) filed an 8K today which stated that their partnership with AstraZeneca was terminated including their Alzheimer's compound AZD1446. This is pretty shitty news for the company as the only glimmer of hope after their recent two phase 2 drug failures was this partnership. The company still has a ton of cash but no real value in the pipeline so expect them to waste the money on an in-license opportunity (Teva just announced they are shedding around 14 development programs under their recently announced restructuring so that could be an opportunity). I currently own 10,000 shares of Targacept which isn't good with this announcement.

 I really like this book: The Biotech Investor's Bible

Tuesday, October 7, 2014

Trading Opportunity - Oxigene (OXGN)

Oxigene announced that will present Phase 2 data for its lead compound Fosbretabulin to treat Ovarian Cancer on November 9th at the International Gynecologic Cancer Society meeting. The company also noted that "additional data are expected to be included in the conference presentation" which tells me that overall survival may be announced at the meeting. The company already reported PFS improvement of 31.5% from the trial but a statistical improvement of overall survival would be a major catalyst for the company as any pivotal trial would likely require overall survival as the primary endpoint. I've provided a copy of the abstract for the meeting below which includes an overall conclusion from the sponsors. Abstract: RANDOMIZED PHASE 2 EVALUATION OF BEVACIZUMAB VERSUS BEVACIZUMAB/FOSBRETABULIN IN RECURRENT OVARIAN, TUBAL OR PERITONEAL CARCINOMA: A GYNECOLOGIC ONCOLOGY GROUP STUDY Aims The Vascular disrupting agent (VDA) fosbretabulin tromethamine selectively targets pre-existing tumor vasculature causing vascular shutdown leading to cell death and necrosis. Anti-angiogenesis agents like bevacizumab, a humanized anti-VEGF monoclonal antibody, might prevent revascularization after/during VDA treatment. Methods Patients with recurrent/persistent epithelial ovarian, tubal, or peritoneal carcinoma; measurable or detectable disease; and < 3 prior regimens were randomized to bevacizumab (15 mg/kg IV q 3weeks) or bevacizumab (15 mg/kg) + fosbretabulin (60 mg/m2) IV every 3 weeks until disease progression or toxicity. Randomization was stratified by disease status (measurable vs. non-measurable), prior bevacizumab, and platinum-free interval. The primary endpoint was progression-free survival (PFS). The study was designed with 80% power at a 10% level of significance to detect a hazard ratio (HR) reduction of 37.5%. Results The study enrolled 107 patients. Median PFS was 4.8 and 7.3 months for bevacizumab and bevacizumab+ fosbretabulin, respectively (HR = 0.685; 90% 2-sided CI=0.47 ~1.00). The proportion responding to bevacizumab was 28.2% (90% CI 16.7 ~ 42.3%) among 39 patients with measurable disease and 35.7% (90% CI 23.5 ~ 49.5%) among 42 patients treated with the combination. Adverse events (> grade 2) were more common in the combination particularly hypertension (35% versus 16%). There was one grade 3 thromboembolic event with the combination. One intestinal perforation in the bevacizumab arm was observed. Conclusion Based on the PFS and tolerability of these two anti-vascular therapies, further evaluation is warranted for this chemotherapy-free regimen. Fosbretabulin in combination with bevacizumab may double the risk of hypertension.

Thursday, October 2, 2014

Lilly (LLY) gives up on Lupus Drug providing less competition for Immunomedics (IMMU) and Anthera (ANTH)

Lilly (LLY) terminated the development of Tabalumab which was being studied for potential treatment of systemic lupus erythematosis (SLE) after the drug did not show efficiency in two phase 3 trials.

Immunomedics (IMMU) and Anthera (ANTH) have drugs in phase 3 trials focused on SLE so this is good news on one hand as it's less competition but bad news on the other as it highlights how difficult this disease is to treat and the prospects of successful trial results of their compounds.  Immunomedics is expected to report top-line results of its compound in Q1 2015.

I own shares of both companies as I'm willing to make the gamble as the upside is significant for both companies.

Wednesday, October 1, 2014

Anthera Pharmaceuticals (ANTH) provides update on partner discussions for lead compound

Anthera (ANTH) announced after the bell that they are in partnership discussions for its lead compound Blisibimod.  Blisibimod is a potential treatment for Lupus and IgA nephropathy and the company said the discussions were specifically related to partnerships for the compound outside of the United States.  This is a very positive development as a partnership with a reputable partner would show that due diligence was done on both the prospects of the compound and the credibility of the leadership team and company.  Anthera has a mere market cap of $40 million with a Phase 3 drug to treat an indication that desperately needs improved options - Lupus.  This tells me one thing - no one believes in the compound or the company.  Firming up a solid partner would help alleviate these concerns and could send the stock much higher.

I currently own 6,000 shares of Anthera but probably won't add to his position until this story plays out.


Tuesday, September 30, 2014

Merrimack (MACK) Reports Good News

Merrimack (MACK) provided updated clinical date on its compound MM-121.  The drug initially showed a lack of statistical benefit in Phase 2 trails but the biomarker data has been analyzed and showed that the compound increased Progression Free Survival in patients with biomarker positive metatastic breast cancer by 74%.  This is encouraging news for both the company and patients and I'd expect Merrimack to provide a strategic update on a pivotal trial in this patient subgroup in the future.


