
I cant wait for what next week brings.
UPDATE 1- Achillion's hepatitis C drug gets FDA fast track
May 15 (Reuters) - The U.S. Food and Drug Administration granted a fast track designation to Achillion Pharmaceuticals Inc's experimental hepatitis C drug ACH-3102, currently in an early-stage trial.
The fast-track status is designed to expedite the regulatory review of drugs that aim to treat serious diseases and fill unmet medical needs.
The New Haven, Connecticut-based company got a fast track designation in January for another of its experimental hepatitis C (HCV) drug ACH-1625, which was in a mid-stage trial.
The hepatitis C virus infects the liver and is the most common cause of viral hepatitis, an inflammation of the liver.
The market for hepatitis C treatments is expected to be worth around $15 billion by 2019, garnering significant investor interest, and most firms trying to develop a drug for the infection have seen stock prices skyrocketing over the past one year.
Shares of Achillion closed at $6.89 on Monday on the Nasdaq.
The recent correction in the stock market has created new buying opportunities for investors. With all the concerns about the global economy, it makes sense to consider investing in companies that are not deeply impacted by recessions. Biotech and healthcare companies are generally insulated from the economy much better than most. The lower stock prices brought about by the correction and the recession-resistant nature of these companies, could allow biotech and healthcare investments to outperform. To be sure, investing in this sector has major risks, but the rewards can be equally large. The names below might be particularly compelling because all are pursuing treatments for hepatitis, which is has blockbuster potential. Some investors might remember that earlier this year, Bristol-Myers (BMY) acquired Inhibitex, Inc. (INHX), a biotech focused on hepatitis treatments, for $26 per share. Many healthcare and major biotech companies are actively pursuing merger and acquisition deals, and more buyouts could occur in this sector. Some corporate balance sheets are loaded with cash, financing costs are low and major pharmaceutical companies need to find growth through acquisitions. Here are some stocks to consider now:
Achillion Pharmaceuticals, Inc. (ACHN) is a biopharmaceutical company focusing on developing treatments for hepatitis C infections. It also has antiviral and antibacterial candidates in the pipeline. ACH-1625 is the most advanced candidate, and it is currently in phase 2 clinical trials. It is a promising hepatitis C inhibitor with the potential for once-daily dosing. Investor interest in hepatitis is strong because it has block-buster market potential and companies (like Inhibitex) pursuing this segment have been the subject of takeovers. This company could be particularly attractive to a suitor because it is so highly focused on hepatitis. Achillion has a strong balance sheet with about $65 million in cash and less than $1 million in debt. At least one insider is taking advantage of the currently low stock price: On June 11, 2012, Dennis Liotta, a director, bought 10,000 shares in a transaction valued at about $61,600. On June 10, he also bought 10,000 shares for a two day total of 20,000 shares.
Here are some key points for ACHN:
Opko Health Inc., (OPK) shares have been in a trading range of around $4.50 to $5 for the past couple of months. With the shares now at the low end of that range, it could be an ideal time to buy. This company is developing specialized drugs and vaccines. It recently announced acquiring a 45% stake in a private Israeli company that is developing a hepatitis B vaccine which has shown promise in clinical studies. The CEO of Opko, Phillip Frost, is a billionaire who has been very successful in past biotech and healthcare-related endeavors. He has also been involved in merger and acquisition deals and he serves as chairman of the board at Teva Pharmaceutical Industries (TEVA). That is another reason why it is particularly interesting to follow and perhaps invest in Opko. On June 14, 2012, Phillip Frost, bought 200,000 shares in a transaction valued at about $922,000. On June 13, he also bought 85,000 shares for a two day total of 285,000 shares. The CEO has been steadily buying over the past few months.
Here are some key points for OPK:
Bristol-Myers Squibb shares have been a great investment for the past several months. Thanks to a stable revenue source and a generous dividend yielding about 4%, this company makes sense for income investors. This company acquired Inhibitex for about $2.5 billion or $26 per share earlier this year. Inhibitex was acquired due to a candidate it developed to treat hepatitis, and the stock went from about $3, to $26 in just months. Bristol-Myers also owns blockbuster drugs such as Plavix and Abilify. The deal for Inhibitex could add growth potential in the future, and more deals are possible thanks to the strong balance sheet.
