BPC March 24 update

Biotech Stock Week in Review ~ March 24, 2017; TEN upcoming March/April biotech catalysts

Weekly watchlist

A number of companies released significant data over the last trading week, all of which were slated for March release in the BioPharmCatalyst FDA Calendar.


Nektar Therapeutics (Nasdaq: NKTR) announced Monday that it met the primary efficacy endpoint in its Phase 3 trial of NKTR-181 in patients with chronic back pain relief. Shares surged to close the day up 43% to $22.11 and closed the week higher at $22.42.

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced that it met the co-primary endpoints its two trials of JZP-110 in adult patients with excessive sleepiness associated with obstructive sleep apnea (OSA). Shares closed the session up 7% to $144.08, before closing the week at $143.14.


Aevi Genomic Medicine, Inc (NASDAQ: GNMX) announced Monday that it did not meet the primary endpoint in its SAGA trial of AEVI-001 in adolescents with mGluR mutation positive (mGluR+) ADHD. Shares ended the session down 59% to $2.21 and closed the week at $1.60, a fall of over 70%.

Xenon Pharmaceuticals Inc. (Nasdaq:XENE) announced Friday that its Phase 2 trial of XEN801 for the treatment of moderate to severe facial acne did not meet both its primary and key secondary efficacy endpoints. Shares tumbled to close down 53% to $4.65.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced Wednesday that it did not meet the primary endpoint in its Phase 2 study of UX007 in glucose transporter type-1 deficiency syndrome (Glut1 DS) patients with seizures. Shares dropped to close down 10% to $70.32 from Wednesday’s close.

Other highlights:

Esperion Therapeutics, Inc. (NASDAQ:ESPR) shares surged Monday to close the day up 74% to $41.22 before closing the week at $38.94. The company announced that its current late-stage program of bempedoic acid will be sufficient to support approval for low-density lipoprotein cholesterol (LDL-C). Data from the trials are due in 2018, with a New Drug Application (NDA) to be filed 1H 2019.

Merck & Co., Inc. (NYSE:MRK) and Pfizer Inc. (NYSE:PFE) received approval Thursday from the FDA for Bavencio (avelumab), the first FDA-approved treatment for Merkel cell carcinoma (MCC).

TEN Upcoming biotech stock catalysts below slated for March/April:

Drug Stage Catalyst

Endometrial cancer

Phase 3 Phase 3 trial did not meet primary endpoint - May 1, 2017.

Voclosporin - AURION trial

Phase 2 48-week data from open-label AURION study presented March 27, 2017 - primary endpoint met.

IV CR845
Uremic pruritus

Phase 2/3 Phase 2/3 top-line data from Part A portion of trial released March 28, 2017 - endpoints met. Phase 3 trial to be initated 4Q 2017.

Anabasum (Resunab)
Cystic Fibrosis

Phase 2 Phase 2 data released March 30, 2017 - primary endpoint met but some concerns regarding efficacy.

Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)

Phase 2b Phase 2b additional interim data released June 23, 2017 at EHA. Final top-line data due 2H 2018.

Alzheimer's disease

Phase 2 Phase 2 data released May 1, 2017 - primary endpoint not met.

Tardive dyskinesia

Approved Approved April 11, 2017.

AIR DNase (PRX-110)
Cystic Fibrosis

Phase 2 Phase 2 interim data released January 3, 2017. Full data released April 12, 2017. Company noted data were positive but results showed efficacy down on January data.

Cystic fibrosis

Phase 2 Phase 2 28-day data due 4Q 2017.

Lactose intolerance

Phase 2/3 Phase 2/3 top-line data released March 28, 2017. Primary endpoint met. End of Phase 2 meeting with FDA planned by the end of 2017 with Phase 3 trial to potentially commence 1H 2018.