Monday, September 29, 2014

Daily Dose - News on Tonix (TNXP), Exelixis (EXEL), and Array (ARRY)

Tonix (TNXP) reported results for it's trial of its lead compound to treat Fibromyalgia.  The drug failed its two primary endpoints though it did show a 30% improvement vs. placebo on pain improvement but this would be expected as the compound is a variation of flexeril, a drug used to treat pain.  I'm getting out of my position as I believe this stock is dead in the water.
Check this out!
Exelixis (EXEL) reported results for it's trial in partnership with Roche of Cobimetinib in patients with BRAF mutation-positive advanced melanoma.  The trial met its primary endpoint of Progression Free Survival which was 9.9 months vs. 6.2 months in the control arm.  Roche has already filed to market the drug in Europe and expects to file an NDA with the FDA by the end of the year.  Exelixis has an option to co-promote the drug in the United States.  I'm holding onto my shares of Exelixis as I believe there's a chance the company could be acquired by Roche.
Check this out!
Array (ARRY) reported phase 2 results for it's trial of Binimetinib in NRAS Melanoma.  PFS was 3.6 months in a group of patients with a very poor prognosis.  The drug is currently in a phase 3 trial with results expected next year.  I'm holding onto my shares of Array as I hope for positive results in the phase 3 study and a subsequent NDA filing next year.

Thursday, September 25, 2014

Trading Opportunity - Tonix Pharmaceuticals (TNXP)

Tonix Pharmaceuticals is due to report top line results in it's pivotal, phase 3 trial of it's lead compound, TNX-102,  as a treatment of Fibromyalgia.   The compound is a reformulated delivery of cyclobenzaprine (many of us with back pain know it as flexiril) which allows the drug to be delivered directly into the blood stream by placing the pill under the tongue.

The market isn't giving it much of a chance with a market cap of only $131 million as of the time of this post but the stock appreciation upside is massive if the results come back positive.  If negative, you will see just the opposite result as there isn't much of a pipeline outside of TNX-102.

I've got 1,000 shares and I may add another 1,000 tomorrow and roll the dice for positive results.

Good luck on your biotech investments and hope everyone has a great weekend!


Rexahn (RNN) receives orphan drug status for lead compound

Rexahn announced they received orphan drug status from the FDA for it's lead compound targeting pancreatic cancer.  The stock is up over 7% in afternoon trading which is typical for such a release but the reality is orphan drug status is worthless if the drug isn't approved.  I'd sell on the uptick and buyback when reality sets back in.

Rexahn receives orphan drug status for lead compound


Tuesday, July 15, 2014

Biotech Investing: Question regarding best emerging biotechs

Which emerging biotech  best for future trade?

Regards,
Nestor E

Hi Nestor,

Yellen's comments didn't do the biotech industry any favors today but it does provide some buying opportunities.   I don't give investment advice but I have purchased a ton of Array (ARRY) and Merrimack (MACK).  They both have solid pipelines and Array has an exceptional list of partners.  They both also have over $100 million in cash to work with.  One high risk but possible high reward opportunity is Oncogenex (OGXI).  They will announce phase 2 results of their compound in bladder cancer this quarter.  If successful, the stock will move as the compound is being testing in a wide variety of cancers with readouts over the next few years.

Thanks for the question Nestor and good luck investing!

Monday, July 14, 2014

Adam Feuerstein responds to my question regarding Mannkind's Alfrezza label

He provides a detailed response to my question on today's article.

I continue to be neutral on Mannkind between their lack of partnership, label, and financial weakness. 

Wednesday, May 28, 2014

Daily Dose - Vivus , Array Biopharma

Vivus (VVUS) may receive an offer from Aspen Investments to purchase the company for $640 million.  Aspen filed with the SEC that it currently holds a 9.65% stake in the company and may make an offer as soon as June 13th for the biotech company.  That would be a premium of approximately 35% from yesterday's close but less than half the one year highs.  Vivus has two approved drugs - Stendra for erectile dysfunction and Qsymia for weight loss.  Vivus licensed Stendra in the US and worldwide but has been unable to secure a partner for Qsymia in the United States which has led to extremely disappointing sales results since the drug was launched over a year ago.  Aspen Investments just incorporated a few months ago so I'm not sure how credible the offer is and if it will lead to additional bids.  I currently own 6,000 shares and I'm long based on hopes that Vivus would secure a marketing partner for Qsymia or be purchased by a big pharma company so maybe these hopes will turn into reality now that there's some interested in the company. 

Array locked up yet another quality partnership.  They are partnering with Biogen on inhibitor discovery and development to target autoimmune diseases.  Array will receive funding for three years, potential milestone payments based on achieving certain development and commercial milestones, and royalties on future sales of approved products.  Array already has a strong list of partnerships including compounds in phase 3 trials with both AstraZeneca and Novartis.  The company carries a market cap of only $543 million and a deep pipeline which is why I have a position of 20,000 shares in the company. 

Tuesday, May 27, 2014

Mannkind – The good, the bad, and the ugly


Mannkind (MNKD) and its investors received very good news last month when its inhaled insulin drug for diabetes, Afrezza, received an overwhelmingly positive FDA advisory panel vote.  The panel voted 13-1 in favor to approving the product in type 1 diabetes and 14-0 in type 2 diabetes.  The drug had been rejected by the FDA and received complete response letters twice before so it looks like the third time may be a charm for Mannkind.  The positive panel vote was a surprise to the market after the FDA released briefing documents that included concerns over efficacy versus currently approved products and safety issues.  The FDA was also likely surprised by the positive vote as it subsequently pushed back the PDUFA date three months to July 15 in order to provide the agency time to for a full review of the application.  The approval of Mannkind is likely due to the overwhelmingly positive ADCOM vote as the FDA rarely goes against the panel recommendations.  Approval is good news for diabetes patients as it offers another treatment option for their disease. 

The bad news for Mannkind and its investors is just how many diabetes patients will use Afrezza as their treatment option.  This won’t be the first inhaled insulin product that’s hit the market and the last drug was a commercial disaster.  Pfizer won approval and introduced its inhaled insulin product Exubera in 2006 but only achieved $12 million in sales (no, I’m not missing a 0, that’s twelve million) in its first year despite a strong investment in launch including a television ad campaign.  After only a year on the market, Pfizer dumped Exubera and took a $2.6 billion write-off.  Will Afrezza meet the same fate and what went bad on commercialization? 

