BPC December 14 update

VCEL obtains FDA Approval for MACI - shares up 57%

Price and Volume Movers

Vericel Corporation (NASDAQ:VCEL) surged over 57% on 112x average volume. The company announced that the FDA has approved MACI for the repair of symptomatic single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults. The FDA actually released a press release on Tuesday evening with the news, causing a flood of activity in yesterday’s evening session but the company waited until pre-market today to issue its press release.

Alcobra Ltd. (Nasdaq:ADHD) announced today that it met with the FDA to discuss its clinical hold of Metadoxine Extended Release (MDX). It expects to provide further details regarding the MDX regulatory path upon receipt of final meeting minutes from the FDA in the coming weeks. Shares pared losses to close down 4% on 5x average volume after being down over 12% until late in the session.

Proteon Therapeutics, Inc. (Nasdaq:PRTO) shares continued to drop on Wednesday with shares falling a further 14% on 10x average volume. The company announced yesterday that its first Phase 3 trial of vonapanitase (PATENCY-1) did not meet its primary endpoint.

Amicus Therapeutics, Inc. (NASDAQ:FOLD) announced after hours that it intends to offer $225m of convertible senior notes. Shares are currently down 10% in the after-hours session.

Shares of vTv Therapeutics Inc (NASDAQ:VTVT) are currently up 15% in the after-hours session following its release of Phase 2 data of TTP273 for the treatment of Type 2 diabetes. Although full data were not released the company did note that “TTP273 demonstrated a statistically significant reduction in HbA1c.”

Shares of Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) shares surged 22% on 100x average volume following presentation of new data from its Phase 2 study of NurOwn in ALS (amyotrophic lateral sclerosis).

Other price movers (+10%):


Northwest Biotherapeutics, Inc (NASDAQ:NWBO): +21%; 6x average volume.

Syndax Pharmaceuticals Inc (NASDAQ:SNDX): +14%; 3x average volume.


MacroGenics Inc (NASDAQ:MGNX): -15%; 4x average volume. RXi Pharmaceuticals Corp (NASDAQ:RXII): -11%.

aTyr Pharma Inc (NASDAQ:LIFE): -11%; 2.5x average volume.

Full pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst

Focal chondral cartilage defects in the knee.

Approved FDA Approval announced December 13, 2016 ahead of PDUFA date of January 3 2017.

Ocular inflammation and pain following cataract surgery

CRL CRL July 25 2016 - due to manufacturing deficiencies. NDA resubmitted, PDUFA date July 19, 2017. Further CRL issued July 11, 2017 with FDA noting deficiencies in manufacturing processes and analytical testing.

MDX (Metadoxine Extended Release (MG01CI))
Adult ADHD

Phase 3 Phase 3 data released January 17, 2017 - failed to meet primary endpoint.

Type 2 Diabetes

Phase 2 Phase 2 primary endpoint met - December 14, 2016.

LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)

Phase 3 Noted on December 14, 2016 that Phase 3 dosing has commenced. Early data released at EHA June 23, 2017.

Genital herpes

Phase 2b Phase 2b data released January 6, 2017. 12-month data released July 24, 2017. Phase 3 to commence 4Q 2017.

Ductal Carcinoma in Situ (DCIS)

Phase 2 Phase 2 trial initiation announced December 14, 2016.

Diffuse-large B-cell lymphoma (DLBCL)

Phase 2 Phase 2 initial data released August 3, 2017 - ORR 19%.

Restoring normal skin contours in for patients with HIV

Phase 3 Pivotal trial data released June 14, 2017 - primary endpoint met.

Alopecia areata

Phase 2a Phase 2 clinical hold placed May 17, 2017 lifted July 10, 2017. Trial completion due 2H 2018.


NDA Filing Planned NDA resubmission due October 2017.