BPC May 24 update

Novartis NVS receives green light from FDA for its spinal muscular atrophy gene therapy

Price and Volume Movers

Novartis (NYSE: NVS) shares closed up 4% to $87.52 following its announcement the FDA approved Zolgensma, the first gene therapy approved for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA). Novartis will price Zolgensma at $2.125m, or an annualized cost of $425,000 per year for five years. Regenxbio Inc. (Nasdaq: RGNX), which earned a $3.5m milestone payment from the approval, saw its shares close up 7% to $45.64. On the other side, Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), which out-licensed its SMA treatment, Spinraza to Biogen, saw its shares close down 4% to $64.72.

Novartis (NYSE: NVS) also announced the FDA approved Piqray (alpelisib/BYL719) in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or metastatic breast cancer.

Incyte Corporation (Nasdaq:INCY) announced the FDA approved Jakafi (ruxolitinib) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older. Jakafi is the first FDA-approved treatment for this indication.

Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) announced a registered direct offering 1,317,060 shares at a price of $4.77 per share, for aggregate gross proceeds of approximately $6.28m. 1,317,060 warrants were also attached to the offering. Shares closed down 23% to $3.66.

Alpine Immune Sciences, Inc. (NASDAQ: ALPN) shares closed down 17% to $4.77 on news it received notice from Gilead of the termination of a license agreement developing protein-based immunotherapies for cancer.

-

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

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

ADVANCERS:

Outlook Therapeutics, Inc. (OTLK): $2.79; +42%.

Immunic, Inc. (IMUX): $11.09; +21%.

Pieris Pharmaceuticals, Inc. (PIRS): $4.41; +18%.

Osmotica Pharmaceuticals plc (OSMT): $2.63; +11%.

LogicBio Therapeutics, Inc. (LOGC): $16.21; +9%.

DECLINERS:

Immuron Limited (IMRN): $3.66; -27%.

Therapix Biosciences Ltd. (TRPX): $2.50; -24%.

Entera Bio Ltd. (ENTX): $2.93; -21%.

DelMar Pharmaceuticals, Inc. (DMPI): $1.86; -17%.

InflaRx N.V. (IFRX): $35.11; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

INCY – Incyte Corporation
Ruxolitinib
Graft versus host disease

Approved FDA approval announced May 24, 2019.
$17.3 billion

JAGX – Jaguar Health Inc.
Mytesi (crofelemer) HALT-D
Cancer related diarrhea (CRD)

Phase 2 Phase 2 interim data due 1H 2019. Timeline missed. Assume 3Q 2019 release.
$7.1 million

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

Approved FDA Approval announced May 24, 2019.
$204.6 billion

NVS – Novartis AG
BYL719 + fulvestrant
HR + Metastatic breast cancer (MBC)

Approved FDA Approval announced May 24, 2019.
$204.6 billion

PFE – Pfizer Inc.
LYRICA (pregabalin)
Primary generalized tonic-clonic (PGTC) seizures - epilepsy

Phase 3 Phase 3 data released May 24, 2019 did not meet primary endpoint.
$236.6 billion