Marinus up on small ganaxolone hope
- Marinus Pharmaceuticals gained nearly 25% in premarket trading on Monday after announcing preliminary data from a Phase 2 study of ganaxolone.
- The trial has only enrolled four patients, but showed a reduction in seizures in three patients with CDKL5, a rare genetic seizure disorder.
- Two patients have continued on in an extension study after six months of treatment. One patient discontinued due to lack of efficacy. Enrollment continues and topline results are expected in mid-2017.
Marinus lost 70% of its value in June — when it was trading at more than $6 per share — after it reported the failure of ganaxolone to reduce seizure activity in a Phase 3 study of patients with adult focal onset seizures.
Ganaxolone is a synthetic version of the naturally occurring anticonvulsant neuromodulator allopregnanolone, a derivative of progesterone. Like the benzodiazepines typically used to treat seizure disorders, ganaxolone is a GABA-A receptor agonist, but it acts on a different site than benzodiazepines.
The drug has multiple failures in its past, including a Phase 2 flop in Fragile X Syndrome, also announced last June, and another mid-stage failure in infantile spasms reported in 2009.
Despite all of the setbacks in the development of ganaxolone, Marinus has stayed optimistic about the compound. The company has relied on sub-group analyses and little glimmers of hope in small studies to continue pushing the compound forward.
Investors have not stayed so positive — the stock was down below $1 last week and now only trades about 15 cents above.
Marinus picked up the compound in 2004 after it was shelved by Purdue Pharma. The company has been developing the drug in an IV formulation, as well as liquid and capsule forms.
- Marinus Pharmaceuticals Statement
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