Dive Brief:
- NeuroDerm said March 1 that preliminary Phase 2 results for ND0612H, its high-dose continuous, subcutaneously delivered levodopa/carbidopa (LD/CD) liquid formulation, met primary and other endpoints for patients with advanced Parkinson's disease. Its shares climbed 23% on the positive mid-stage news.
- The Israeli biotech, which is developing drug-device combinations for treating central nervous system disorders, said the small study of 38 patients, dosed via a belt pump, showed a statistically significant reduction in OFF-time (when people can’t perform routine activities) and an increase in patients ON time. It also showed a statistically significant reduction in dyskinesia.
- NeuroDerm said that after meeting with the European Medicines Agency, it decided to modify its EU clinical and regulatory development strategy, basing it on its restarted and amended iNDiGO Phase 3 efficacy study—expected to conclude in 2018—instead of bioequivalent pharmacokinetic studies.
Dive Insight:
NeuroDerm CEO Oded Lieberman described the preliminary Phase 2 results as "extremely encouraging," demonstrating ND0612’s potential to make a meaningful difference in the lives of Parkinson’s patients. More than 1 million Americans live with the disease.
Some analysts agreed. Cowen raised its price target on NeuroDerm to $70, up from $38, asserting that the results could transform care for late-stage Parkinson’s patients. Analyst Ken Cacciatore described initial feedback as very positive. "In general, our consultants believe the efficacy looks comparable to [AbbVie’s] Duodopa [approved by the Food and Drug Administration in 2015], but with a significantly improved use and safety profile. These data substantially de-risk the program. The current valuation is dramatically understated," he said.
Investors are showing increasing interest in the company. In fourth-quarter 2016, Opaleye Management was among a number of hedge funds and other institutional investors boosting their stakes in NeuroDerm.
NeuroDerm’s modified strategy in the EU, in addition to supporting a broader label, should "increase the clinical and commercial potential of ND0612 while not affecting our clinical timelines," Lieberman said. The CEO emphasized that the pharma aims to achieve its clinical and regulatory objectives as quickly as possible so as to offer a safer and more effective alternative to current treatment options.
NeuroDerm has another subcutaneous LD/CD product candidate in its pipeline, ND0612L, for treating moderate Parkinson’s disease. In addition, the pharma is developing ND0701, a novel subcutaneously delivered apomorphine formulation for patients with moderate to severe Parkinson’s disease who don’t respond well to LD/CD.
On Feb. 28, the Michael J. Fox Foundation led a policy forum on Capitol Hill attended by 200 Parkinson’s patients and caregivers. The group urged House members to support policies that safeguard federal research funding and access to care.