Monday, September 08, 2014

Keryx Biopharmaceutical's Kidney Drug Approved - What Now?

Keryx Biopharmaceuticals, Inc. (KERX-$17.01) announced that the FDA approved Ferric Citrate (formerly known as Zerenex) for the control of elevated serum phosphorus levels in patients with chronic kidney disease (CKD) on dialysis. The share price declined more than 5% on the news, however, on investor concerns that an unexpected safety warning – the drug package label must include the potential risk of “iron-overload” – could slow market share uptake.

Given hemochromatosis is a known risk with the iron-based phosphate binding, the sell-off had more to do with a “profit-from-the-news” event than the putative warning label.

Premium subscribers at were one-step ahead of Wall Street, having been told earlier: “Given uncertain commercialization prospects for the oral phosphate binder Zerenex in obtaining meaningful market share in the dialysis-treatment space due to managed care and competitive risks, investors might look to lock in existing gains with a suggested options hedge strategy – at least until a clearer picture emerges on the potential use of Zerenex in managing elevated serum phosphorus levels and iron deficiency anemia in non-dialysis dependent (NDD) CKD patients.”

Editor David J Phillips no longer holds a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy. 

Tuesday, August 19, 2014

PropThink Readers Profit from Amicus Therapeutics Buy Recommendation

Premium readers over at are already up 15% since our buy recommendation was issued on Amicus Therapeutics [FOLD] on August 7 at $4.00 per share.

Though the investment premise remains intact, investors might want to either lock in existing short-term gains, or buy September 5 calls as downside insurance against any near-term clinical surprises.
Investment Thesis
Albeit the investment thesis is focused on monotherapy use of its proprietary, pharmacological chaperone migalastat in Fabry Disease, there are value-creating assets that should become more visible in late 2015. In the treatment of Pompe disease (a glycogen storage disorder caused by deficiency of an enzyme called acid α-glucosidase), Amicus is looking to leverage its proprietary Chaperone-Advanced Replacement Therapy  platform to improve currently marketed ERTs through co-administration of a pharmacological chaperone prior to ERT infusion, and/ or to develop next-generation ERTs that consist of a proprietary lysosomal enzyme therapy co-formulated with a pharmacological chaperone.
Those investors looking for additional insight into our thoughts on FOLD and visible catalysts, such as Study 012, might consider opening their wallets and purchasing a subscription to

 Editor David J Phillips no longer holds a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy. 

Tuesday, August 12, 2014

OncoGeneX Checkup

Ignore OncoGeneX’s (OGXI-$3.15) reported second-quarter 2014 loss of 47 cents per share. Investors, instead, should focus on existing positives and visible price catalysts coming up in the next 12-months, including:

  • Management: "Based on our current expectations, we believe our capital resources will be sufficient to fund our currently planned operations into the third quarter of 2016. " Company recently completed a $22.4 million cash raise.
  • The SYNERGY Trial: Results of the SYNERGY trial will be presented at the European Society for Medical Oncology (ESMO) 2014 Congress (September) in Madrid.
  • Management addressed previous unknown  raised in a recent PropThink article on ENSPIRIT trial too: "We expect to evaluate both progression-free survival, or PFS (PFS rate at 14 weeks in 170 patients), and overall survival, or OS (OS at 100 events), during the first interim futility analysis"; The first of two analyses is expected to be conducted in 2014.
  • The Borealis-2 Trial (investigator-sponsored, randomized Phase 2 trial evaluating apatorsen in combination with docetaxel treatment compared to docetaxel treatment alone in patients with advanced or metastatic bladder cancer who have disease progression following first-line platinum-based chemotherapy) could be a big win, as more than 50% of patients with urothelial cancer of bladder discontinue FIRST-LINE treatment with cisplatin either due to non-responsiveness and/ or toxicity.


Editor David J Phillips holds a financial interest in OncoGenex common stock. The 10Q Detective has a Full Disclosure Policy.

Thursday, July 31, 2014

Making Sense on OncoGeneX

OncoGeneX’s (OGXI - $2.98) stock collapsed in the last four months, losing almost 80% in market value after reporting that its lead asset, the antisense drug custirsen, failed in a pivotal phase III prostate cancer trial. Sporting a market cap of just $60 million and holding more than enough days’ cash on-hand to fund visible clinical catalysts for a second drug that might prove useful in combination with chemotherapy, the company could make for an interesting turnaround play

OGXI is a speculative, micro-cap oncology company – now, essentially a call option with multiple shots on goal: $60 million in cash/equivalents provides a backdrop to a market capitalization of just $60 million; excluding dilutive effect of outstanding options and warrants, and OGXI trades essentially at cash.

Despite an off-putting non-response from management (detailed in article), risk-tolerant investors can own OGXI at its all-time lows ahead of incremental value-driving events. ESMO 2014 may provide hope for failed lead candidate, custirsen, as management should present further analysis from its phase 3 SYNERGY trial. A positive efficacy signal in a subset of patients may drive interest in OGXI once again, ahead of more catalysts into early 2015.

