During 2010, 73% of major clinical and regulatory outcomes were positive. For 2011 we expect a similar figure of 75%—underscoring a robust success rate. We have focused mainly on innovative programs (vs. formulations or me-too drugs) and are encouraged to note the biopharma industry’s progress from increasingly harnessing the fruits of “new science”.
Milestones in Tables 1 and 2 have been selected based on their potential to have a high impact on company valuation and/or an industry-wide impact through addressing high unmet medical needs. Accordingly, we have focused on results of late-stage clinical trials and key regulatory actions.
Of the 15 high-impact events in 2010 (through November 22), 11 were positive with only 4 negative outcomes. Importantly, of the negative developments, three of the four still have a decent chance of eventually becoming successful.
One of the most highly anticipated events earlier in the year was the FDA decision on Intermune’s Pirfenidone for idiopathic pulmonary fibrosis. Despite a positive 9–3 advisory panel vote in March, the FDA issued a Complete Response Letter (CRL) requesting an additional clinical study to support the efficacy claim for Pirfenidone. While the path forward for potential U.S. approval is far from clear, Intermune also has submitted a Marketing Authorization Application to the European Medicines Agency, and there remains some hope that the drug could be approved in Europe—helped by additional data from Japanese studies by Shionogi (www.shionogi.co.jp). This decision is expected in the first half of 2011.
In the popular obesity area, two closely watched drugs were also met with CRLs from the FDA. In case of Arena Pharmaceuticals (www.arenapharm.com) and Eisai’s (www.eisai.com) Lorcaserin, FDA had serious issues with both safety and marginal efficacy, and the fate of the drug seems pretty much sealed for now.
Vivus’ Qnexa, on the other hand, still has hope as its strong efficacy was never in question and many of the safety concerns raised by the FDA and advisory panel have been subsequently addressed with data from an extension study. We expect Qnexa to be ultimately approved in the second half of 2011.
Bydureon for diabetes from Amylin—and partners Alkermes and Eli Lilly—was expected to be a shoo-in at the FDA, and many were surprised by the setback. The main issue involves the potential for QT prolongation, which the companies were remiss in not having fully addressed prior to NDA submission. Previous studies with Bydureon (and exenatide—the underlying active drug) have not shown any significant association with QT prolongation. We expect Amylin to complete a thorough QT study and refile by the end of 2011, with approval expected in mid-2012.
Of the positive developments in 2010, the following are most notable:
- Approval of enoxaparin, a generic Lovenox from Momenta Pharmaceuticals and Novartis which begins to set forth a pathway for future biosimilar approvals,
- Approval of Novartis’ Gilenya, the first oral drug for MS,
- Positive panel recommendation for Human Genome Sciences and partner GlaxoSmithKline’s Benlysta, a potential new therapy for lupus,
- Amgen’s much-needed pipeline success with Xgeva (denosumab) for fracture prevention.