BPC April 18 update

Ultragenyx (RARE) meets primary endpoint in XLH - shares +20% AH ; Nivalis (NVLS) merger shares +28; Argos (ARGS) spikes 84% AH following ADAPT update

Price and Volume Movers

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares have spiked up in the after-hours session following its announcement of 24-week data from its Phase 3 trial of burosumab (KRN23) in adults with X-linked hypophosphatemia (XLH). The study met its primary endpoint with patients exhibiting a statistically significant improvement in serum phosphorus levels, with 94% of patients achieving normal levels compared to 8% on placebo (p<0.0001). The trial met two of three secondary endpoints, missing the pain measured by the Brief Pain Inventory Question 3 endpoint. Shares are currently up 20% to $72.

Nivalis Therapeutics Inc (NASDAQ:NVLS) shares are currently trading up 28% to $3.37 following news that it will merge with privately owned Alpine Immune Sciences. Following the merger, current Alpine shareholders will own approximately 74% of the combined company and current Nivalis shareholders will own 26%.

Argos Therapeutics Inc. (Nasdaq:ARGS) reported an update from its Phase 3 ADAPT trial of Rocapuldencel-T in combination with sunitinib/standard-of-care for the treatment of newly diagnosed metastatic renal cell carcinoma (mRCC). As a refresher, the Independent Data Monitoring Committee (IDMC) recommended in February that the trial be discontinued due to lack of efficacy noting that it was unlikely to demonstrate a statistically significant improvement in overall survival. The company conducted its own selective post-hoc subgroup analysis in the first third of patients enrolled and as a result the company believes that based on the data it has reviewed the trial should be continued until completion. Shares are currently up 84% to $0.72. However, it should be noted that just prior to the negative IDMC ruling in mid-February, shares were trading at $4.40.

Motif Bio plc (Motif) (NASDAQ:MTFB) shares closed up 17% to $8.42 following its announcement that its Phase 3 REVIVE-1 trial of iclaprim in patients with acute bacterial skin and skin structure infections (ABSSSI), met its primary endpoint of non-inferiority (NI) (10% margin) compared to vancomycin.

Cytokinetics, Inc. (NASDAQ:CYTK) shares closed 10% higher to $12.95. The move followed an announcement Monday by S&P Dow Jones Indices that the company will be added to the S&P SmallCap 600 index on April 24.

Arena Pharmaceuticals, Inc. (NASDAQ: ARNA) shares dropped 14% to $1.28 following the pricing of its offering of 60m shares at a price of $1.15 per share for gross proceeds of $69m.


Other major price movers:

ADVANCERS:

XOMA Corporation (NASDAQ:XOMA): $7.41; +14%.

Akari Therapeutics PLC (ADR) (NASDAQ:AKTX): $14.97; +12%.

Catabasis Pharmaceuticals Inc (NASDAQ:CATB): $1.62: +10%.

Immune Pharmaceuticals Inc (NASDAQ:IMNP): $2.48; +9%.

Diffusion Pharmaceuticals Inc (NASDAQ:DFFN): $2.95; +8%.

Innocoll Holdings PLC (NASDAQ:INNL): $2.14; +8%.

 

DECLINERS:

Cleveland BioLabs, Inc. (NASDAQ:CBLI): $3.49; -27%.

Sunshine Heart Inc (NASDAQ:SSH): $1.35; -18%.

Sorrento Therapeutics Inc (NASDAQ:SRNE): $1.60; -15%.

Repros Therapeutics Inc (NASDAQ:RPRX): $1.07; -11%.

OHR Pharmaceutical Inc (NASDAQ:OHRP): %0.831; -10%.


Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst

ARGS
rocapuldencel-T (AGS-003) ADAPT Trial
Metastatic renal cell carcinoma (mRCC)

Phase 3 Phase 3 trial recommended be discontinued for futility - February 22, 2016. Noted April 18, 2017 that it intends to continue the trial to 290 events (analysis of overall survival) 1H 2018.

MTFB
Iclaprim -REVIVE-1
Acute bacterial skin and skin structure infections (ABSSSI)

Phase 3 Phase 3 data released April 18, 2017 - primary endpoint of non-inferiority met.

RARE
KRN23 Burosumab
X-Linked Hypophosphatemia (XLH) - adult

BLA Filing Phase 3 data released April 18, 2017 - primary endpoint met. BLA fiing announced August 24, 2017.

RDHL
BEKINDA
IBS-D

Phase 2 Phase 2 top-line data due September 2017.