Wednesday, June 11, 2014

Conatus Pharma Breaks From Intercept's Shadow?

Bloggers and traders alike are scratching their heads, looking for answers as to why Conatus Pharmaceuticals (CNAT) is up more than 48% intraday today.

Short of any company comments and my own hubris, Premium subscribers over at profitably know why: "Intercept  Isn't the Only Way to Play Liver Disease."

To wit: [excerpt] "Similarly focused on liver disease treatments, Conatus Pharmaceuticals (CNAT) has risen and fallen on parallel developments for Intercept Pharmaceuticals’ (ICPT) obeticholic acid (OCA). A series of favorable emricasan updates in the next 12 months, including Phase 2 updates in patients with nonalcoholic fatty liver disease (NAFLD) or fibrotic nonalcoholic steatohepatitis (NASH), could allow investors to better assess the commercial opportunities of CNAT’s lead compound – and finally free the stock price from Intercept’s shadow."

Editor David J Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy. 

Friday, May 02, 2014

Agios Impresses With Leukemia Drug: Investor Beware

Agios Pharmaceuticals (AGIO-$43.35) moved substantially higher in price last month after reporting Phase 1 data demonstrating that its cancer metabolism drug AG-221, a first-in-class inhibitor of IDH2 mutations, generated promising clinical activity, including complete remissions in several patients whose blood cancers harbored the IDH2 mutation. 

What has investors excited is that in addition to being well-tolerated, a “substantial” reduction of plasma 2-HG was achieved: Called an “oncometabolite” for its role in cancer metabolism, the metabolite 2-hydroxygluturate (2HG) is a by-product of the mutated IDH2 gene. The initial findings support the theory that inhibiting mutant forms of IDH2 suppresses the growth of these 2HG-producing tumor cells.

Investors are making the quantum leap – based on a small safety/dosing trial – that curtailing 2-HG supply will normalize gene expression….

Editor David J Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy. 

Biotech Losers of 2013: AVEO, INFI, AFFY, and ARIA -- Where Are They Now?

Despite having lost more than 90% of its market valuation in the last 21 months, AVEO Pharma’s (AVEO-$1.24)  management believes it can drive shareholder creation by moving forward with other early-stage, oncology assets, principally by securing partnerships after providing proof-of-concept data. Though attractive commercial opportunities do exist, even in highly competitive markets, for targeted cancer therapies, we question this management team’s ability to develop their putative “first-in-class clinical assets” following the debacle of the VEGF receptor tyrosine kinase inhibitor, tivozanib, last year.

Evolving Phase 1 data presented at the American Society of Hematology (ASH) meeting held in early December 2013 showed not only that earlier safety concerns on Infinity’s (INFI-$9.20) lead asset, IPI-145, had been overblown, but that the drug demonstrated impressive efficacy in patients with either relapsed or refractory CLL (81% of patients had been treated with three or more systemic therapies), too.

Unfortunately, we’ll never know the “would of, could of” commercial potential for Affymax’s (AFFY-$0.70) anemia treatment, brand-name Omontys. After posting sales of $34.6 million for the nine-month period ending December 31, 2012 – slightly below analyst forecasts, suggestive of the contract-grip Amgen held on dialysis centers – Omontys was recalled on February 23, 2013 following reports of severe hypersensitivity reactions including anaphylaxis in 0.2% (or about 50 patients) — including fatal reactions in 0.02% of the 25,000 patients –within 30 minutes of their first IV dose in the post-marketing phase. Almost a third of the reported cases required prompt medical intervention and in some cases hospitalization, according to an FDA safety alert issued at the time.

Though the stock price of Ariad Pharma (ARIA-$7.25) has rallied off its 52-week low of $2.15 per share (October 31, 2013) to the $7.00 level, it still trades significantly below its 52-week high of $23.00 (September 11, 2013). The relief rally has stalled for two reasons:
  • Iclusig competes in an already crowded market for just two rare cancers. According to National Cancer Institute statistics, approximately 5,200 new cases of CML and 1,800 new cases of Ph+ ALL are diagnosed each year in the United States; and,
  • Ariad has discontinued – after consulting with the FDA – its EPIC trial, which had been designed to investigate the use of Iclusig in the front-line settings.

Editor David J Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy. 

Thursday, April 03, 2014

AbbVie's IV Lifeline Still Humira

Investors in AbbVie (ABBV-$53.34) are reminded each quarterly earnings release of the continued importance of Humira to the drug maker's success.  AbbVie's flagship drug, an efficacious treatment for autoimmune diseases like rheumatoid arthritis (RA) and psoriasis, contributed $10.7 billion in global sales (56% of worldwide revenue of $18.8 billion) last year.

AbbVie is spending more than 16% of annual sales to develop a pipeline -- including promising treatments for multiple sclerosis, blood cancers, and hepatitis-C (across all patient types) - with the goal of reducing its dependence on Humira. Fortunately, the Chicago-based drug manufacturer has a stable of off-patent, legacy drugs that contribute sizable sales to this R&D effort.

Editor David J Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy.