MRNS stock is moving higher in pre-market trading after an Oppenheimer analyst gave the stock a thumbs up.
Oppenheimer analyst Jay Olson initiated coverage of Marinus Pharmaceuticals with an Outperform rating and $6.00 price target. The analyst views the company as a leader in orphan epileptic disorders. Its lead development candidate ganaxolone has shown “promising” clinical data, says Olson, who estimates peak revenues of $1.1B in 2030.
Personally I’m not real jazzed about the 2030, time line as I may be dead in 11 years, hopefully not but I’m just saying that’s a heck of a long time horizon for revenue estimates.
On December 9, 2019, Marinus Pharmaceuticals announced clinical and regulatory updates for its orphan seizure programs in tuberous sclerosis complex, or TSC, CDKL5 deficiency disorder, or CDD, and PCDH19-related epilepsy. Marinus intends to initiate a Phase 2, open label study to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with seizures associated with TSC. Patient stratification from the company’s PCDH19-related epilepsy Phase 2 trial identified a subpopulation of patients with improved ganaxolone responses, those with low levels of allopregnanolone-sulfate, or Allo-S. Based on these data, the company performed a biomarker analysis to identify other rare genetic epilepsies that may benefit from the GABAA-receptor modulatory effects of ganaxolone and announced TSC as the next planned orphan epilepsy program to study the effect of ganaxolone on seizures as well as the expanded utility of a potential biomarker. The planned Phase 2 study will be conducted at approximately four to six sites in the United States and enroll approximately 20-40 patients ages two to 65. Patients will undergo a 4-week baseline period followed by a 12-week treatment period. The primary endpoint for the study is percent change in 28-day primary seizure frequency through the end of the 12-week treatment period relative to the 4-week baseline period. The European Medicines Agency has granted orphan drug designation for ganaxolone for the treatment of CDD. Enrollment in the study continues to progress as planned and the company remains on-track to report top-line data in Q3 2020. International site initiation and enrollment is continuing in the Violet Study, a single pivotal Phase 3 study evaluating oral ganaxolone in children with PCDH19-RE. The study will enroll up to 70 patients between the ages of one and 17 with a confirmed PCDH19 mutation. Patients are stratified into biomarker positive and negative groups, which could potentially provide the epilepsy community with the first diagnostic blood test that predicts the likelihood of a treatment response. The company remains on-track to report top-line data in 2021.