Here are some key points for BMY:
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Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

Disclosure: I am long ACHN. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article. (More...)
Achillion Pharmaceuticals' (ACHN) share price has been driven down recently from when I first suggested considering this stock to buy. I even doubled down on the initial purchase in The Young And Restless Retirement Portfolio.
I followed up with this report which detailed some of the successful results in phase II trials, as noted by Milind Deshpande, Chief Scientific Officer and President of Research and Development:
"We are very pleased to see that the profile of ACH-3102 continues to exceed our expectations for providing a truly improved barrier to resistance. As the first-ever clinical trial to evaluate a NS5A inhibitor as a single direct-acting antiviral in combination with ribavirin, we are extremely encouraged by these initial results that demonstrate rapid suppression of the HCV GT1b virus and a well-tolerated safety profile through 12 weeks of therapy," commented Milind Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer of Achillion. "As this data set continues to mature, we look forward to reporting initial SVR results at a medical meeting in the second quarter, and are planning to expand enrollment in the study to include non-CC GT 1b treatment-naïve patients later this quarter pending regulatory discussions."
At the time of that report, it was also stated that the company will report further clinical results more frequently:
ACHN has 3 drugs in its pipeline at various stages of clinical trials that could be considered potential hepatitis C oral, once per day, treatments.
Sovaprevir (ACH-1625) and ACH-3102 are nearing phase III trials, which will be the most significant, and ACH-2684 is nearing phase II trials. As of today, the company has stated that throughout the balance of 2013, more updates on success or the lack thereof will be given on each.
Achillion Has Made Progress And More Announcements Are Soon To Come
A few weeks ago the company released this report which detailed the positive progress on the efficacy and safety of ACH-3102, as well as the differentiation of the drug from other drugs being tested:
Dr. Andrew Muir, Principal Investigator and Assistant Professor of Medicine and Director of Gastroenterology/Hepatology Research at Duke Clinical Research Institute commented, "The preliminary results from this novel study of a single DAA, an NS5A inhibitor, plus ribavirin demonstrates the safety, high barrier to resistance, and preliminary efficacy of ACH-3102. The profound activity of ACH-3102 as a single DAA, along with its safety profile and lack of virologic breakthrough to date makes this a very promising compound to study further in combination with other oral agents, including sovaprevir, for the treatment of HCV."
Having the progress measured by Duke Clinical Research, and have the principal investigator impressed, is very encouraging to say the least.
In addition to that report, ACHN presented at the ILC conference with an even more focused progress report, aimed directly at the promising results of both ACH-3102 and sovaprevir in treating hepatitis types 1a and 1b:
"The high barrier to resistance observed with ACH-3102 to date continues to confirm our view that ACH-3102 is unique from other NS5As and has the promise to be a cornerstone therapy in the treatment of Hepatitis C," said Milind Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer of Achillion. "The unique characteristics of ACH-3102, combined with the unique profile of our 2nd generation PI, sovaprevir, which also has a higher barrier to resistance than typical PIs, make for what we believe is a high potential combo, competitive against both current and emerging combinations."
Supportive of these findings, other sources have honed in on the potential of ACHN in this huge market. As reported in this article, the effectiveness of the drug regimen is duly noted:
Achillion Pharmaceuticals Inc. said an experimental drug against hepatitis C suppressed the virus in 63 percent patients in a study. Four weeks after completing a 12-week course of treatment with the medicine, ACH-3102, the liver-damaging virus, was undetectable in the blood of five out of eight patients, New Haven, Connecticut-based Achillion said in a statement today. Two didn't respond to the drug while on treatment, and in another the virus returned three weeks after therapy ended.
Achillion said last week its started a trial of ACH-3102 in combination with its lead drug, sovaprevir, in as many as 50 patients, and expects to report interim results in the third quarter. Other drugmakers, including Bristol-Myers Squibb Co. (BMY), are also trying to develop treatments that are faster and safer than the current standard, and which don't involve injections. Achillion "will continue to explore and execute opportunities to combine our agents with other compounds that could further shorten the treatment duration or provide additional flexibility for treatment regimens to broadly cure HCV," Chief Executive Officer Michael Kishbauch said in the statement.
