Replies to post #122104 on Biotech Values

[DewDiligence]

I would add ACHN to my short list of HCV candidates.

I would not short it, but at the current valuation it’s clearly less appealing than it was when I recommended it to biomaven one month ago (#msg-63571059). In general, I refrain from shorting small-cap names that are not outright scams (and the outright scams are usually impossible to borrow for shorting).

[mcbio]

ACHN is having a stellar day (up 20%) to $7.22 as I write this message.

Perhaps it's on the pricing of the offering, I'm not sure. Surprised to see the stock up so much on no concrete news. (They did announce after close that they have begun dosing in the 3-month portion of the Phase 2 trial of 1625.)

In turn I would add ACHN to my short list of HCV candidates.

Short list of HCV candidates to invest in? ; )

Even if 2684 achieves pangenotypic status it will need to be paired outside its class to boost the drugs resistance profile IMO. BMY pairing their P.I. with their NS5A already ended in failure. Will ACHN meet the same fate?

Yes, of course, 2684 (and 1625) will in all likelihood need to be paired with an HCV nuke or a drug from another HCV class, but so what? Why can't that be done? Sure, it would be better if they had the entire combo in-house (they have the PI+NS5A combo in-house but, like you say, what we saw before from BMY probably means this won't be enough) but that's not to say that they can't hook up with a partner down the road.

The P.I. class therefore may only find limited usage in nulls and non-responders on top of nukes and the chance of a safety issue, even at this MC, is more risk than I would be willing to bear.

Way too early to say it will be limited usage for the PI class. And it's not like ACHN has a $5B market cap so they don't need to have a bonafide blockbuster on their hands in 1625, 2684, and/or 2928 for the stock to do well longer-term. And do you really want to talk chance of safety issue and risk to market cap in light of the VRUS valuation? lol ACHN is a VRUS nuke safety (or other) issue away from really being in great shape but I don't think they necessarily need that to do well.

How will investors in ACHN react to expensive phase 2 testing in combination with PEG/Riba when the end of their usage is in sight?

And the end of their usage could be in sight with a combo of 1625 or 2684 plus a nuke or an HCV drug from another class. All told, though I give credit where credit is due and ackowledge that VRUS is in great shape, I'm not sold that the VRUS nukes will be the end-all-be-all of next gen HCV therapy. It's a big enough market that there will likely be room for multiple drugs from multiple classes.

[mcbio]

ACHN - initiates Phase 2 1625 12-week dosing

http://finance.yahoo.com/news/Achillion-Initiates-12Week-pz-3563379023.html?x=0&.v=1

Achillion Initiates 12-Week Dosing in Phase 2 Trial of ACH-1625 for the Treatment of Chronic Hepatitis C

NEW HAVEN, Conn., June 22, 2011 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN - News) today announced that the Company has initiated patient dosing in segment 2 of its Phase 2 clinical trial of ACH-1625 for the treatment of hepatitis C virus (HCV) for genotype 1 treatment naive HCV-infected patients. ACH-1625, discovered and advanced by Achillion, is a potent small molecule inhibitor of HCV protease, an enzyme necessary for viral replication.

The clinical trial has advanced into the second segment of a Phase 2a, randomized, double-blind trial evaluating the safety, tolerability and antiviral activity of oral ACH-1625 in combination with standard of care (SOC) consisting of pegylated interferon alfa-2a and ribavirin. Patients will be randomized to receive once daily doses of 200 mg, 400 mg or 800 mg of ACH-1625 in combination with SOC for 12 weeks of dosing. Patients will continue to receive an additional 12 weeks of pegylated interferon alfa-2a and ribavirin and eligible to discontinue treatment at week 24 if they achieve extended rapid virologic response (eRVR) at week 12. Patients who do not achieve an eRVR will continue to receive SOC until week 48.

The trial will take place in the United States and Europe and is designed to enroll approximately 60 HCV-infected patients. The 12-week complete early virologic response (cEVR) trial results are anticipated to be announced in the fourth quarter of 2011.

"Initiating the second segment of this Phase 2 clinical trial allows us to build upon the robust RVR results we observed with ACH-1625, and to further augment the safety and efficacy database by taking the opportunity to study multiple doses of ACH-1625," commented Elizabeth A. Olek, D.O., Vice President and Chief Medical Officer of Achillion. "We expect that the results will provide important insight to benchmark the activity of our once-daily protease inhibitor and we look forward to reporting cEVR results by the end of this year."

"This next study segment with ACH-1625 is yet another important milestone achieved for this potentially best-in-class protease inhibitor and for Achillion's broader HCV pipeline," said Michael D. Kishbauch, President and Chief Executive Officer of Achillion. "It should be recalled that, very recently, we announced the start of Phase 1 on Achillion's high-potency, pan-genotypic protease inhibitor, ACH-2684, and with the upcoming start of Phase 1 on our first NS5A inhibitor, ACH-2928, Achillion remains poised to deliver on a number of clinical milestones over the next few quarters that we believe will significantly enhance our overall position within the important and promising HCV market."

About ACH-1625

ACH-1625 is a HCV protease inhibitor designed and synthesized based on crystal structures of enzyme/inhibitor complex. ACH-1625 is an open chain, non-covalent, reversible inhibitor of NS3 protease. In preclinical studies, ACH-1625 demonstrated high potency, unique pharmacokinetic properties and an excellent safety profile at high drug exposures. ACH-1625 has rapid and extensive partitioning to the liver, as well as high liver/plasma ratios. ACH-1625 has shown low single-digit nanomolar potency that is specific to HCV. It is equipotent against HCV genotypes 1a and 1b at IC50 of approximately 1nM.

In the first segment of a Phase 2a clinical study, HCV-infected patients receiving doses of 200 mg, 400 mg, or 800 mg of ACH-1625 in combination with SOC achieved a rapid viral response of 75 -- 81% compared to an RVR of 20% for patients receiving SOC only. ACH-1625 was well tolerated at all doses with no serious adverse events reported and adverse events which were reported as mild to moderate and transient.

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 and The American Association of Liver Disease estimates that up to 80% of individuals become chronically infected following exposure to the virus. If left untreated, chronic hepatitis can lead to permanent liver damage, which 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 hepatitis C and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.