Recently, dreadful, scandal-plagued, bio-dreck Galena Biopharma (GALE) released results (here) from their Phase 2 study of GALE-401 aka Anagrelide CR. This is a new “Controlled Release” formulation of an existing generic drug, Anagrelide. Anagrelide is used to lower a patient’s platelet count. And just like Galena itself, there is less to Anagrelide CR than meets the eye.
Galena, at the time run by (now-disgraced) Mark Ahn, picked up the rights to the product in January 2014, through the acquisition of Mills Pharma, a paper entity with one asset, Anagrelide CR. Mills was owned by a firm called Aceras Biomedical. Aceras was funded by now defunct bucket shop Rodman & Renshaw, and run by a ragtag group of refugees from Steve Rouhandeh’s stock touting SCO entities and Lindsay Rosenwald’s bio-dreck factory Paramount. [Read more about stock promoter Steve Rouhandeh and SCO in a Wall Street Journal article by Aaron Elstein, here.]
Interestingly enough, Mark Ahn still serves on the board of Steve Rouhandeh’s recently re-awakened Plasmatech Bio (PTBI) stock promotion, where SCO is one of the largest holders, and several current and former SCO employees are on the Board of Directors.
Given the nature of the players involved, if the sharks at Aceras really had a winner, they would have tried to take it public during the greatest bull market in biotech history, or at the very least stuffed it into reverse merger with a shell they controlled. Instead they sold Anagrelide CR to pathetic, desperate, Galena. As typical for Galena, even the purchase price for Mills was obfuscated. Here is the relevant language from the press release:
Under the terms of the agreement, Galena paid an up-front payment to Mills Pharmaceuticals’ owners. Additionally, Mills Pharmaceuticals owners are eligible to receive one-time payments of up to 4,000,000 shares with the achievement of specified regulatory milestones. The owners of Mills Pharmaceuticals are also eligible to receive $3 million upon FDA approval of a new drug application in respect to GALE-401.
However in the corresponding 8-k filing, available here, Galena details the actual purchase price of Mills:
- $1.60mm in cash up-front.
- $0.20mm in cash post-closing, subject to adjustments.
- Up to 3mm shares of GALE upon initiating the first clinical trial of Anagrelide CR aka GALE-401.
- Up to 3mm shares of GALE upon initiating the first Phase 3 clinical trial of GALE-401.
- $3mm in cash upon FDA approval of GALE-401.
While Galena management, and the sell side shills hype GALE-401 incessantly, the truth is that the drug is doomed to failure both clinically and commercially.
Here is what Galena said about GALE-401 at the time of the acquisition:
GALE-401 is expected to greatly decrease the adverse event rate relative to the approved product…Existing data strongly suggest reducing the Cmax while maintaining the overall exposure to the drug, or AUC (area under the curve), reduces the rate of adverse events without compromising efficacy. GALE-401 significantly decreases the Cmax by up to 70% while preserving nearly 100% of the AUC.
We can compare the recent GALE-401 study, with an older study of generic Anagrelide of comparable size. Back in 1992, a study of regular anagrelide was conducted on 19 evaluable patients. Further information on this study is available here.
So how did Galena do? Were they able to reduce adverse events versus normal, generic, Anagrelide? Were they able to match generic Anagrelide’s efficacy? What about dosing convenience with the new controlled release formulation?
First lets examine safety. According to Galena:
The Phase 2 study demonstrated that GALE-401 was well tolerated with primarily Grade 1 and 2 toxicities in 16 of the 18 subjects
16 out of 18 subjects (89%) reported “toxicities” aka adverse events, aka side-effects.
In the 1992 study, only 8 out of 19 patients (42%) reported adverse events. The regular formulation causes fewer than half as many adverse events as Galena’s supposedly improved version. GALE-401 fails on safety.
OK, Galena bulls ask, maybe there is a difference in efficacy, favoring GALE-401? Let’s see.
In Galena’s recent study of 18 patients, results showed:
Complete Responses in 7 out of 18 patients, a CR rate of 39%. Partial responses in 7 out of 18 patients, a PR rate of 39%, leading to a combined ORR (Overall Response Rate) of 78% (14/18).
How does this compare to generic Anagrelide? The study from 1992 showed complete responses in 13 out of 19 patients, for a CR rate of 68%. Partial responses in 3 out of 19 patients, a PR rate of 16%, leading to a combined ORR of 84%.
While the ORRs are close, generic Anagrelide is still superior. And comparing CRs, which is obviously the most important measure, generic Anagrelide outperforms GALE-401 by an astounding 174%. Cheap, generic, Anagrelide provides nearly double the rate of complete responses as Galena’s lame CR formulation.
Well, even if it is not as safe, and not as effective, maybe Galena’s Anagrelide CR will be more convenient for patients and improve compliance?
In the Galena study GALE-401 was taken by patients twice a day. In the generic Anagrelide study, surprise, the regular formulation was also taken twice a day.
Results are summarized in the table below.
Controlled release formulations are supposed to have a gentler side-effect profile, be equally if not more effective, and be more convenient for patients with less frequent dosing. Galena’s Anagrelide CR failed in every possible way. GALE-401 is a clinical joke.
Commercially, Galena’s track record is so poor it would be better if they had no record. Their first attempt, the re-launch of a me-too fentanyl product called Abstral, was both controversial and mind-bogglingly underwhelming. See a chart of recent drug (re)launches, including Galena’s Abstral, here.
Galena has touted GALE-401 as having a $200mm market opportunity (TAM). Back in January 2014 the company said:
GALE-401 has an estimated peak market size of approximately $200 million in the U.S.
But this is bullshit. The entire American market for Anagrelide in 2014 was barely $100mm. A prescription for generic Anagrelide can be purchased for about $25.00/month.
The competitive landscape is fierce. Take a look at the current providers of Anagrelide in the US:
Galena can hardly sell a highly addictive pain killer, how are they going to compete against a cheap generic, that works better, has a lower rate of side effects and is equally convenient with twice a day dosing?
Not that it matters, as Anagrelide CR is clinically irrelevant, but Galena has no IP protection in the US for GALE-401. From the most recent Galena 10K:
Anagrelide hydrochloride, the sole active pharmaceutical ingredient, or “API,” in GALE-401, has been approved for many years and, thus, it is not possible to obtain composition of matter patents that cover anagrelide hydrochloride. As a result, competitors who obtain the requisite regulatory approval can offer products with the same API as GALE-401, so long as the competitors do not infringe any formulation patents that we may have or may obtain or license, if any. The only patent protection that we have or are likely to obtain covering GALE-401 are patents relating to very specific formulations, methods using these formulations, and methods of manufacturing and packaging. We have two granted patents in the United Kingdom that expire in 2019 and we are prosecuting pending patent applications in other territories including but not limited to the U.S. and Europe, which may not issue prior to any potential commercialization of GALE-401.
GALE-401, just more lies from Galena management and their sell side shills, whoring themselves out for banking business.