le marché apprécie les chiffres mais quelques déconvenues ou retards au niveau du pipeline...
Cubist Pharmaceuticals, Inc. (CBST) today announced unaudited fourth quarter and full-year 2013 revenues and a pipeline update as the Company positions itself to achieve its Building Blocks of Growth long-range goals. Cubist delivered 22% total net revenue growth in the fourth quarter compared to 2012, and CUBICIN® (daptomycin for injection) achieved blockbuster status with over $1 billion in worldwide sales for the full year.
Total net revenues for Q4 2013 of $299.7 million, which include DIFICID® (fidaxomicin) revenues since the completion of Cubists acquisition of Optimer Pharmaceuticals, Inc. on October 24, 2013, were up 22% compared to $245.9 million in Q4 2012. Full-year 2013 total net revenues were $1.1 billion, up 14% compared to $926.4 million in 2012. U.S. CUBICIN net product revenues in Q4 2013 increased 15% to $248.9 million from $216.0 million in Q4 2012. Full-year 2013 U.S. CUBICIN net product revenues were $908.0 million, up 12% compared to $809.2 million in 2012.
With strong revenue growth, two strategic acquisitions, and excellent progress on our pipeline, 2013 was a remarkable year for Cubist, said Michael Bonney, Chief Executive Officer of Cubist. CUBICINs achievement of blockbuster status is a reminder of the critical need for safe and effective antibiotics around the world and underscores the potential of our pipeline advancing toward commercialization. As we begin 2014, we are more excited than ever by our future growth prospects, and we expect to continue to drive our Building Blocks of Growth through internal discovery and development efforts, as well as through highly focused business development activities.
PIPELINE UPDATE
Prioritizing Investments
Entering 2014, Cubist is prioritizing its portfolio investments in a disciplined manner to optimize those assets with the highest near-term potential. The prioritization and distribution of investments in 2014 will allow the Company to maximize the value of its lead candidates in alignment with Cubists Building Blocks of Growth goals, while continuing to grow its leadership in antibiotics and acute care. The Company hopes to deliver at least four new antibiotics in support of the Infectious Diseases Society of America (IDSA) goal of 10 new antibiotics by 2020.
In an industry challenged with a lack of productivity in R&D, Cubist is entering 2014 with a rich pipeline of innovative and promising drug candidates, said Mr. Bonney. As we work to bring our promising ceftolozane/tazobactam and tedizolid phosphate antibiotics programs to commercialization on a global basis and with other new development programs on the horizon it is critical that we prioritize our portfolio investments on programs that offer the highest near-term potential impact for patients and maximize the benefit to our shareholders.
Continuing Momentum of Ceftolozane/tazobactam
Ceftolozane/tazobactam is being developed to treat certain Gram-negative infections, including those caused by Pseudomonas aeruginosa, as well as extended-spectrum beta-lactamase (ESBL)-producing Escherichia coli (E. coli), Klebsiella pneumoniae, and other Enterobacteriaceae bacteria classified by the U.S. Centers for Disease Control and Prevention (CDC) as serious threats. According to the CDC, an estimated 51,000 healthcare-associated Pseudomonas aeruginosa infections occur in the U.S. each year, and nearly 26,000 healthcare-associated Enterobacteriaceae infections are caused by ESBL-producing Enterobacteriaceae.
The Company recently announced that it met the primary endpoints for its pivotal Phase 3 clinical trials of ceftolozane/tazobactam in complicated urinary tract infections (cUTI) and complicated intra-abdominal infections (cIAI). The data from these trials are being submitted for presentation at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) to be held in May 2014.
Cubist plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval in the cUTI and cIAI indications in the first half of 2014. In the second half of 2014, the Company plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in these indications.
Additionally, in the first half of 2014, the Company expects to initiate a pivotal Phase 3 trial to assess the safety and efficacy of ceftolozane/tazobactam in Hospital-Acquired Bacterial Pneumonia (HABP)/Ventilator-Associated Bacterial Pneumonia (VABP). With recent FDA guidance creating greater clarity on an expedient route to registration in the indication and with opportunities for synergies with the planned Phase 3 trial of tedizolid in HABP/VABP the Company is ending its open label trial of ceftolozane/tazobactam in HABP/VABP and focusing its resources on the registrational study.
Advancing Tedizolid Phosphate
Tedizolid phosphate is being developed for the treatment of certain serious Gram-positive infections, including those caused by methicillin-resistant Staphylococcus aureus (MRSA). The CDC categorized MRSA as a serious public health threat, estimating that there are more than 80,000 invasive MRSA infections and more than 11,000 related deaths per year in the U.S.
