BPC February 15 update

Editas (EDIT) scores patent win - shares +29%; Ardelyx (ARDX) jumps on tenapanor data; Acura (ACUR) delist to OTC

Price and Volume Movers

The US Patent and Trademark Office ruled today that patents awarded to the Broad Institute CRISPR-Cas9 technology will remain. This follows a patent battle between Broad and the University of California over rights to the technology. News of the patent win saw shares of Editas Medicine Inc (NASDAQ:EDIT) close up $29% to $24.30. In 2014, the company formed a license agreement with the Broad Institute to access intellectual property and technology related to the CRISPR/Cas9 genome editing systems. On the other side, shares of Crispr Therapeutics (NASDAQ:CRSP) closed down 8% to $15.90 while shares of Intellia Therapeutics Inc (NASDAQ:NTLA) dropped 9% to $12.50, paring earlier losses in the the early afternoon session. Both companies were founded using CRISPR/Cas9 biology based on research carried out at the University of California.

Acura Pharmaceuticals, Inc. (NASDAQ:ACUR) announced after hours that its shares will begin to trade on the OTCQB Market under the same ticker from February 23, 2017. Shares are currently down 34% to $1.16.

Ardelyx, Inc. (NASDAQ: ARDX) announced that it met the primary endpoint in its Phase 3 trial of tenapanor for the treatment for hyperphosphatemia in patients with end-stage renal disease (ESRD) who are on dialysis. The company noted a 39% diarrhea rate which resulted in 7.8% of patients discontinuing the trial, lower than its previous Phase 2 trial. Shares rose 14% to $13.00.

Auris Medical Holding AG (NASDAQ: EARS) announced the pricing of its previously announced public offering of 10m shares and 10m warrants, with each warrant entitling its holder to purchase 0.70 of a common share (one unit).  The offering will be priced at $1 per unit for gross proceeds of $10m. Shares closed down 19% to $0.934.

Celsion Corporation (NASDAQ:CLSN) also announced the pricing of a public offering with expected total gross proceeds of approximately $5m. The offering, priced at $0.23 per share included warrants to purchase 0.75 of a share of common stock, at an exercise price of $0.23 per share. Shares closed down 33% to $0.22.

Evoke Pharma, Inc. (NASDAQ:EVOK) shares spiked at the open and closed up 30% to $3.40 following notice that it has received a letter from FDA exempting its late stage product, Gimoti from a Human Factors (HF) Validation study requirement prior to submission of a New Drug Application (NDA), slated for filing by the end of 2017.

Other major price movers:


Zosano Pharma Corp (NASDAQ:ZSAN): $2.85; +41%.

Novan Inc (NASDAQ:NOVN): $6.44; +34%.

AVEO Pharmaceuticals, Inc. (NASDAQ:AVEO): $0835; +15%.

Dicerna Pharmaceuticals Inc (NASDAQ:DRNA): $3.22; +14%

Novavax, Inc. (NASDAQ:NVAX): $1.62; +14%.


Delcath Systems, Inc.(NASDAQ:DCTH): $0.156; +21%.

Galectin Therapeutics Inc (NASDAQ:GALT): $1.47; -18%.

Bellerophon Therapeutics Inc (NASDAQ:BLPH): $0.90; -12%.

Neothetics Inc (NASDAQ:NEOT): $1.41; -10%.

BioDelivery Sciences International, Inc.(NASDAQ:BDSI): $1.90; -7%.


Full pipeline updates:

Pipeline Database Updates

Drug Stage Catalyst

ALKS 5461
Major depressive disorder

NDA Filing Phase 3 data from first two trials did not meet their endpoints. FORWARD-5 data, the third of three trials did meet endpoints - October 2016. Rolling NDA filing has commenced - noted August 21, 2017 with submission to be completed January 2018.

Hyperphosphatemia in end-stage renal disease (ESRD) patients

Phase 3 Phase 3 released February 15, 2017 - primary endpoint met. Second Phase 3 trial initiation due following feedback from FDA.

Gimoti - EVK-001
Female diabetic gastroparesis

Phase 3 Phase 3 data released July 2016. Primary endpoint not met. However, the company noted December 15, 2016 that following discussions with FDA regarding possible NDA submission, no new efficacy or safety study would be required, if bioequivalence criteria were met in a pharmacokinetic (PK) trial. PK data released October 23, 2017, criteria met with NDA filing due 1Q 2018.

Progressive multiple sclerosis (progressive MS)

Phase 2b Phase 2b top-line data released October 28, 2017 at Joint ECTRIMS – ACTRIMS meeting. Primary endpoint met.

Tumor Treating Fields (TTFields) LUNAR
Non-small cell lung cancer (NSCLC)

Phase 3 Phase 3 commencement of enrollment announced February 15, 2017. Data due 2021.

Psoriatic Arthritis

Phase 1b Phase 1b data released September 28, 2017 - insufficient meaningful clinical effect for further development.

AL Amyloidosis

Phase 3 Phase 3 trial fully enrolled - noted August 8, 2017. Data likely due 2020.

Previously-treated patients with AL amyloidosis and persistent cardiac dysfunction

Phase 2b Phase 2b data due 2Q 2018.

Parkinson's disease

Phase 2 Phase 2 planned for 2Q 2017.

SB 9200
Hepatitis B (HBV)

Phase 2a Phase 2a initial data released May 23, 2017. Additional data from first cohort to be presented September 7, 2017. Second cohort top-line data released November 15, 2017.

HETLIOZ (tasimelteon) - pediatric formulation

Phase 1 PK trial initiated with full enrollment due by end of 2017.

HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

Phase 3 Phase 3 initiated 4Q 2016. Data due 2018.

Atopic dermatitis

Phase 2 Phase 2 data released September 13, 2017 - primary endpoint not met. Phase 3 trial to commence 1H 2018.

HETLIOZ (tasimelteon)
Jet Lag Disorder

Phase 2 Phase 2 data due in 1Q 2018.