Yes, the Afrezza device is much smaller and more convenient to carry and use than what was referred to as the “bong” but was this sole reason for Exubera’s demise?  There’s the argument that inhaled insulin will be a blockbuster because diabetics hate using needles but if this were true, wouldn’t these needle fearing aichmophobiacs have used the “bong” regardless of it’s bulky size?  The FDA approved it for both type 1 and type 2 diabetes so the drug did work.


The really bad news and reality is that Afrezza will face many of the same commercial challenges as Exubera.  These include a restrictive label, premium pricing and coverage challenges, marketing to a broad range of physicians, and lack of superiority to existing products. 

After reading through the FDA briefing documents, my guess is that the Afrezza label will look much like Exubera’s.  This could include a pulmonary test prior to initiating therapy and possibly every six months thereafter, restrictions for patients with lung diseases such as COPD and asthma, and warnings for patient groups such as pregnant or nursing mothers and children.  These restrictions will significantly impact the size of the potential patient market. 

Exubera carried a 30% premium without a superiority profile over injected insulin that led to insurance coverage challenges.  Managed-care companies scrutinize new drugs now more than ever for their cost vs. benefit and Afrezza wasn’t statistically superior to current injectable treatments, which will likely put the drug in a higher co-pay tier than it’s injectable rivals.  The higher co-pay will be another negative for commercialization. 

Lastly, it’s going to be very expensive to market a drug for an indication that’s currently prescribed by just about every family doctor in America.  Not only do you have to sell the benefits of inhaled insulin to an extremely fragmented network of doctors, you have to educate them on how to use the device so that they can teach their patients.  Also, how keen will already overworked family doctors be to actively subscribe an alternative product that requires a preliminary lung test and follow-up tests every six months?  The marketing challenges will make it all but impossible for Mannkind to go it alone, which leads me to the ugly…

How can things get more ugly than the commercial challenges?  It can!  It’s a combination of an astronomical market cap, horrible balance sheet, and lack of a commercial partner. 

I have reviewed hundreds, if not thousands, of company financials over my professional and investment career and I can’t think of another company with such an over-inflated market value than Mannkind.  Its market cap currently exceeds $2.5 billion dollars while having over $200 million in liabilities (including a $100 million note maturing mid-2015), only $35 million in cash, and a measly $17 million in owner’s equity.  The company has over 388 million shares outstanding with plans in place to increase this count in the coming months via a $50 million ATM agreement.  Mannkind lost over $50 million last quarter, which is shocking since the two pivotal trials for Afrezza ended last year.  They are in a very weak financial position to negotiate a deal with commercial partners and potential suitors will take advantage of this, if a commercial partner can be secured at all due to the many challenges listed under the bad section of this article. 

Mannkind - The good, the bad, and the ugly.  There’s plenty of each with the Mannkind saga and only time will tell how this movie ends.  My recommendation is to steer of the stock until the story unfolds in the coming year. 



Friday, May 23, 2014

The BiotechInvestor.com Inbox - Oncosec

Lance Werner @lancewerner17
@Biotechinvesto9 Wanted to see if you have any thoughts on a new one I'm checking out $ONCS? They have some strong results coming 6/2 @ ASCO

Thanks for the tweet Lance.  I pulled the abstract (below) for the upcoming ASCO conference and the results of Oncosec's (ONCS) compound in the phase 2 trial for Melanoma look encouraging.  29 patients have been enrolled and the overall response rate is 33%, with 11% complete response.  I would expect a press release on the day of the abstract which could possibly move the shares.  Let's circle back when the results are formally announced. 
Abstract:
Background: Interleukin-12 (IL-12) promotes anti-tumor activity through multiple mechanisms, including augmentation of adaptive and innate immune responses. Intratumoral (IT) delivery of IL-12 via electroporation (EP) avoids systemic toxicity while promoting systemic antitumor immunity. This phase 2 study explores the systemic efficacy, clinical response and safety of IT plasmid IL-12 injection (pIL-12) followed by EP in patients (pts) with advanced melanoma. Methods: This single-arm, open-label phase 2 study plans to enroll 30 pts with in-transit or M1a melanoma. One treatment cycle consists of IT pIL-12-EP on days 1, 5, 8 in up to four lesions per cycle. A maximum of four cycles at 12-week intervals are allowed. ORR was assessed by a modification of RECIST for cutaneous lesions with restaging performed every 12 weeks. The primary endpoint is best ORR within 24 weeks of first treatment. Pre- and post-treatment tumor biopsies were obtained in all patients. Ongoing analyses to assess safety and emerging efficacy data are being utilized to inform future studies. Results: 29 pts have been enrolled and have received at least one treatment cycle. The ORR is 33% (9/27), with 11% CR (3/27). Regression of non-injected lesions was seen in 62% (13/21) of pts with evaluable lesions. Transient pain (56.5%) and inflammation (17.4%) at the treatment site were the most common grade 1/2 drug-related adverse events (AEs), with no grade 3/4 drug-related AEs. Exploratory analyses indicate a doubling of intratumoral NK cells from pre-treatment through day 11 and at day 39, and increased frequency in activated circulating NK cells. Conclusions: Local treatment with pIL-12-EP is well tolerated without severe systemic side effects. Regression of treated and non-treated tumors suggests successful induction of systemic anti-tumor response. Local and systemic increases in NK cells are consistent with the expected pharmacodynamic effect of IL-12. Based on these data, an expansion protocol to evaluate increased treatment frequency is planned for melanoma patients. Clinical trial information: NCT01502293.

Wednesday, April 23, 2014

Daily Dose - Spectrum Pharmaceuticals, OncoGenex Pharmaceuticals

The Nasdaq Biotech Index pulled back 1.54% after two strong days of gains.  Here are some highlights for the day.

