Akari up on advancement of nomacopan

Thinly traded nano cap Akari Therapeutics (NASDAQ:AKTX) is up 28% premarket on robust volume on the heels of its design of a two-part Phase 3 clinical trial evaluating Coversin (nomacopan) in pediatric patients hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA), an Orphan Drug- and Fast Track-tagged indication.

Part A will confirm the dosing scheme while Part B will assess the efficacy of the confirmed dosing regimen.

The trial should commence this month.

Concurrently, the company announced results on six paroxysmal nocturnal hemoglobinuria (PNH) patients receiving nomacopan in a long-term Phase 2 study. All are transfusion-independent after being transfusion-dependent at entry.

https://seekingalpha.com/news/3522906-akari-28-percent-premarket-advancement-nomacopan

ASLAN Pharma up 85% premarket on positive ASLAN004 data

Nano cap ASLAN Pharmaceuticals (NASDAQ:ASLN) rockets 85% premarket on increased volume after reporting positive preliminary data from a multiple ascending-dose study evaluating ASLAN004 for the treatment of moderate-to-severe atopic dermatitis (AD).

The first three participants who received the lowest dose (200 mg) for at least one month experienced reductions in EASI (AD scale) scores of 85%, 70% and 59% from baseline. EASI scores continued to fall at week 4 with maximum efficacy expected at weeks 6 – 8.

The second dose cohort should open later this month. The trial should wind up in H2 2020.

On the safety front, ASLAN004 was well-tolerated with no serious adverse events or discontinuations.

ASLAN004 is a fully human monoclonal antibody that binds to the IL-13 receptor α1 subunit (IL-13Rα1), blocking signalling of two pro-inflammatory cytokines, IL-4 and IL-13, which are central to triggering symptoms of atopic dermatitis, such as redness and itching of the skin.

https://seekingalpha.com/news/3522830-aslan-pharma-85-percent-premarket-positive-aslan004-data

Merck’s Keytruda nabs accelerated review in U.S. for bladder cancer

The FDA grants Priority Review status to Merck’s (NYSE:MRK) supplemental marketing application seeking approval to use Keytruda (pembrolizumab) to treat patients with high-risk non-muscle invasive bladder cancer with carcinoma in-situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder) and who have failed to respond to standard-of-care Bacillus Calmette-Guerin therapy.

The agency’s action date should be next month.

Shares up 1% premarket on light volume.

https://seekingalpha.com/news/3522842-mercks-keytruda-nabs-accelerated-review-u-s-bladder-cancer

EMA accepts Orchard’s marketing application for OTL-200 for MLD

The  European Medicines Agency (EMA) has validated Orchard Therapeutics’ (NASDAQ:ORTX) Marketing Authorization Application for OTL-200, an ex vivo, autologous, hematopoietic stem cell-based gene therapy that has been developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, for the treatment of metachromatic leukodystrophy (MLD).

Validation confirms that the submission is sufficiently complete to begin the formal review process.

Shares are up 2% premarket.

https://seekingalpha.com/news/3522849-ema-accepts-orchards-marketing-application-otlminus-200-mld

Lexicon loses appeal of sotagliflozin CRL; shares down 10% premarket

The FDA has denied Lexicon Pharmaceuticals’ (NASDAQ:LXRX) appeal of the CRL it received in March related to its marketing application for type 1 diabetes med sotagliflozin. Undeterred, it now plans to appeal the decision to the Center for Drug Evaluation and Research (CDER).

The company will host a conference call this morning at 8:00 am ET to discuss the matter.

Shares down 10% premarket on light volume.

https://seekingalpha.com/news/3522852-lexicon-loses-appeal-sotagliflozin-crl-shares-10-percent-premarket

Xenon Pharma up 23% premarket on Neurocrine Bio partnership in epilepsy

Thinly traded micro cap Xenon Pharmaceuticals (NASDAQ:XENE) is up 23% premarket on light volume in response to its license and collaboration deal with Neurocrine Biosciences (NASDAQ:NBIX) to develop treatments for epilepsy.

Under the terms of the agreement, NBIX will have an exclusive license to XEN901 and certain preclinical candidates. The partnership also includes a multiyear research collaboration aimed at discovering and developing additional assets.

XENE will receive $30M upfront, a $20M equity investment from NBIX based on a share price of $14.196, a $25M milestone payment triggered by the FDA acceptance of the XEN901 IND (expected to be filed in mid-2020), up to $1.7B in additional milestones, low double-digit-to-mid-teen royalties on U.S. sales of XEN901 and slightly lower royalties on ex-U.S. sales of XEN901.

XENE will be eligible to receive tiered mid-single-digit-to-low double-digit royalties on U.S. sales of other products (slightly lower rates ex-U.S.).

The company will host a conference call this morning at 8:30 am ET to discuss the collaboration.

https://seekingalpha.com/news/3522869-xenon-pharma-23-percent-premarket-neurocrine-bio-partnership-epilepsy

Aquestive +8% premarket on completing NDA submission for libervant buccal film

Aquestive Therapeutics (NASDAQ:AQST) completes the rolling submission of a NDA to the FDA for its therapeutic candidate Libervant (diazepam) Buccal Film for the management of seizure clusters.

The company plans to share the results from the single dose crossover study at the upcoming American Epilepsy Society 2019 Annual Meeting.

Shares are up 8% premarket.

https://seekingalpha.com/news/3522871-aquestive-plus-8-percent-premarket-completing-nda-submission-libervant-buccal-film