In December 2013 the FDA accepted the Companys NDA for tedizolid phosphate with Priority Review. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of June 20, 2014. In the first half of 2014, the Company plans to submit a MAA to the EMA.
Tedizolid is also being developed for potential use in HABP/VABP, with Phase 3 pivotal studies expected to begin in the first quarter of 2014.
Investments in CDAD Programs DIFICID and Surotomycin
Clostridium difficile-associated diarrhea (CDAD) represents a large, growing market opportunity. Linked to more than 14,000 deaths in the U.S. per year and with deaths related to C. difficile increasing 400% from 2000 to 2007, the CDC lists C. difficile as one of three pathogens that represents an urgent threat.
Cubists commitment to combating CDAD currently includes the re-launch of DIFICID® (fidaxomicin) in 2014. The Company expects to target its efforts on positioning the product for appropriate patients while communicating the significant value of DIFICID. DIFICID is the first therapy approved for CDAD in more than 25 years and has demonstrated superiority on sustained clinical response compared to vancomycin. In August 2013, DIFICID received a one-year extension of the U.S. Centers for Medicare and Medicaid Services (CMS) new technology add-on payment (NTAP). The NTAP program, available to new technologies demonstrating a substantial clinical improvement and meeting specific cost thresholds, is designed to support timely access of innovative therapies used to treat Medicare beneficiaries in the inpatient setting.
The Companys ongoing Phase 3b clinical trial of DIFICID for the prevention of CDAD in patients undergoing hematopoietic stem cell transplant, often referred to as bone marrow transplantation, is progressing as scheduled and Cubist expects to review interim data in the first quarter of 2014.
CDAD is a relapsing condition in which patients and their physicians often require multiple therapeutic options. Accordingly, while the Company invests in DIFICID, it will also continue disciplined investments in its Phase 3 surotomycin CDAD program. The Company continues to expect to announce top line data from the surotomycin Phase 3 studies in 2015. The timeline for regulatory submissions will be determined following a full review of the Phase 3 data, but the Company expects the NDA submission for surotomycin will occur later than the previous forecast.
Update on Bevenopran Program
In light of the FDA planned March 2014 Advisory Committee meeting to discuss the potential for elevated cardiovascular events related to mu-opioid antagonists, and the challenges associated with enrolling patients in the current Phase 3 bevenopran trial, Cubist has decided to suspend its Phase 3 efficacy studies of bevenopran, a mu-opioid antagonist, in patients with chronic non-cancer pain and opioid-induced constipation (OIC). Instead, the Company will focus on the completion of its long-term safety trial of bevenopran, which is progressing ahead of schedule. Based on results of the safety trial and, following the Advisory Committee meeting, further guidance from the FDA on the potential for a more feasible Phase 3 efficacy study design, a decision will be made on how to best proceed with the bevenopran program in 2015.
Commitment to Discovery and Early-Stage Development
Cubists leadership in infectious diseases includes innovation in the discovery of new treatments to combat serious and life-threatening infections.
On January 10, Cubist announced that it submitted a Clinical Trial Application (CTA) to the Dutch Competent Authority and Ethics Committee to initiate a first-in-human study of CB-618 in the Netherlands. CB-618 is a novel, broad-spectrum beta-lactamase inhibitor (BLI) investigational compound discovered by Cubist. The purpose of the proposed study will be to evaluate the safety, tolerability and pharmacokinetics of CB-618. Based on in vitro pre-clinical studies, CB-618 has been shown to increase the spectrum of activity of certain beta-lactam antibiotics. If successful, CB-618 could help combat resistance to certain beta-lactam antibiotics and serious infections, including those caused by carbapenem-resistant Enterobacteriaceae (CRE) and Klebsiella pneumoniae carbapenemases (KPCs). CB-618 is part of a platform research approach by Cubist to identify new BLIs, which could potentially restore and expand the utility of certain existing antibiotics.
Other Pipeline Updates
In February 2013, Cubist entered into an option agreement under which Cubist has the exclusive right to acquire Adynxx, Inc. following receipt of data from Adynxxs Phase 2 trial for its lead product candidate, AYX1, a potential treatment for the reduction of acute pain and prevention of persistent and chronic pain following surgery. Following review of the Phase 2 data, Cubist has decided to not exercise its right to acquire Adynxx. While AYX1 was well-tolerated in this trial and the observed clinical response may warrant continued exploration, the magnitude of the clinical response did not meet Cubists exercise criteria.
The Company plans to host a conference call to discuss complete fourth quarter and year-end 2013 financial results on January 23rd at 5:00 p.m. ET. The conference call and webcast information is below.
