Neurochem is abandoning development of its Phase III oral beta-amyloid fibrillogenesis inhibitor Alzhemed (tramiprosate) for Alzheimer's disease, the Canadian firm said Nov. 8.

The announcement marks a major setback in the company's Alzheimer's program and highlights the challenges of developing promising AD drugs. Neurochem does, however, plan to move a follow-on compound, NRM-8499, into preclinical development.

The decision to discontinue development of Alzhemed is not entirely surprising. Neurochem announced in August that Phase III North American data showed no statistically significant difference in favor of Alzhemed versus placebo (¹ Also see "Poor “Site Effect” Profile? Neurochem Reports Inconclusive Alzheimer’s Data For Alzhemed" - Pink Sheet, 27 August, 2007.).

The drug had been given FDA fast track designation in July (² Also see "Neurochem’s Alzhemed On Fast Track For Alzheimer’s Disease" - Pink Sheet, 24 July, 2007.).

If approved, Alzhemed would have been the first drug on the market targeting the amyloid plaques characteristic of the disease, rather than Alzheimer's symptoms.

Spurred by the outcome of the Alzhemed program, the company also reported that it has refocused its business strategy on near-term goals.

"Neurochem will be dedicating its resources to build a strong and diversified pipeline of product candidates, which could potentially provide cash flow in the near term," CEO Francisco Bellini said during a same-day third quarter earnings call.

"As a result, our business model is moving away from one of a traditional biopharmaceutical company. We are building one that encompasses shorter-term projects, which could contribute to the funding of R&D activity complementing our longer term prescription drug program geared at the sustaining the company's substantial growth, including the next generation program with AD based on solid intellectual patent protection with a long patent life," Bellini noted.

The company is not giving up on tramiprosate altogether; in line with the new strategy, Neurochem is creating a separate company to market the agent as a branded nutraceutical, which will likely launch in 2008.

Neurochem noted that while tramiprosate did not show statistical significance in the primary endpoint in the North American trial, a preliminary post-hoc analysis, which allowed adjustment for potential confounding factors, showed a dose-dependent reduction in hippocampal atrophy in patients treated with tramiprosate versus placebo. Patients receiving 200 mg of tramiprosate per day experienced 65 percent less atrophy (P=0.036) and patients treated with tramiprosate 300 mg per day presented no atrophy (P=0.003).

Neurochem will halt its European Phase III clinical trial of tramiprosate, but plans to complete the ongoing North American study, which is considered "pivotal" to designing future trials for NRM-8499, Bellini noted.

The company will now direct its efforts to developing NRM-8499, a pro-drug of tramiprosate. Pharmacokinetic studies "have already demonstrated greater than five-fold increases in brain concentration as compared to the original tramiprosate molecule," according to Neurochem.

The firm is next conducting toxicology studies on NRM-8499, results of which are expected in the first quarter of 2008.

Neurochem also provided updates on several other ongoing programs. The firm said it had filed a response in September to a second FDA "approvable" letter for Amyloid A amyloidosis treatment Kiacta (eprodisate) (³ Also see "Neurochem Gets Second FDA “Approvable” Letter For Kiacta" - Pink Sheet, 18 July, 2007.).

The firm plans to initiate a Phase II clinical trial of eprodisate in diabetic patients in Canada in early 2008.

-Brooke McManus (b.mcmanus@elsevier.com)

Neurochem Drops Phase III Alzheimer’s Disease Candidate, Refocuses Pipeline

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