A New Orphan Drug, a New NDA, Two New PDUFA Dates and Other Research Developments

The FDA granted Orphan Drug status to TauRx’s LMTX for the treatment of frontotemporal dementia. LMTX is a small molecule Tau aggregation inhibitor. LMTX failed to demonstrate a cognitive improvement over placebo in two phase III trials.

The FDA accepted the NDA for Intra-Cellular Therapies’ lumateperone for Acute and residual schizophrenia, in mid-December 2018 and set a PDUFA Date of 9/27/19. The drug has Fast Track priority designation. It’s a dopamine receptor phosphoprotein modulator (DPPM) with selective action on dopamine D2 and D2 receptors, along with serotonin 5-HT2A receptors.

The PDUFA Date for Shield Therapeutics’ ferric maltol (Feraccru) is 7/27/19.

Amgen dropped co-development rights for axalimogene filolisbac in December 2018.

Axovant announced that in a 34-patient, 28-day, Phase II trial, nelotanserin did not reduce REM sleep behavior disorder compared to placebo in patients with dementia with Lewy bodies. Axovant has ceased development of nelotanserin for the treatment of dementia with Lewy bodies after the drug missed the primary endpoint.

In a 128 patient, Phase II trial, AbbVie’s veliparib added to cisplatin and etoposide did not improve progression-free survival compared to placebo (6.1 vs 5.5 months) in patients with extensive-stage small-cell lung cancer. Additionally, in a 130 patient, Phase II trial, veliparib added to FOLFIRI with or without bevacizumab did not improve progression-free survival compared to placebo (12 vs 11 months) in patients with metastatic colorectal cancer.

Marinus announced that in a 6-month, 11 patient, Phase II trial, treatment with ganaxolone resulted in a 54% reduction in seizure frequency in patients with PCDH19-related pediatric epilepsy who were positive for the neurosteroid allopregnanolone-sulfate (Allo-S) biomarker. In 4 patients that were negative for Allo-S, there was a 247% increase in seizure frequency. Marinus announced topline data from the 34-day, 58 patient, Phase II MAGNOLIA trial, where treatment with a 60-hour infusion of ganaxolone decreased the HAM-D17 score more than placebo by 4.2 points at 60 hours and 4.1 points at 34 days in 10 patients with postpartum depression that received the highest dose (140 µg/kg/h). Marinus is developing an oral form of ganaxolone and has an ongoing Phase II postpartum depression trial. 
Menlo announced that in an 8-week, 204 patient, Phase II trial, 33% of patients treated with serlopitant achieved a 4 point or > worst-itch numeric rating scale (WI-NRS) score compared to 21% with placebo in patients with pruritus associated with psoriasis.  

GenSight expects to report topline 48-week data from the Phase III RESCUE trial that is evaluating GS010 for the treatment of Leber’s hereditary optic neuropathy in 1Q19. 

XBiotech also has early trials evaluating bermekimab in the treatment of patients with moderate to severe atopic dermatitis.

Mesoblast’s remestemcel-L is approved as Temcell in Japan. The company plans to submit a BLA for remestemcel-L and request accelerated review in early 2019.

Novartis announced that in an 87 patient, Phase Ib dose ranging trial, treatment with alpelisib plus fulvestrant resulted in progression free survival (PFS) of 5.4 months at the maximum tolerated dose in postmenopausal women with PIK3CA-altered or PIK3CA-wild-type ER+ ABC breast cancer that progressed during or after antiestrogen therapy. PFS was longer in patients with PIK3CA-altered vs PIK3CA-wild-type tumors.