Biotech Stock News (AVXL) (BIIB) (LLY) (SPHS) (KBIO)

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Anavex Life Sciences (NASDAQ:AVXL)

On November 9, 2015 Anavex reported positive phase 2a results for the trial treating patients with Alzheimer’s. This trial tested two clinical endpoints. The first clinical endpoint was testing for safety as it is customary with all phase 2a trials. The second endpoint was looking for preliminary clinical efficacy. In terms of safety, the company’s clinical compound Anavex 2-73 was safe and well tolerated in all patients.

In terms of efficacy the drug displayed that it worked in improving patient clinical outcome. The drug was able to score positively with 32 patients in all four preliminary tests measured:

  • Event-related Potentials
  • P300 Tests
  • mini-mental state examination — MMSE
  • Cogstate battery

All tests came out positive as an early look. One test in particular known as the “Event-Related Potentials” test was very good. That is because it showed that Anavex 2-73 achieved statistical significance. Meaning, that the trial obtained a p-value of p < 0.0007. Why is this p-value a good thing? All clinical trials, that attempt to muster through the FDA, must obtain a statistically significant p-value. While these results are preliminary in nature, it doesn’t dispute the fact that they are positive.

Why the huge drop the same day results were announced? The results weren’t bad but there were a lot of bearish articles that tanked the share price. I would say it is best to ignore those articles as they don’t jump into the science of the results. Instead, they only look at the promotions the company has done in the past. After all, if the results of the PART A portion of the trial were so bad patients wouldn’t have gone on to join the PART B portion.

Other big pharma companies like Biogen (NASDAQ:BIIB) and Eli Lilly (NYSE:LLY) have been struggling with their Alzheimer’s research. Biogen saw a setback with its Alzheimer’s drug in the phase 1b trial, known as the PRIME trial. This is because the company saw good results using its 3 mg and 10 mg of its drug, but failed to produce similar results in the 6 mg dose cohort. The drug was shown to reduce amyloid plaques in the brain but had no effect on cognition like Anavex did. Eli Lily saw some good preliminary results, but they tested their trials by comparison. That means they took one of their phase 3 trials and compared it with the other. They looked at patients who took treatment early compared to those that took treatment afterwards. If you want to pick a winner in the Alzheimer’s space then Anavex is your best bet based off statistical results to date.

Sophiris Bio (NASDAQ:SPHS)

On November 10, 2015 shares of Sophiris closed the day up 180% after the company announced positive phase 3 results for patients with BPH. First we must understand what BPH stands for to move further. BPH stands for  benign prostatic hyperplasia — enlarged prostate gland. This is a fairly big market as there are about 3 million people in the U.S. each year who are diagnosed with this problem. The phase 3 trial that is treating these patients with BPH uses a drug known as PRX302.

The reason for the huge surge in share price is because the company met on the primary endpoint of the study. The drug demonstrated statistical significance in the International Prostate System Score — IPSS – which was measured by a total score from baseline. The primary endpoint of the study produced an IPSS score of 7.60 versus 6.58 on the placebo side. This means that the drug achieved statistical significance with a p-value of p = 0.043.

The company will now have to run another phase 3 trial with a similar clinical design. Only if the next phase 3 trial comes out positive can Sophiris file an NDA to the FDA for approval of its PRX302 drug. As mentioned above the market opportunity is huge, because this drug could potentially earn the company $2 billion in revenue annually. I think that these positive results should be reproduced again, and the share price should continue to trade up accordingly.

Kalabios Pharmaceuticals (NASDAQ:KBIO)

On November 14, 2015  Kalabios announced that it would liquidate its assets as it could no longer continue operations with the current cash on hand. In addition, the company stated that they would not be able to obtain the proper funding in the amount of time allotted, to continue with other drugs in the pipeline. The whole downward spiral started with two different trials failed to produce meaningful results.

Back in 2014 Kalabios had announced that its Asthma drug, KB003, had failed a phase 2 study. That was a bad setback, but things got worse the next year when the company announced that its phase 2 drug in patients with Cystic Fibrosis had also failed. Both of these failures really hit the company hard but it seemed like it was going to recover until this new liquidation announcement. Beforehand the company announced that it wanted to target new oncology compounds.

The two oncology compounds that were going to be targeted were hematological malignancies — blood cancer of the bone marrow — and  Chronic myelomonocytic leukemia — another blood cancer. The hematological malignancies drug was in a phase 2 clinical study, and the CMML study was in a phase 1 study. The lack of money really took this down, as the company chose to liquidate instead of attempt to find funding for the company. Did theytake the easy way out? That’s hard to say for sure, but one thing for sure is that investors have been left holding the bag. The company in charge of this liquidation effort is known as The Brenner Group.

[Image Courtesy of Flickr]

1 COMMENT

  1. Hi Terry, nice concise and unbiased article discussing the preliminary science and results. May I point out that they also got back preliminary ACDS-ADL (Activities of Daily Living) for the 12-week Part B on 14 of the first 32 patients and 11 of 14 (78%) showed improvement with an AVERAGE improvement for all 14 patients of over 3 points. I know it is a small sample size and a short time frame, yet the FDA considers ACDS-ADL one of two critical metrics in AZ treatments.

    The leading drug, Aricept and it’s ‘heir apparent’ RVT-101 have both shown statistical significance to NOT be able to improve patients ACDS-ADL from baseline. They both claim ‘improvement’ which simply means patients do not regress as MUCH as they do w/o the drug but their critical ADL do nonethe less regress and steadily over 4 years. The FDA itself said the improvement over the placebo was not enough to have any functional bearing on patients lives and that in any event stressed that the drugs did NOT improve patient ACDS-ADL.

    The likelihood of improvement in 78% of patients even in that small sample size is minuscule when compared to the huge amount of data Aricept and RVT have run vs placbo in large and long-term studies and even vs untreated nursing home populations done to test ACDS-ADL regression in AZ populations in general. The FDA also said that a single point in improvement in patient ADL (over baseline) is a measurable difference in their quality of live and reduction of both caretaker time and cost of support. This test to me is the most impressive both in terms of likelihood of it’s outcome and the promise to AZ patients if the results are replicated in the other 18 patients and over time for all. This would mean the ONLY drug ever that can actually improve ADL in AZ patients and increase quality of live and reduce cost of care. I don’t think this should be overlooked.

    Again thanks for a great article!

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