Spectrum (SPPI) announced that it's multiple myeloma treatment met it's primary endpoint and the company plans to submit an NDA in the 3rd quarter of this year with an expected PDUFA date in 2015.  The drug is an improved form of the chemotherapy drug melphalan.  If approved, analysts believe the peak sales of the drug could be as high as $50 million annually.  This is another near term potential catalyst for Spectrum on top of it's August 9th PDUFA date for it's lymphoma drug Belinostat.  The company already markets four drugs with sales of $155 million in 2013 and a number of other drugs in it's pipeline and partnerships with Biogen, Allergen, Bristol-Myers Squibb and others.  With a market cap of $453 million I consider the company to be a great value which is why I hold a long position in the company. 

OncoGenex (OGXI) announced the company and Teva received the Fast Track designation for it's partnered compound Custirsen in 2nd line castration resistant prostate cancer.  The drug already has Fast Track status for the same drug in first line CRPC and lung cancer, all of which are currently in phase 3 trials.  I wouldn't too much value on the designation as it's worthless if the drug isn't successful in it's phase 3 pivotal trial.  They expect enrollment to be complete in the 2nd line CRPC trial in the 2nd half of this year so I would expect top line results in the middle of 2015.  In the meantime, they should report top line results of the first line CRPC any time within the next few months.  They already announced the required number of events (deaths) have occured and that they are now analyzing the data so fingers crossed!

Hope everyone is having a great week and feel free to email or tweet me any questions,

Shane

Tuesday, April 22, 2014

Biotech Stocks make strong gains for second straight day - why?

The Nasdaq Biotech Index is up 3.4% after a strong gain on Monday after reports over the weekend that Pfizer had approached UK based AstraZeneca with a $101 billion takeover bid.  The 25% premium combined with the potential for the largest acquisition in the industry's history has many biotech investors excited about the prospects of further consolidation at premium prices.

I'd also keep an eye on oncology focused biotech as the biggest showcase event of the year, ASCO, is coming up at the end of May.  I'd expect press releases in the coming weeks on abstract presentations and a general run-up in these stocks going into the conference.

I'll be reporting more on ASCO as it nears. 

Thursday, April 10, 2014

Another reason to believe Mannkind shares are overvalued

I already believe Mannkind is overvalued due to it's $2.5 billion market cap, weak balance sheet, and lack of commercial partner.  Now they have another possible inhaled insulin competitor with a planned phase 3 trial next year.  Take a look at Adam's article for the details. 

Tuesday, April 8, 2014

Sunesis announces results at AACR - Wedbush reiterates $10 price target

As expected, Sunesis (SNSS) reported positive preliminary results of it's lead compound, Vosaroxin, in a phase 1b/2 study in AML and MDS.  The drug, combined with decitabine, had an overall response rate of 67% in the 24 patients treated.  Webush commented on the results and reiterated it's $10 price target on Sunesis. 

Monday, April 7, 2014

AACR Trading Opportunity - Sunesis

The American Association of Cancer Research 105th annual meeting is underway which sometimes provides opportunities for short term trades ahead of press releases for data presentations.  I think Sunesis (SNSS) is such an opportunity as updated phase 1b/2 data on it's lead compound, Vosaroxsin, in AML and MDS will be presented tomorrow.  I pulled the abstract and it's showing an 67% response rate which is a solid result and I'd expect a press release tomorrow to communicate these results.  I purchased 5,000 shares ahead of the data presentation. 

Friday, April 4, 2014

Exelixis upgraded by Piper Jaffray

Exelixis (EXEL) received an upgrade from Piper Jaffray this morning to buy with a price target of $11.  The firm believes that the market has "washed out" it's lead drug in phase 3 development for prostate cancer.  As I recently blogged, I believe in Exelixis' long term potential as they have a number of other shots on goal including phase 3 trials in HCC and RCC with the same compound as well as a phase 3 compound partnered with Roche in Melanoma which will report results later this year. 

Thursday, April 3, 2014

Second Quarter Biotech Trade Catalysts

There are a number of anticipated biotech trade catalysts in the second quarter which could either make you rich or put you in the poor house.  Let's take a look at a few and I'll provide my positions in the stock.

Mannkind (MNKD) - Yes, they almost had a sweep of yes votes at the Adcom meeting earlier in the week but the upcoming PDUFA decision might not be so convincing.  I believe that Afrezza will receive approval for both type 1 and type 2 diabetes but I think the label and post-marketing studies will be disappointing for investors and will either keep the stock in neutral or send it down.  Take a look at the price action of Chelsea's after it received Northera approval a few months ago to get an idea of where my logic.  I currently hold naked calls of Mannkind at $7 and $10 so I'm semi-short the position (I don't think the stock will go above these prices at the time of expiration).

OncoGenex (OGXI) - They should be reporting phase 3 results of it's lead compound in prostate cancer anytime this quarter.  The company is partnered with Teva on the trial and has a market cap of only $159 million so if the trial meets its primary endpoint of overall survival, I think it's really going to move.  The drug was successful in it's phase 2 trial and they used the same design in the phase 3 trial so they just need a repeat of performance which is why I have confidence in the trial and hold 2,000 shares of the company.

Exelixis (EXEL) - They were hammered last week after reporting that their phase 3 trial of Cometriq in prostate cancer wasn't stopped early for efficacy.  I still think Cometriq could meet it's primary endpoint of overall survival and the company has additional phase 3 trials in HCC and RCC which will read out in 2015-2016 so there are other shots on goal.  I own 26,000 shares so I have a great deal of confidence in the companies long-term potential.

Merrimack (MACK) - The company expects to report phase 3 results of it's compound, MM-398, in pancreatic cancer.  If the trial meets it's primary endpoint of overall survival, I would expect a big move as it validates the companies delivery technology and also provides a huge opportunity to treat second-line pancreatic patients which currently have limited treatment options.  The company has a number of other compounds in development, a partnership with Sanofi, and a market cap of only $529 which makes it one of my favorite holdings at 15,400 shares.

