BPC December 03 update

GBT +48% accelerated approval pathway; TSRO acquired by GSK +58%

Price and Volume Movers

Global Blood Therapeutics, Inc. (Nasdaq: GBT) shares rallied to close up 48% to $46.62 following news the FDA will allow an accelerated approval pathway for voxelotor for the treatment of sickle cell disease (SCD). GBT plans to request a pre-NDA meeting in 1Q 2019.

TESARO Inc (NASDAQ: TSRO) shares surged to close up 58% to $73.50 following news that it will be acquired by GlaxoSmithKline plc (NYSE: GSK) for approximately $5.1b (£4b). The transaction is expected to complete in 1Q 2019. 

argenx (Euronext & Nasdaq: ARGX) shares closed up 13% to $109.82 on news of a license agreement for cusatuzumab (ARGX-110) with J&J. Cusatuzumab is currently in development in a Phase 1/2 combination study with Vidaza in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). J&J will pay argenx $300m upfront and will purchase $200m of newly issued shares. Argenx will also be eligible to receive potentially up to $1.3b in milestone payments and double-digit royalties.

Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced positive topline results its Phase 3 clinical trial evaluating its Zydis orally dissolving tablet (ODT) formulation of rimegepant for the acute treatment of migraine.  The trial met both of its co-primary endpoints of pain freedom and freedom from most bothersome symptom (MBS) at 2 hours using a single dose. Shares closed up 25% to $42.62 on the data.

Sienna Biopharmaceuticals, Inc. (NASDAQ:SNNA) shares slid to close down 38% to $6.31 following news that it failed to reach the primary endpoint in its Phase 2b trial of SNA-120 for the treatment of mild-to-moderate psoriasis patients. The company expects to commence Phase 3 trials in 2H 2019.

Vanda Pharmaceuticals Inc. (Nasdaq:VNDA) announced that its Phase 2 trial of tradipitant in patients with idiopathic and diabetic gastroparesis, met the primary endpoint of the study of change in nausea score as measured by patient daily diaries (change of -1.2 for tradipitant versus -0.7 for placebo, p=0.0099). Shares closed up 26%.

Ocular Therapeutix, Inc. (NASDAQ: OCUL) announced the FDA has approved the resubmission of its New Drug Application (NDA) for Dextenza for the treatment of ocular pain following ophthalmic surgery. The company previously received two Complete Response Letters (CRLs) in 2016 and 2017. Shares closed down 14% to $5.73, a common trend for small cap stocks following approval.

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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:

vTv Therapeutics Inc. (VTVT): $1.99; +18%.

Apellis Pharmaceuticals, Inc. (APLS): $18.06; +18%.

Provention Bio, Inc. (PRVB): $2.70; +17%.

Bellerophon Therapeutics, Inc. (BLPH): $1.10; +16%.

Aeterna Zentaris Inc. (AEZS): $3.87; +15%.

DECLINERS:

Adial Pharmaceuticals, Inc. (ADIL): $2.83; -26%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $4.04; -22%.

Kodiak Sciences Inc. (KOD): $7.95; -14%.

Agios Pharmaceuticals, Inc. (AGIO): $56.81; -14%.

Arsanis, Inc. (ASNS): $3.38; -13%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AFMD – Affimed N.V.
AFM13 with Keytruda
Hodgkin Lymphoma - Cancer

Phase 1b Phase 1b data at ASH noted 46% CR; 88% ORR.
$212.9 million

BGNE – BeiGene Ltd.
Tislelizumab - BGB-A317
Relapsed or refractory classical Hodgkin lymphoma (cHL)

Phase 2 Phase 2 data at ASH 2018 noted ORR 86%; CR 61%.
$7.9 billion

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Zydis ODT formulation of rimegepant
Migraine

Phase 3 Phase 3 data released December 3, 2018 - primary endpoints met.
$1.5 billion

CELG – Celgene Corporation
Liso-cel (JCAR017) - TRANSCEND CLL-004
Chronic Lymphocytic Leukemia (CLL)

Phase 1 Phase 1 data at ASH noted 81% ORR; 43% CR.
$47.7 billion

GBT – Global Blood Therapeutics Inc.
GBT440
Sickle cell disease

Phase 3 Pre-NDA meeting scheduled for 1Q 2019. NDA to be filed under accelerated approval.
$2.2 billion

GILD – Gilead Sciences Inc.
Yescarta (axicabtagene ciloleucel)
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer

Phase 2 2-year data at ASH 2018 noted 39% of patients were in an ongoing response. OS not yet reached.
$84.8 billion

NVS – Novartis AG
AVXS-101
Spinal muscular atrophy (SMA) Type 1

PDUFA priority review PDUFA date under priority review May 2019. Exact date not released.
$201.9 billion

OCUL – Ocular Therapeutix Inc.
DEXTENZA
Ocular inflammation and pain following cataract surgery

Approved FDA approval announced December 3, 2018.
$219 million

PGNX – Progenics Pharmaceuticals Inc.
PyL
Prostate cancer

Phase 3 Phase 3 dosing of first patient announced December 3, 2018. Data due early 2020.
$388.9 million

RDHL – Redhill Biopharma Ltd.
RHB-105 - ERADICATE Hp 2
H. pylori

Phase 3 Phase 3 trial met primary endpoint. NDA filing 1H 2019.
$173.5 million

REGN – Regeneron Pharmaceuticals Inc.
REGN1979
B-cell non-Hodgkin lymphoma

Phase 1 Phase 1 data released at ASH 2018. 100% ORR; 80% CR (10 patients).
$41.3 billion

SNNA – Sienna Biopharmaceuticals Inc.
SNA-120
Pruritus associated with psoriasis

Phase 2b Phase 2b trial did not meet primary endpoint. Phase 3 trial to be initiated 2H 2019.
$73.6 million

VNDA – Vanda Pharmaceuticals Inc.
Tradipitant
Gastroparesis

Phase 2 Phase 2 primary endpoint met - December 3, 2018.
$1.4 billion