There are also indications that the investment community is beginning to see the potential for the stock as well as the drugs. This article offers some insight from a key analyst in this sector, Brian Skorney, Senior Analyst at Robert W. Baird & Co.:
Achillion Pharmaceuticals remains undervalued as investors continue focusing almost exclusively on Gilead Sciences (GILD) for hepatitis C treatment, a market so large that ACHN has the opportunity to find and penetrate segments Big Pharma is not expected to reach for years......."I think Gilead is a great powerhouse on hepatitis C, but the market opportunity is so large from a volume perspective that I just can't see one company really dominating the market; to that extent, there will be pockets where pricing is going to have an opportunity to create a market where Gilead may not even want the price to penetrate for a number of years," Skorney said........"I think Achillion has a great opportunity with a regimen for hepatitis C that I think will come very close to matching Gilead's profile."
As far as I am concerned, this is a wonderful endorsement of the recent progress that Achillion has delivered.
Further progress reports will be forthcoming this week, as the company will be a key presenter at three upcoming conferences:
Investors will surely be looking for more positive news at these events.
Other Reports On The Advancements In Hepatitis C Drug Treatments
There have been updates on a variety of progress reports covered by Medpage Today in various articles, and you can visit that link for all of the latest advancements in the treatment of hepatitis C, and see that some drugs are progressing, and some have not lived up to expectations.
In this particular report, it was duly noted that an all oral, triple combination of drugs appeared to be effective in the most difficult form of hep C to treat; viral genotype 1a, which is the primary focus of the ACHN drug regimen:
Hitting hepatitis C virus (HCV) from three directions was a winner in a phase II trial involving patients with the particularly hard-to-treat viral genotype 1a, researchers said here.
Excellent sustained 12-week viral response were seen in a phase II study of patients infected with HCV genotype-1 receiving various regimens that were interferon free and combined the investigational agents daclatasvir, asunaprevir, and BMS-791325.
The study was done by Bristol-Myers (BMY), and now that ACHN has released company earnings the other day, investors have been further emboldened by Achillions' progress, with this statement by Michael Kishbauch, President and CEO:
"Our goal to advance our potentially best-in-class HCV compounds continued to pick up significant momentum during 2013. We believe that the 12-week safety and efficacy results recently announced on ACH-3102, our second-generation pan-genotypic NS5A inhibitor, continue to support its differentiated profile with a high barrier to resistance and no viral breakthrough," commented Michael D. Kishbauch, President and Chief Executive Officer of Achillion. "We recently initiated an all-oral Phase 2 program evaluating sovaprevir and ACH-3102 for the treatment of genotype 1 HCV, and we expect to achieve a number of significant catalysts over the next few months including interim RVR results from the -007 trial in the third quarter, followed by SVR results during the fourth quarter. Furthermore, we remain focused on evaluating additional assets with complementary mechanisms of action that could potentially expand our treatment regimens to treat subsets of patient populations not adequately addressed by current regimens."
ACHN is focused on developing a strong pipeline of drugs, and while I believe ACHN could still be a takeover target, the company does have over $200 million in cash to see these trials right through phase III, on its own. By going after the type 1 viral strain, ACHN seems to be making a clear statement that their drugs work better than the myriad of competitors.
The risks of course, is the failure of any of the drugs during clinical trial phases, and the risk of losing an entire investment. That being said, I believe at the current price, the shares offer a risk/reward in this market that cannot be overlooked.
My Opinion
I remain bullish on the potential of ACHN and believe that the company will be a vital part of a $20-$30 billion market. Whether on its own, or gobbled up by a larger company, investors could profit from the success of ACHN.
The recent pullback in price makes for yet another compelling entry point.
Disclaimer: The opinions of the author is not a recommendation to either buy or sell any security and is strictly his opinions. Please do your own research prior to making any investment decisions.
NEW HAVEN, Conn., May 15, 2013 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (ACHN) today announced the appointment of David Apelian, M.D., Ph.D., MBA, to the position of Executive Vice President and Chief Medical Officer, effective May 28, 2013. Dr. Apelian wll assume responsibility for the clinical development of Achillion's portfolio of compounds to treat HCV.
