The release of solid clinical trial data is vital to the success of any small pharmaceutical company. These data releases can draw prolonged interest from small pharma investors, who buy shares in the hopes that the results are indicative of success in further clinical trials or the final parts of the FDA approval process.
The following 2 companies have (very) recently released new clinical trial data for their flagship products at the 65th American Academy of Neurology Annual Meeting, which is one of the biggest conferences for any nervous system-affecting drugs to "announce" themselves and draw attention from the medical community.
Those following these companies will spend the next few days pouring over the results, which may result in more overall interest from small pharma investors.
1.) Acadia Pharmaceuticals (ACAD) Releases Detailed Phase III Results at AAN 2013
In a press release yesterday, Acadia relayed some of the details of the Phase III trial for pimavanserin that were presented at AAN.
As you may recall, the -020 study tested pimavanserin's efficacy in the treatment of Parkinson's Disease Psychosis (PDP) and met its primary endpoint. The initial data release in late November 2012, which unveiled this news, sent ACAD over 130% higher in a single trading session due to the initial disbelief from the market that Acadia's drug actually works as well as shareholders had hoped.
Today, ACAD is nearly 25% higher on additional details, which I think detail why the drug has a much better shot at FDA approval than we thought. Adverse events were limited to odd urinary tract infections, and falls which would be classified as mild to moderate. The company is now valued at roughly $676 million.
The efficacy was based on something known as the SAPS-PD scale, which is honestly the best way that doctors can evaluate PDP patients. Pimavanserin outperformed the placebo arm with a comfortable margin (p=.001), and also had stellar performance in its secondary endpoint measuring motoric tolerability of the drug using the Unified Parkinson's Disease Rating Scale (UPDRS), which should ease concerns about the second most frequent adverse event (falls) in PDP patients on Pimavanserin.
Ultimately I think that Acadia could see a lot more interest in the next few trading sessions as investors think about the huge prospects that this drug could have in an untreatable neurodegenerative disease, and while we move closer to a potential NDA submission. These results also bode well for Pimavanserin's other development programs, which include Pimavanserin for the treatment of schizophrenia and Alzheimer's Disease Psychosis (ADP).
2.) Brainstorm Cell Therapeutics (OTCQB:BCLI) Ends Successful Phase I/II Trial For NurOwn
Also presenting at AAN was Brainstorm Cell Therapeutics, which announced positive phase I/II results for its NurOwn cells that are currently being developed for the Amyotrophic Lateral Sclerosis (ALS) indication.
The cells met all primary endpoints regarding the safety and tolerability in the trial, and also provided some encouraging results for ALS patients. This was shown with a statistically significant slowdown of disease progression in ALS patients treated with NurOwn cells as measured by FVC (Forced Vital Capacity) and ALSFRS-R (Revised ALS Functional Rating Scale) score which are designed to measure the progression of ALS.
Due to the lack of treatment options for ALS patients, this autologous stem cell therapy could be very exciting given that they are able to continue this kind of performance in future trials. Bullish sentiment is causing the stock to move higher today, with BCLI up nearly 7% at this time of writing this article to reach a new valuation of ~$38 million.
As Brainstorm moves into Phase II trials that will have the participation of the Mayo Clinic, I think the company should see interest from a broader base of biotech & small pharma investors. Phase II and III development will be a long and stressful process, but it generally provides investors with the biggest chance of outsized return on investment.
Also note that NurOwn received orphan drug designation in early 2011, and has substantial peak revenue potential given that it ultimately reaches the market despite a relatively small patient base.