Anthera (ANTH) - They will report phase 2 results of it's lead compound in Lupus.  I visited the family last week and we went to the casino and I played craps and roulette.  My investment in Anthera feels very similar to the bets I placed at the casino.  Let's hope that the outcome is better.....I own 3,000 shares of this small company with a small market cap of $66 million.  Lupus patients have limited treatment options so a positive result would be huge for patients and for this little biotech.

Navidea (NAVB) - The company has a PDUFA date with the FDA of June 16th to expand it's mapping product into neck and head cancers.  If approved, it would drastically expand the potential market for lymphoseek and provide much needed revenue growth to help fund it's two other phase 3 imaging studies in Alzheimer's and Parkinson's. 

If you own any of these stocks I wish you luck in the coming months and I'll be sure to report the outcomes of these catalysts!  Biotechinvestor.com

Daily Dose - Mannkind, Cyclacel, and a Vivus downgrade

I'm sorry for being out MIA for the past week but just returned from a trip back east to see my family.  There has been a lot going on since I left for my trip. 

Mannkind MNKD won a convincing  13-1-1 and 14-0 advisory panel for it's inhaled insulin drug Afrezza in type 1 and type 2 diabetes, respectively.  That took just about everyone by surprise as the stock soared after the vote.  I predicted a positive vote in type 2 but was shocked by the type 1 vote.  I also predicted a strict label which I'm sticking to as the panel echoed the FDA's concerns for patients with asthma and COPD.  I'd also expect two post-approval studies for lung cancer though Mannkind is already performing a phase 3 study to evaluate the long-term effects so this might alleviate some of those requirements.  I still believe Mannkind is overpriced as it's carrying a $2.5 billion market cap, lacks a commercial partner, has a negative book value, is burning through cash, and I expect an unfavorable label.  I guess time will tell how this story plays out.

Cyclacel CYCC announced a public offering of stock after the market closed today.  Management has been communicating that they have "plenty" of cash to get through their recruitment of their lead product in AML but looks like they are going to the well again.  They expect full enrollment in their phase 3 trial in AML in Q4 due to expanding the enrollment centers into Europe.  I only own 2,000 shares and the stock is down after hours due to the announcement. 

Vivus VVUS was downgraded by Piper Jaffray from $8 to a $3 price target.  They have concerns about the lack of sales traction of their obesity drug Qsymia and inability to land a large pharma partner.  I own 6,000 shares so I share in these concerns but I'm not giving up hope.  The new management team was able to land solid partnerships in the United States and abroad for their erectile dysfunction drug Stendra so I have confidence in their ability to land a partner for Qsymia (did you hear my Vivus management?   You stink at selling.   Close a damn deal before you burn through your cash!!!!!) 

Stay tuned for more from the Biotechinvestor.com

Friday, March 28, 2014

Mannkind Adcom Prediction

I predict a positive vote at Tuesday's ADCOM meeting for Mannkind MNKD with the following caveats based on the briefing documents.  I've provided some of the FDA reviewers comments below. 

-  Type 2 diabetes only (Type 1 not approved)
-  Limited use to restrict patients with lung disease (asthma, COPD, etc) or at high risk of lung disease (ie smokers)


In conclusion, the available data from clinical pharmacology studies for
AFREZZA does not conclusively support use in patients with underlying lung diseases.  The data from clinical studies will be considered along with the available limited PK results to develop
the final labeling recommendations.

Reviewer’s comment: The study results show that the primary objective of noninferiority
of Afrezza TI Gen2 to insulin aspart was met (noninferiority margin 0.4%). However, Afrezza TI Gen2 was statistically worse than insulin aspart. (Type 1 comment)
 
The FDA statistician also performed extensive sensitivity analyses for study 175 and
verified the Sponsor’s sensitivity analyses. In brief, the FDA statistician concluded that
sensitivity analyses supported the superiority finding. (Type 2 comment)
 




Thursday, March 27, 2014

Daily Dose - Mannkind, Threshold, Arena

Mannkind's MNKD day of reckoning is upon them.  They have their adcom meeting on monday for their inhaled insulin product which has already been rejected by the FDA twice.  Will the third time be a charm?  Maybe but between the chances that the drug gets approved, the commercial potential for inhaled insulin, and Mannkind's insanely high market cap....I'm not a buyer.  In fact, I'm short via naked January $10 calls as I don't see the company worth more than $3 billion in January even if the drug is approved.

Threshold THLD announced that their partner Merck KGaA is initiating a phase 1 trial of their lead compound, TH-302.  The drug is already in two phase 3 studies for pancreatic cancer and soft tissue carcinoma and a number of phase 2/1 studies.  I like Threshold based on TH-302's broad potential in a number of cancers, it's partnership with Merck, and relatively small market cap of under $300 million.  The downside is that Threshold is a one trick pony with TH-302 with nothing else of value in the pipeline.  I own 3,000 shares so we will see how these results play out next year.

Arena ARNA announced the initiation of a phase 2 trial of it's lead compound, Lorcaserin, in smoking cessation.  The company has a market cap of $1.4 billion and sales of Lorcaserin to treat obesity have been underwhelming since it's launch last year.  I feel the company is undervalued and hold a short position via naked July $10 calls.  There's no way this company will be worth north of $2.5 billion come July though there may be long-term potential for the smoking cessation opportunity.


Tuesday, March 25, 2014

Exelixis provides phase 3 prostate cancer trial update after hours

Exelixis EXEL had a busy day issuing press releases today.  The first came before the market opened with the announcement of European approval of Cometriq for MTC (a rare form of thyroid cancer).  The second came after the market closed which was an update on Cometriq's phase 3 trial in the much more lucrative prostate cancer indication.  The company was notified by the IDMC that the planned interim analysis of the trial has been completed and that the trial will proceed to its final analysis.  The good news is that the drug made it through the futility checkpoint.  The bad news is that the trial could have been concluded early if the results through the interim analysis were exceptional - so statistically significant through the current number of events (deaths) that the trial would be successful no matter how the rest of the events played out. 