In addition, Achillion also announced today the expansion of its clinical operations group with the recent addition of Kevin Kucharski, formerly head of clinical operations at Pharmasset, who will serve as Senior Vice President of Clinical Operations.
"We are delighted that David is bringing his wealth of hepatitis and drug development expertise to Achillion. His operational and regulatory experience, highlighted by the regulatory approval of antivirals such as interferon alpha-2b/ribavirin and entecavir, will greatly benefit Achillion as we accelerate our development plans and seek to initiate registrational studies with sovaprevir and ACH-3102 during the latter part of next year," commented Michael D. Kishbauch, President and Chief Executive Officer of Achillion.
Dr. Apelian commented, "It is a very exciting time for me to be joining Achillion as Chief Medical Officer, and I look forward to further accelerating the development of the all-oral, interferon-free regimens containing sovaprevir and ACH-3102 for the treatment of chronic HCV."
Mr. Kishbauch also stated, "As Achillion continues its maturation into a late-stage biopharmaceutical company, the addition of both Kevin and David will expand our bandwidth, providing critical expertise and the support necessary for our HCV clinical development programs and our planned Phase 3 development."
David Apelian, M.D., Ph.D., MBA
Dr. Apelian brings to Achillion more than 13 years of industry experience from Bristol-Myers Squibb, Schering Plough and GlobeImmune where he focused on hepatology and infectious disease clinical development. Dr. Apelian was Clinical Director in the Infectious Diseases Group at Bristol-Myers Squibb (BMS), serving as medical co-lead for the clinical development and NDA submission of entecavir for chronic hepatitis B viral infection (HBV). Prior to BMS, Dr. Apelian served as Clinical Director in the Department of Hepatology/Gastroenterology at Schering Plough, coordinating a supplemental NDA filing for interferon alpha-2b and ribavirin for the treatment of pediatric patients with chronic hepatitis C viral infection (HCV). Most recently, Dr. Apelian served as Senior Vice President of Research and Development and Chief Medical Officer at GlobeImmune, where he was responsible for clinical development, regulatory affairs, clinical immunology, development of companion diagnostics, as well as target discovery and preclinical research.
Dr. Apelian completed his residency training in Pediatrics at New York Hospital, Cornell Medical Center. He received his M.D. from the University of Medicine and Dentistry of New Jersey, and his Ph.D. in Biochemistry and B.A. from Rutgers University. He also holds an M.B.A. from Quinnipiac University
Kevin Kucharski
Mr. Kucharski comes to Achillion with more than two decades of clinical operations expertise, with a focus on conducting U.S. and global clinical trials, ranging from Phase 1 through Phase 4. Prior to joining Achillion, Mr. Kucharski was Vice President of Clinical Operations at Pharmasset since 2007, and then at Gilead Sciences following their acquisition of Pharmasset. While at Pharmasset, Mr. Kucharski provided effective oversight and execution of various global clinical development projects including PSI-7977 and PSI-938. Prior to Pharmasset, Mr. Kucharski was Senior Director of Clinical Operations at Altana Pharma US, a Nycomed Company, where he established the Clinical Operations group. Prior to that, Mr. Kucharski held positions of increasing responsibility in Clinical Operations at Schering-Plough Research Institute. Mr. Kucharski was awarded his B.S. in Biology Research from the University of Scranton.
About HCV
The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.
About Achillion Pharmaceuticals
Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease, including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.
Forward-Looking Statements
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to: the potency, safety, tolerability, effectiveness and other characteristics of sovaprevir and ACH-3102; Achillion's expectations regarding timing for the commencement, completion and reporting of results of its -007 clinical trial of sovaprevir and ACH-3102 in combination with ribavirin, as well as with respect to the advancement into later stages of its clinical studies; and the expected contributions of Dr. Apelian and Mr. Kucharski and the planned expansion of the clinical operations group. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage clinical trials of sovaprevir, ACH-3102 and its other product candidates; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; successfully execute on its business plans and strategies; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2012 and its subsequent SEC filings.
In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.


