BPC June 06 update

​La Jolla LJPC +92% on mid-stage data; Xeris XERS PDUFA date extension

Price and Volume Movers

La Jolla Pharmaceutical Company (Nasdaq: LJPC) shares closed up 92% to $10.24 following the release of interim analysis of its Phase 2 trial of LJPC-401 in patients with hereditary hemochromatosis (HH). The change in Transferrin Saturation (TSAT) from baseline to the end of treatment (16 weeks), the primary efficacy endpoint, was statistically significant, with a mean reduction in TSAT of 42% compared to placebo patients who had a mean reduction of 6% (p<0.0001). Top-line results of LJ401-HH01 are due in 2H 2019.

Protalix BioTherapeutics, Inc. (NYSE American: PLX) shares closed up 35% to $0.50 on news that it plans to file a Biologics License Application (BLA) for pegunigalsidase alfa for the treatment of Fabry disease in 1Q 2020 via the FDA’s Accelerated Approval pathway, using data from its completed Phase 1/2 and ongoing Phase 3 BRIDGE clinical trials.

CRISPR Therapeutics AG (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: CRSP) announced they have entered into a collaboration and license agreement to develop products for the treatment of Duchenne Muscular Dystrophy (DMD) and Mytonic Dystrophy Type 1 (DM1). CRISPR will receive a $175m up-front payment from Vertex and is eligible to receive milestone payments from Vertex of up to $825m. Shares of CRISPR are trading up 10% $41.01 after hours.

Motif Bio plc (Nasdaq: MTFB) shares slid 43% to $1.13 on the release of minutes from its Type A meeting with the FDA regarding its Complete Response Letter (CRL) issued for iclaprim, for the treatment of acute bacterial skin and skin structure infections (ABSSSI). The minutes noted an additional clinical trial will be required prior to granting marketing approval to address the continued concerns about potential liver toxicity.

Xeris Pharmaceuticals, Inc. (Nasdaq: XERS) announced the FDA has extended the PDUFA date for its New Drug Application (NDA) of Gvoke (ready-to-use glucagon injection) by three months to September 10, 2019. The extension follows submission of an information request from the FDA, which constitutes a major amendment to the NDA, and a subsequent extension. Shares, which closed down 16%, are trading down a further 6% to $7.51 after hours.

Jaguar Health, Inc. (NASDAQ: JAGX) announced a 1-for-70 reverse stock split of its shares will be effective as of June 7, 2019. Shares closed down 21% to $0.13.

Insys Therapeutics, Inc. (NASDAQ: INSY) has agreed to pay $225m and will plead guilty in its settlement following investigations of the company using kickbacks and other marketing practices for its painkiller product, Subsys. The company has agreed to pay $195m relating to civil statutory claims pursuant to the False Claims Act. The company also agreed to plead guilty to five counts of mail fraud which included $30m in fines and forfeiture. Shares of the company closed up 33% to $1.15.

ContraVir Pharmaceuticals, Inc. (NASDAQ:CTRV) shares closed up 42% to $7.08 after the nano-cap traded as high as $15.75 early in the session. The move was on the back of speculative trading following the release of data from a preclinical trial in mice of CRV431 on liver fibrosis.

Celgene Corporation (NASDAQ:CELG) announced that the FDA has accepted for review the New Drug Application for ozanimod for the treatment of people with relapsing forms of multiple sclerosis (RMS) in the United States. The European Medicines Agency (EMA) also accepted for review the Marketing Authorization Application for ozanimod for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS) in the European Union. The PDUFA date is March 25, 2020, while a decision from the EMA is expected in 1H 2020. The acceptance followed an initial Refusal to File letter received from the FDA following its initial application in February 2018.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Avadel Pharmaceuticals plc (AVDL): $1.92; +20%.

Titan Pharmaceuticals, Inc. (TTNP): $1.4; +15%.

Novan, Inc. (NOVN): $2.99; +14%.

Cidara Therapeutics, Inc. (CDTX): $1.68; +13%.

Protagonist Therapeutics, Inc. (PTGX): $10.66; +10%.

DECLINERS:

Benitec Biopharma Limited (BNTC): $1.04; -34%.

Axovant Gene Therapies Ltd. (AXGT): $5.69; -16%.

Allena Pharmaceuticals, Inc. (ALNA): $4.69; -16%.

Aeterna Zentaris Inc. (AEZS): $2.46; -13%.

ADMA Biologics, Inc. (ADMA): $3.77; -13%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AVXL – Anavex Life Sciences Corp.
ANAVEX 2-73 - AVATAR
Rett syndrome

Phase 2 Phase 2 trial initiation announced June 6, 2019.
$139.5 million

AXGT – Axovant Gene Therapies Ltd.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

Phase 1/2 Phase 1/2 three-month data due 1H 2020.
$111.2 million

AZN – Astrazeneca PLC
Calquence
Chronic lymphocytic leukaemia

Approved FDA Approval announced November 21, 2019.
$125.1 billion

BMY – Bristol-Myers Squibb Company
Ozanimod - RADIANCE
Relapsing Multiple Sclerosis

PDUFA PDUFA date March 25, 2020.
$140.5 billion

GSK – GlaxoSmithKline PLC
Mepolizumab
Severe eosinophilic asthma - pediatric

Approved FDA Approval announced June 6, 2019.
$113.5 billion

LLY – Eli Lilly and Company
Taltz (Ixekizumab) and adalimumab
Psoriatic Arthritis

Phase 3 Phase 3 trial met primary and key secondary endpoints - December 17, 2018.
$114.9 billion

MTFB – Motif Bio plc
Iclaprim
Acute bacterial skin and skin structure infections (ABSSSI)

CRL CRL issued February 14, 2019. Noted June 6, 2019 that further trials are required.
$2.9 million

PLX – Protalix BioTherapeutics Inc. (DE)
Pegunigalsidase alfa (PRX-102)
Fabry disease

BLA Filing BLA filing due April 2019.
$46 million

VTVT – vTv Therapeutics Inc.
TTP399
Type 1 Diabetes

Phase 1/2 Phase 2 Part 2 data due late-1Q 2020.
$93.3 million

XERS – Xeris Pharmaceuticals Inc.
Glucagon Rescue Pen
Severe hypoglycemia

Approved FDA Approval announced September 10, 2019.
$236.2 million