Now we wait for the top line results in the coming months. 

Cyclacel reported earnings after the market closed with an update on phase 3 enrollment in AML

Cyclacel (CYCC) reported earnings after the market closed today and provided an updated on enrollment for it's phase 3 trial of it's lead compound to treat AML in elderly patients.  They have expanded into approximately 80 sites which brings total enrolling sites to approximately 120 after you included the 40 sites in the United States.  They have now enrolled 60% of patients in the trial and expect to complete enrollment by the end of the year with top line results in 2015.  They have an agreed upon Special Protocol Assessment with the FDA for this trial.  They are also evaluating the opportunity to begin a second phase 3 trial for the same compound to treat MDS and expect to provide an update on this opportunity later this year. 

I own a small position of 2,000 shares in Cyclacel.  The company has a small market cap of $68 million with a phase 3 trial expecting results in 2015 in an indication (AML) which needs improved treatment options so I believe the stock is a good value. 

Exelixis wins European approval for Cometriq in MTC; Phase 3 prostate results due soon

Exelixis ($EXEL) announced this morning it won European Commission approval for it's lead compound, Cometriq, in MTC (rare version of thyroid cancer).  This was widely expected after the CHMP issued a positive opinion in December though it has lifted the stock a few percent while the broader Nasdaq Biotech Index is down. 

The real news that will move the stock should come out within the next few months which is Cometriq's results in it's phase 3 trial in prostate cancer.  If successful analysts expect Cometriq sales could top $1.7 billion by 2020.  The company also has three additional active phase 4 trials for Cometriq in prostate (pain reduction), MTC (overall survival), HCC, and RCC not to mention a countless number of phase 2 trials for the compound. 

They also have a number of impressive partnerships including Genetech which expects to announce phase 3 results of a partnered compound, XL518, in Melanoma later this year. 

The company has a market cap of $1.26 billion which seems like great value based on the above near term catalysts and opportunities.  I own 13,000 shares of Exelixis so I have a great deal of confidence in the upside of this biotech. 

Monday, March 24, 2014

Celator Pharma announces earnings after hours

Celator (CPXX) announced year end 2013 and Q4 2013 earnings after the market closed today.  I listened on the call and have the following updates.

-  The company has a phase 3 trial enrolling in their lead agent CPX351 in secondary AML and expects enrollment to be completed by the end of the year.  They already have over 50% of planned enrollment completed in the targeted enrollment of 300 in the 41 sites currently active. 

-  Overall survival is expected in the beginning of 2016.  

-  They have a number of IST's (investigator sponsored trials) ongoing throughout the US including Fred Hutchinson in Seattle and Cincinnati Children's Hospital.  

-  The company has an extremely low market cap of only $86 million for a company with a phase 3 candidate.

-  It's one of the few biotech's trading near a 52 week low. 

I feel like Celator has a great risk vs. reward profile between it's $86 million market cap and phase 3 trial in secondary AML  I currently own 3,000 shares of Celator and may add to my position leading into the full enrollment of the phase 3 trial and top line results. 

Thursday, March 20, 2014

Ziopharm climbs 12% after $11 price target from Mizuho Financial Group

This may be the first Mizuho Financial Group price target that I've seen in the biotech industry in my 20 years of trading - I'd take this price target with a grain of salt.  Ziopharm has a drug in phase 2 trials which will report around the end of the year but they are a long way from commercializing anything (if ever) and already carry a heft $540 million+ market cap.

Aveo jumpes 19% in after hours trading after regaining rights of AV-203 from Biogen

Yes, you read the headline correctly.  Aveo JUMPED after Biogen gave the drug back to them when it should have gone down as it lost it's major pharma partner' support of the drug.  The drug just finished up a phase 1 safety study but it's many, many years away from possible commercialization (if ever).  On that note, I sold my 4,000 shares for $1.94 in after hours trading after buying them earlier in the week for $1.69 as a day trading strategy.  It worked out well for me but I'm dumbfounded by the optimism driving up the share price in after hours.  I expect the stock will come back down to earth in the coming weeks. 

Wednesday, March 19, 2014

European Marketing Authorization Application Update - Navidea Bio

Navidea (NAVB) provided an updated on it's ongoing European Marketing Authorization Application for Lymphoseek a few hours after the market closed today and the stock jumped over 5% in after hours trading.  The company presented oral explanations to the CHMP to address questions regarding the application.  The CHMP stated that it will continue it's review of the application though the company believes that the they are satisfied with the safety and efficiency for the breast cancer and melanoma indications so their review now turns to the head and neck cancer indications.  Unlike the FDA approval which only included use for breast cancer and melanoma, Navidea include the results of the head and neck cancer within the European application which weren't available during the FDA review.  Navidea has a supplemental NDA review target date on Lymphoseek for head and neck cancer on June 16th. 

I've been a long time investor of Navidea and believe that the upcoming catalysts, including EU approval, expanded approval in head and neck cancer by the FDA, and the expanded labeling sNDA at the end of the year, provide tremendous upside for this stock. They also have two phase 3 studies in imaging agents for Alzheimer's and Parkinson's which provide additional long-term catalysts and value. 

Trade Updates - Aveo BioPharma and Bind Therapeutics

Aveo (AVEO) - Purchased 4,000 shares at $1.69.  I don't necessary feel great about the companies near term prospects but they had approximately $120 million in cash at the end of the year and their current book value is $87 million so I felt it was a good value play.  They have an opportunity to in-license or hope for some positive developments in their preclinical program.  I'm selling back at $1.90.

Bind (BIND) - Purchased 1,000 shares at $12.10.  The company went public last year at $15 and is running two phase 2 trails of it's lead compound in Lung and Prostate cancer with results expected later this year.  They had over $80 million in cash at the end of Q3 and three outstanding partnerships - Amgen, Pfizer, and AstraZeneca.  With a market cap just under $200 million, this seemed like a great buy at this price. 

