Your Daily Pharma Scoop: Galectin Phase 3 Trial, Acer NDA, Chiasma SPA Amendments

Analysis focus: GALT

Galectic (NASDAQ:GALT) was our focus ticker in one of the articles in the NASH series we did in February. There, we discussed what makes Galectin stand out among the NASH companies. First, it is not a one-trick pony and has a diverse pipeline outside NASH. Second, unlike Genfit and Intercept, it targets a more difficult area of NASH, namely cirrhosis and fibrosis, and therefore the bets are very high here.

Heres what we wrote: With GR-MD-02, Galectin is also targeting a different spectrum of NASH. The companys programs with GR-MD-02 are focusing on NASH treatment in individuals with advanced fibrosis and cirrhosis. As we mentioned before, Genfit (OTCPK:GNFTF) and Intercept (ICPT) are targeting what can be termed as the sweet spot of the NASH spectrum. Galecctin is one of the few companies targeting advanced stage of the disease. This makes it both an interesting and at the same time risky bet in the NASH space.

Now, GR-MD-02 has done very well in the various trials it has been through. It has actually been shown to reverse fibrosis and cirrhosis in animal models. That is a tremendous achievement; just to halt the advance of fibrosis is a noteworthy achievement in itself, but to reverse it is a miracle. So thats what happened in the animal model.

In a phase 2 trial, the drug candidate failed to meet its primary endpoint; however, in a specific subset of patients, it showed strong efficacy. As we said before: In December, Galectin reported results from the phase 2b clinical trial, NASH-CX, which was evaluating the lead candidate in NASH cirrhosis patients with portal hypertension. The study failed to meet the primary endpoint of a statistically valid reduction in hepatic venous pressure gradient (HVPG) compared to placebo in all subjects. This sparked a sell-off initially but a subset (50%) of the participants, those who did not have esophageal varices, showed a statistically significant improvement in HVPG compared to placebo. Despite the mixed data, GALT now plans to advance GR-MD-02 to phase 3 stage.

Now, esophageal varices cause a change in portal vein blood pressure, and HVPG or Hepatic Venous Pressure Gradient was the primary endpoint in the study. So, it makes sense that patients without esophageal varices benefitted more from the drug than otherwise. As the trial data said, A statistically significant clinical outcome effect of GR – MD – 02 treatment was observed on reducing the development of esophageal varices in patients without varices at baseline.

So now Galectin has announced its plans to take the drug candidate to phase 3 trial in patients without esophageal varices. The primary endpoint will be either the change in hepatic venous pressure gradient (HVPG) or the progression to esophageal varices (the FDA has agreed that either may be acceptable).

Stocks in News: Analysis of ACER, CHMA

Acer Therapeutics on track for Edsivo NDA in June/July

Discussion: Acer Therapeutics (NASDAQ:ACER), a small biotech with only $113mn in market cap, will submit an NDA for Edsivo by June/July. The target indication is a rare inherited disease called vEDS or vascular Ehlers-Danlos syndrome, where excessive collagen causes translucent skin, easy bruising and fragile arteries, muscles and internal organs. Last year, the company reported positive results from a pivotal trial of Edsivo. There may be between 2200-5000 vEDS patients in the US, and the drug is already approved in some EU territories. In 2010, a trial, as reported in Lancet, was stopped early due to significant benefits observed.

FDA signs off on redefinition of certain endpoints in Chiasma’s late-stage study of Mycapssa in acromegal

Discusison: The FDA has agreed to redefine certain secondary endpoints in a phase 3 study of Chiasmas (NASDAQ:CHMA) MYCAPSSA in adults with acromegaly. The trial was under an SPA, and redefining the original fourth and fifth secondary endpoints to descriptive statistics measured within each treatment group will favorably help the company.

In other news

Pfenexs (NYSEMKT:PFNX) PF708 has demonstrated a comparable profile with Eli Lillys Forteo in osteoporosis patients. PFNX will file an NDA next quarter.

The FDA has accepted a resubmitted BLA from Coherus (NASDAQ:CHRS) for Neulasta biosimilar, and PDUFA is on November 3. The original filing was rejected last year citing the need for a reanalysis of a subset of subject samples with a revised immunogenicity assay and additional manufacturing-related process information.

Mynomo (NYSEMKT:MYO) announced that its MyoPro powered brace will likely get Medicare reimbursement from Jaunary 1, 2019.

Sanofi (NYSE:SNY) now owns over 95% of Ablynx through a tender offer in theUS and Belgium, and is trying to acquire all remaining shares after its 3.9B scoop up of the company.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.


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