and sofosbuvir was safe and well-tolerated with 100 percent SVR4 for
treatment-naïve genotype 1 HCV (“Proxy Study”)
NEW HAVEN, Conn., Dec. 22, 2014 (GLOBE NEWSWIRE) — Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced positive interim results from two studies supporting a
short duration, potentially best-in-disease regimen of its proprietary
NS5A and nucleotide inhibitors, ACH-3102 and ACH-3422.
“We believe that achievement of 100 percent SVR4 in six weeks in the
ACH-3102 proxy study, combined with the high potency and safety
demonstrated by ACH-3422, highlights the ability of our exceptional,
fully owned portfolio to excel in the HCV market,” commented Milind
Deshpande, Ph.D., President and Chief Executive Officer of Achillion.
“We look forward to initiating in 2015 short duration, pan-genotypic
Phase 2 therapeutic trials to evaluate the doublet of ACH-3102 and
ACH-3422, with the ultimate goal of improving patient care and access to
Phase 2 “Proxy Study” evaluating six-week
treatment consisting of ACH-3102, second-generation NS5A inhibitor, and
sofosbuvir for treatment-naïve genotype 1 HCV
Achillion today announced 100 percent SVR4 results from the ongoing
six-week trial. This study is an interferon-free, ribavirin-free, Phase 2
open-label, randomized study to evaluate the efficacy, safety, and
tolerability of six weeks of 50 mg of ACH-3102 and 400 mg of sofosbuvir,
a marketed nucleotide polymerase inhibitor, once daily, in
treatment-naïve genotype 1 HCV-infected patients. The primary objective
of the study is determination of sustained viral response 12 weeks
(SVR12) after completion of therapy.
Eighteen patients were enrolled, including twelve active and six
observational patients. Mean baseline HCV RNA viral load was 10 million
(7 log10) IU/ml, range 2 million (6.23 log10) – 97 million (7.99 log10) IU/ml, including seven patients with baseline HCV RNA viral load exceeding 6 million (6.78 log10) IU/ml. Of the 12 active patients enrolled, ten patients were genotype 1a and two were genotype 1b.
Four weeks after the completion of therapy, 100 percent (n=12/12)
achieved SVR4, independent of baseline viral load, gender, and IL28B
status. No post-treatment viral relapse has been observed to date. SVR12
results will be reported during the first half of 2015. The combination
of ACH-3102 and sofosbuvir was well-tolerated with no serious adverse
events, no discontinuations due to adverse events, and no clinically
significant laboratory or ECG abnormalities.
Dr. Deshpande further commented, “The ACH-3102 Phase 2 results continue
to support the best-in-class profile of our second-generation NS5A
inhibitor. Despite the presence of high baseline viral loads in
patients, including one patient with nearly 8 log10 HCV RNA
at baseline, ACH-3102 in combination with a nucleotide demonstrated
rapid suppression of viral replication. We believe the ability to
achieve 100 percent SVR4 after only six weeks of therapy highlights the
role ACH-3102 could play in unleashing the full potential of a NS5A-nuc
ACH-3422: Phase 1 study in healthy subjects and proof-of-concept in treatment-naïve genotype 1 HCV-infected patients
Achillion also announced today interim study results demonstrating that
ACH-3422 achieved proof-of-concept in a Phase 1 trial for patients with
treatment-naïve genotype 1 HCV. In the 700 mg dose group, mean maximal
reduction in HCV viral RNA load of 4.8 log10 IU/ml was
observed within 14 days with 3 out of 6 patients achieving undetectable
HCV RNA ( < 10 IU/mL, “target not detected”). The pharmacodynamic
characteristics of ACH-3422 provided sustained antiviral activity
resulting in an additional 1.4 log10 reduction in HCV RNA between day 7 and day 14 of dosing.
“The safety profile, potent antiviral activity, and high barrier to
resistance observed with ACH-3422 in this Phase 1 trial exhibit the
important characteristics a nucleotide inhibitor provides in HCV
treatment regimens,” commented Dr. David Apelian, Executive Vice
President of Clinical Development and Chief Medical Officer at
Achillion. “The data, combined with the Phase 2 proxy study results,
lead us to believe that the doublet regimen of ACH-3102 and ACH-3422 can
be a highly competitive, regimen to cure HCV. Furthermore, the ability
to explore a triplet regimen with sovaprevir, our protease inhibitor,
may allow for shorter treatment durations especially in harder-to-treat
This adaptive design Phase 1 trial is a randomized, double-blind,
placebo-controlled trial investigating the safety, tolerability,
pharmacokinetics and antiviral activity of ACH-3422. The trial has
evaluated escalating doses ranging from 50 mg to 700 mg of ACH-3422 in
healthy volunteers in single ascending dose cohorts followed by 14-day
multiple ascending dose cohorts. All doses were well-tolerated with no
significant adverse events, ECG or laboratory abnormalities noted.
Cohorts of treatment-naïve genotype 1 HCV-infected patients were
enrolled and received once daily treatment with ACH-3422. Patients in
the 50 mg, 150 mg and 300 mg cohorts received seven days of treatment;
patients in the 500 mg and 700 mg cohorts were treated for 14 days. All
doses of ACH-3422 were well-tolerated with no treatment-related SAEs, no
discontinuations due to adverse events, and no clinically
significant laboratory or ECG abnormalities.
The Company will host a conference call and simultaneous webcast on
Monday, December 22, 2014 at 8:30 a.m. Eastern time. To participate in
the conference call, please dial (866) 205-4820 in the U.S. or (419)
386-0004 for international callers. A live audio webcast of the call
will be accessible at http://www.achillion.com or http://ir.achillion.com.
Please connect to Achillion’s website several minutes prior to the
start of the broadcast to ensure adequate time for any software download
that may be necessary.
A replay of the webcast will be available for 30 days on www.achillion.com.
Alternatively, a replay of the conference call will be available
starting at 12:00 p.m. Eastern time on December 22, 2014, through 11:59
p.m. Eastern time on December 28, 2014 by dialing (855) 859-2056 or
(404) 537-3406. The replay passcode is 57460707.