Tuesday, March 18, 2014

The BiotechInvestor Inbox - What do you think of Provectus (PVCT)?



 


What do you think of Provectus (PVCT)? Legit or snake oil? Moffitt Cancer Center thinks legit.

Thank you for the question EJ.  I'm not investing in Provectus.  I'm not sure about snake oil, but I don't invest a company with a $366 million market cap, no large pharma partner, traded on the OTC bulletin board, only $15 million in cash, and only 4 employees working for the company with their pay listed as $1.19 million EACH under yahoo's profile page which is ridiculous for a company of this size and in the development stage.  There are much better places to put my money and I'm definitely not going to put it in these guys pockets. 





Great article by Adam and The Street on today's Seeking Alpha article on Mannkind


Great article by Adam at The Street on today's Seeking Alpha article on Mannkind

Monday, March 17, 2014

Trade Updates - Rigel Pharma, KaloBios Pharma

The Nasdaq Bio Index is up a solid 1.48% to start off the week.  Oxigene is off to a strong start, up over 7% thus far for the day.  I added two new positions to my portfolio late last week.

I purchased 3,000 shares of KaloBios (KBIO) at $3.09.  The company is expecting phase 2 results in the 4th quarter for cystic fibrosis patients with chronic PA lung colonization.  They also expect phase 2 results on their trials in AML and MDS (Leukemia) in the 2nd quarter of 2015.  They have two solid partnerships with Sanofi and Novartis as well as $76 million in cash (end of Q4) which makes me feel better about investing in a company with a small market cap of $101 million.  They are a long ways away from getting a product to market but at $101 million and for the reasons mentioned above, they seem to be a great value. 


KBIO

 
3.07  


4,000   $3.0884   -$83.59   -0.68%   $12,280.00  


I purchased 3,000 shares of Rigel (RIGL) at $3.98 and to my surprise the stock jumped up over 13% by the end of the week so it's currently trading at $4.50.  They have phase 2 results in dry eye expected in the 2nd half of the year and they are also initiated a phase 3 study of their drug to treat ITP, an autoimmune disease that affects approximately 60,000 people in the United States.  They have a very strong cash position of $212 million as of the end of the year and partnerships with AstraZenenca, BerGenBio, and Daiichi Sankyo. 


RIGL

 
4.58  


3,000   $3.9792   $1,792.41   15.00%   $13,740.00



Thursday, March 13, 2014

Analyst calls out Aveo CEO on earnings call

After numerous clinical failures and a stock price that has lost over 75% of it's value in the last twelve months, Aveo's leadership (if that designation is appropriate) plans to use the $118 million in cash at the end of 2013 to invest in an early state compound that's not going to see human trials until 2015 and attempt to "partner" their later stage compounds which have thus far been failures.  Hats off to Brian Klein at Stifel, Nicolaus, & Company for asking the question that everyone else is thinking and see the response from the companies "leadership". 

Brian Klein - Stifel, Nicolaus & Company, Incorporated, Research Division
Okay. I guess, last question then is, was there ever any thought between you and the Board to essentially shut down all clinical activities that are being sponsored by AVEO and just out-license all of your products and return the cash and anything else you'll get from partners back to investors?

Tuan Ha-Ngoc - Chief Executive Officer, President, Principal Financial Officer and Director
Obviously, we're -- as we defined our strategy together with the Board, we have looked at all the past value creation. And obviously, that's our responsibility, to look all the various paths. And we agree, together with the board, that the path that is outlined today with our strategy will present the optimal path for value creation.

New Price Target - Chelsea Therapeutics

Chelsea (CHTP) ended the day up almost 14% after Wedbush put a $8 price target on the small biotech a month after it received it's first FDA approval for the drug Northera which treats symptomatic neurogenic orthostatic hypotension (it's a condition of dizziness of people with diseases such as Parkinson).  Wedbush believes that there is a strong chance of the company securing a partner which has been my bull thesis on the stock as well.  One thing that has me a bit worried about it's commercial potential is my recent conversation with a friend that runs a non-profit that supports folks with Parkinson's.  He had not heard of Northera which surprised me if the condition is prevalent in that population of patients as some of Chelsea's numbers have indicated.  He was very aware of the upcoming results of Abbvie's LCIG in Parkinson's.  Just some food for thought. 
CHTP

 
5.95  


2,000   $5.7426   $394.83   3.43%   $11,900.00  

Tuesday, March 11, 2014

Talk about timing - Oxigene reports positive topline results from randomized phase 2 study of Zybrestat - stock is up over 18%!

I wrote an article yesterday about the most overpriced and underpriced biotechs.  Oxigene (OXGN) was my pick for most undervalued due to it's upcoming phase 2 results of it's lead compound, Zybrestat, to treat Ovarian cancer.   Results are out and they are communicating positive topline results; stock is up over 18% in the last few minutes of trading.  


Monday, March 10, 2014

The Most Overpriced and Underpriced Biotech Stocks? Mannkind and Oxigene


Mannkind Corporation had a market cap of almost $2 billion and it’s my vote for the most overpriced biotech stock.  Most investors already know the story of Mannkind – Inhaled insulin product Alfrezza, already rejected twice by the FDA, prior inhaled insulin product launched by Pfizer was a commercial flop, and a third and possible final showdown with the FDA set to unfold in the coming month with a ADCOM meeting on April 1st and a PDUFA date for April 15th.  So many investors ask, “Is the third time a charm?” 

I would say that the chances of a successful approval are less than 50%.  The results that have been shared (emphasis on shared as the full results haven’t been released) have been mediocre at best.  The Phase 3 results in Type 1 diabetes was numerically worse than injectable Novolog and the results in Type 2 diabetes was statistically better than Novolog but patients gained weight on the drug.  We don’t have much safety data but we do know that the inhaled insulin causes a persistent cough that the company says is benign.  My real concerns are what I refer to as “clues” which make me wary of approval.  They include the company’s unwillingness to release the full results from the trial, their inability to secure a marketing partner (if the data was that great, why can’t they get someone to partner), and the prior two FDA rejections. 

