Stocks in News: KMDA, VVUS,
Discussion: Kamada Ltd. (KMDA) announced that EMA advisory group CHMP approved the general design of the Phase 3 clinical trial of inhaled alpha-1 antitrypsin (‘AAT) therapy for the treatment of alpha-1 antitrypsin deficiency (‘AATD). On the heels of the positive news, the share price climbed 4%. Based on the ongoing positive discussions with the FDA, the company expects to submit a revised trial protocol next year.
Two of the company’s drugs successfully completed Phase 3 trial and are in market now. Glassia (‘IV ATT) indicated for AAT deficiency was FDA approved in 2010 and is marketed in the U.S. by Shire. The same drug’s inhaled variant is in the above news. The other candidate that recently reached the market is commercially knows as KamRab/ KedRab (‘IM Anti-Rabies). The latter is indicated for prophylaxis for Rabies and is distributed through Kedrion Biopharma.
Phase 2 trial for Inhaled AAT was successfully completed but subsequently the NDA in the U.S. was withdrawn in June 2017. FDA is currently reviewing the path forward for the drug and its trial indications. Three more pipeline candidates of the company have reached Phase 2 trial without any major setbacks or reported major adverse events. These are D1-AAT (‘IV) indicated for Type 1 diabetes, G1-AAt (‘IV) in Graft vs Host Disease (‘GvHD) and L1-AAT (‘IV) in lung transplant. The company is either actively seeking partners or are in agreed collaboration for further development of all these candidates to move to the next trial phase.
Incidence of Rabies in the U.S. is extremely rare. Only three cases were reported in 2017 and exposure to all three reported cases was in most probability outside the country. However, the annual public health costs related to disease detection, prevention, and control have exceeded $300M. These public health costs include the vaccination of companion animals, animal control programs, maintenance of rabies laboratories, and medical costs, such as those incurred for rabies postexposure prophylaxis (PEP).
AAT deficiency is an under-recognized genetic disorder that leads to defective production of alpha-1 antitrypsin protein. AAT protein protects the body from the neutrophil elastase enzyme, a key tool for white blood cells to fight infection. This genetically transmitted disorder leads to lesser degree of AAT activity in the blood and lung. The situation is further complicated by deposition of abnormal AAT protein in the liver.
Even if AATD is a global phenomenon, its prevalence varies by population. It is estimated that the disorder affects about one in 1500-3500 individuals with European ancestry. People of Asian descent are usually not affected by the disorder. Even if the disorder is rare, estimates that 80,000 to 100,000 individuals in the United States have a severe deficiency of AAT suggest that the disease is under-recognized. It is estimated that globally more than 3M have allele combinations associated with severe AATD.
The small cap with $215.41M market cap last traded at $5.35; rather close to its 52-wk high of $6 it hit in January 2018. The stock has been gradually rising since last April and is right now close to its proper valuation. Those who could buy it at a long undervalued phase may look further to an even higher price as revised FDA review path leading to a fresh MAA and commencement of pivotal trial in EU become closer.
Nano cap Vivus (VVUS) announced positive data from a Phase 1 clinical trial evaluating VI-0106 in healthy volunteers. The company claims that the once-daily dosing may be adequate for the low tacrolimus concentrations. The low concentration is needed for the treatment of pulmonary arterial hypertension (‘PAH) with minimal monitoring of drug levels. The announcement spiked the share price 15%. Currently, tacrolimus indicated for PAH is the online pipeline candidate for the company. Tacrolimus has an interesting history. Last year, the company acquired the rights to commercialize tacrolimus and ascomycin for the treatment of PAH and related vascular diseases.
As a small biopharma Vivus has a good mix of commercial and clinical stage drugs and candidates. Their main focus is serious medical conditions that the company describes as “life-limiting”. Some of those conditions are obesity, sleep, and sexual health. They have two approved products spanning US and EU markets. Qsymia (phentermine and topiramate extended-release) capsule is approved in the U.S. and is indicated as an additional drug to regimen that includes the reduced-calorie diet and enhanced physical activity to manage chronic overweight.
Even if approved, FDA required a Risk Evaluation and Mitigation Strategy (‘REMS) for Qsymia. FDA recommends REMS as a strategy to manage potential serious risks associated with a drug to ensure that the benefits are superior to the risks associated with the drug. Company’s other commercial product avanafil, a phosphodiesterase 5 (PDE5) inhibitor, is marketed in two different names – Stendra and Spedra –in the U.S. and the EU respectively. In both the territories the drug is recommended for the indication of erectile dysfunction (‘ED).
In other news
IsoRay (ISR) announced a direct offering of its 11M common shares at 0.75/ share to institutional investors. Warrants to half of the number of those will be offered to such institutional investors. On the news of the offering previous day’s gains were wiped off and the share closed 40% down.
SCYNEXIS (NASDAQ:SCYX) announced selection of the 600 mg dose of SCY-078 (300 mg/12 hours) as the optimal one for its Phase 3 program in serious fungal infections. Phase 2b dose-finding study showed the amount to be the best combination of efficacy and tolerability.
BioXcel announced the formation of a Clinical Advisory Board on lead candidate BXCL501 for the treatment of agitation.
ReShape Lifesciences (NASDAQ:RSLS) announced that Boston’s Brigham and Women’s Hospital implanted its first ReShape Balloon for the treatment of obesity.
MiRagen Therapeutics (NASDAQ:MGEN) begun a 12-subject Phase 2 clinical trial evaluating MRG-201 in patients with a predisposition for keloid formation (scarring).
Achillion launches early-stage study of next-gen complement factor D inhibitorDosing is underway
Achillion Pharmaceuticals (NASDAQ:ACHN) is continuing the Phase 1 clinical trial evaluating next-generation complement factor D inhibitor, ACH-5548, in healthy volunteers.
A subgroup analysis of MediciNova’s (NASDAQ:MNOV) MN-166 (ibudilast) in patients with amyotrophic lateral sclerosis (ALS) demonstrated differentiated response pattern. It showed that “early ALS” subgroup showed more robust response.
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