Even if the drug is approved, then comes the challenge of commercializing inhaled insulin.  Will people use inhaled insulin or are the comfortable with their current product?  Remember, the needles are much smaller, less painful (if at all), and less expensive than they were when Alfrezza first went down the path to develop inhaled insulin.  Are people scared of the long-term effects of inhaling a medication and the impact on their lungs including the very serious and deadly lung cancer?  Can Mannkind land a formidable marketing partner?  If the pharma giant Pfizer was unsuccessful in marketing inhaled insulin, what makes anyone think that another company will be successful?  Will anyone want to pay a premium for inhaled insulin versus their current insulin product knowing that Alfrezza will cost significantly more than Novolog or the other products on the market? 

Are there any dirty secrets in the trial data?  Will Alfrezza receive a positive ADCOM vote?  Will the FDA approve Alfrezza?  Can they secure a marketing partner?  Will it be a commercial success?  It seems to me there is way to many questions to justify a market cap of almost $2 billion until some of these questions are answered which is why Mannkind is my vote for the most overpriced biotech stock. 

On the other end of the spectrum is OXiGENE which has a whopping market cap of approximately $12 million.  The company’s lead drug is Zybrestat which is a vascular disrupting agent currently focused on Ovarian and Anaplastic Thyroid Cancer (ATC).  The drug works by blocking the blood flow to the tumor that then kills the cancerous tumor cells.  The company has invested over $200 million to date in this approach to tackling cancer and hopes these investments will pay off in the upcoming readout of it’s phase 2 trial in Ovarian cancer. 

Zybrestat is currently in a randomized phase 2 trials in ovarian cancer in combination with Genetech/Roche’s Avastin.  Ovarian cancer leads to 14,000 deaths in the US each year and has a 5-year survival rate of only 47%, which has been largely unchanged since the 90’s.  The trial is being conducted in 67 clinical sites and has enrolled 107 patients randomized 1:1 to receive Avastin and Zybrestat or Avastin and placebo.  The primary endpoint is an improvement of progression free survival from 50% to 65%, which is expected to report in the first half of this year (so any day now).  The trial has already passed two independent safety committee reviews and a futility analysis.  

With an upcoming phase 2 readout in a Ovarian cancer population that’s in dire need for improved treatment options, a positive result would make OXiGENE’s $12 million market cap the underpriced bargain of the century. 

Thursday, March 6, 2014

Daily Dose - Navidea Bio, Rigel Pharma

The Nasdaq Biotech Index pulled back 2.62% today after strong gains earlier in the week. 

Navidea Biopharmaceuticals jumped 8.11% after announcing full year 2013 results and providing guidance of $5 million - $6 million in 2014 revenues for it's lymphatic mapping agent Lymphoseek.  The agent was approved March 2013 in the United States for breast cancer and Melanoma and has already captured 10% market share for this indication within it's first twelve months of sales.  I believe Navidea has a great deal of upside potential as there are a number of upcoming catalysts which can significantly expand the addressable market for Lymphoseek and create shareholder value.  There are TWO supplemental NDA reviews coming up for the agent; one to expand use for head and neck cancers and one to expand the labeling to support more broader use on June 16 and October 16, respectively.  Also, the European Medicines Agency is expected to make a decision on approval within the EU sometime this month.  These three events have the potential to significantly increase Lymposeek's addressable market in 2014.  Also, let's not forget that Navidea has TWO additional imaging agents for Alzheimer's and Parkinson's in Phase 3 trials expected to read out in 2015 and 2016, respectively.  I have two long positions in Navidea but I may add to this position in the coming months. 

NAVB Apr 19 '14
$2 Put
  0.20  


-100   $0.30   $914.01   29.62%   -$2,000.00

NAVB Jan 17 '15
$2 Put
  0.50  


-100   $0.538   $293.36   5.37%   -$5,000.00
Rigel Pharmaceuticals climbed over 10% today after announcing promising preclinical results of it's treatment, R118, in Peripheral Artery Disease.  The company also announced that the compound will now enter phase 1 human trials.  I feel like the jump might be a bit overdone since the drug is still many, many years from approval assuming the trials are successful but it is a company to keep an eye on and I may take a long position in the coming months.  The company does have a reasonable valuations of $395 million when you take into account they have $212 million in cash and some other programs with solid partners such as AstraZeneca.  It gives me great comfort when a major pharmaceutical player partners with a small biotech as you know they've done extensive due diligence into the company that's unavailable to the retail investor. 

Thursday, February 27, 2014

Daily Dose - Oncogenex, Merrimack, KaloBios

A number of buy recommendations came out this morning for some of my holdings and one reports earnings after market close. 

Oncogenex OGXI was upgraded to buy by CRT Capital with a $20 price target.  Oncogenex will report phase 3 results of it's prostate cancer drug, Custirsen, sometime in Q2.  The stock has a discounted market cap of $172 million for a company with three phase 3 and seven phase 2 trials running which is why I hold a significant position. 

OGXI

 
11.695  


5,000   $11.01   $3,377.46   6.13%   $58,475.00
KaloBios KBIO was upgraded to buy by JMP Securities with a $8 price target.  The company took a recent hit after it's flagship Asthma drug failed in a phase 2 study but the company has other compounds with promise and great partnerships with Sanofi and Novartis so I'm looking to buy at these levels.

Merrimack MACK will report Q4 and full year results after the bell today.  The stock has been on a tear since pushing back expected results from it's pancreatic cancer drug, MM-398, about six months due to longer overall survival trends.   Merrimack is my pick up the year as pancreatic cancer is one of the deadliest and has few treatment options (think the late Patrick Swayze) so a survival benefit would be outstanding for patients and for the Merrimack.

MACK

 
5.005  


15,400   $2.9977   $30,871.84   66.81%   $77,077.00