BPC October 07 update

​Akcea AKCA shares rally 31% on license deal with Pfizer PFE; Pfenex PFNX FDA Approval +16%

Price and Volume Movers

Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc (NASDAQ: IONS), saw its shares climb 31% to $19.87 following news of a license agreement with Pfizer Inc. (NYSE:PFE) for AKCEA-ANGPTL3-LRx, a therapy being developed to treat patients with certain cardiovascular and metabolic diseases. Akcea, and Ionis will each receive $125m upfront, and will also be eligible to receive milestone payments of up to $1.3b and double-digit royalties on annual worldwide net sales.

Pfenex Inc. (NYSE American: PFNX) announced that the FDA approved the New Drug Application (NDA) for PF708 for the treatment of osteoporosis in certain patients at high risk for fracture. Shares closed up 16% to $8.62.

Sorrento Therapeutics, Inc. (Nasdaq: SRNE) shares closed down 26% to $1.78 following news of an intra-day direct offering of 10,869,566 shares and warrants to purchase up to an additional 10,869,566 shares, at a combined purchase price of $2.30 per share and related warrant, for gross proceeds of approximately $25m.

NGM Biopharmaceuticals, Inc. (Nasdaq: NGM) shares closed down 20% to $10.78 following the release of interim data from Cohort 4 of its Phase 2 trial of aldafermin in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH). Falling short of investor expectations, the data noted a statistically significant change in the absolute liver fat content (LFC) of -7.9% compared to -2.0% in the placebo arm (p<0.05). The relative reduction was -39.6% compared with -5.9% for placebo (p<0.05). Top-line data of the full cohort are due in 1Q 2020.

Ritter Pharmaceuticals, Inc. (NASDAQ:RTTR) announced that it has engaged A.G.P./Alliance Global Partners as financial advisor to explore and evaluate strategic alternatives. Shares closed up 50% to $0.38.

Intersect ENT, Inc. (NASDAQ:XENT) announced that data from its ASCEND investigational drug-coated sinus balloon did not meet its primary endpoint, an intra-patient comparison of frontal sinus patency grade at day 30. Shares closed down 4% to $16.36.

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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:

Xenetic Biosciences, Inc. (XBIO): $1.30; +22%.

Jounce Therapeutics, Inc. (JNCE): $3.20; +14%.

Assertio Therapeutics, Inc. (ASRT): $1.33; +9%.

Liquidia Technologies Inc. (LQDA): $3.96; +8%.

Galectin Therapeutics, Inc. (GALT): $4.15; +8%.

DECLINERS:

DiaMedica Therapeutics Inc. (DMAC): $2.05; -11%.

Capricor Therapeutics, Inc. (CAPR): $3.01; -10%.

Adaptimmune Therapeutics plc (ADAP): $1.10; -10%.

Allakos Inc. (ALLK): $70.10; -9%.

Sarepta Therapeutics, Inc. (SRPT): $80.96; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADXS – Advaxis Inc.
ADXS-PSA and Keytruda - KEYNOTE-046
Castrate-resistant prostate cancer

Phase 1/2 Phase 1 updated data noted median overall survival of 33.6 months - October 7, 2019.
$8 million

AIM – AIM ImmunoTech Inc.
Ampligen, Intron A, and celecoxib
Colorectal cancer

Phase 2 Phase 2 interim data due around 2Q 2020.
$2.7 million

AIM – AIM ImmunoTech Inc.
Ampligen plus Intron A
Ovarian cancer

Phase 1/2 Phase 1 interim data likely around January/February 2020 timeframe.
$2.7 million

DRRX – DURECT Corporation
DUR-928
Nonalcoholic steatohepatitis (NASH)

Phase 1b Phase 1b trial to be completed 1H 2020 with data due following completion of trial.
$355.1 million

DRRX – DURECT Corporation
DUR-928
Psoriasis

Phase 2 Phase 2 top-line data due 4Q 2019.
$355.1 million

JNJ – Johnson & Johnson
STELARA (USTEKINUMAB)
Plaque psoriasis - pediatric

sNDA Filing sBLA filing announced October 7, 2019.
$350.6 billion

KOD – Kodiak Sciences Inc
KSI-301
Diabetic macular edema (DME)

Phase 1b Phase 1b data to be presented at the Retina Subspecialty Day October 11, 2019.
$696.2 million

MITO – Stealth BioTherapeutics Corp.
SBT-272
Rare neurodegenerative diseases

Phase 1 Phase 1 trial to be initiated by end of 2019.
$238 million

NGM – NGM Biopharmaceuticals Inc.
NGM282
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 cohort 4 data noted statistically significant change in liver fat content (LFC) of -7.9% compared to -2.0% increase for placebo. Top-line data from full cohort due 1Q 2020.
$658.4 million

NKTR – Nektar Therapeutics
NKTR-358
Atopic Dermatitis

Phase 1b Phase 1b trial initiation announced October 7, 2019.
$3 billion

PFNX – Pfenex Inc.
PF708
Osteoporosis

Approved FDA approval announced October 7, 2019.
$281.2 million

PRQR – ProQR Therapeutics N.V.
Sepofarsen
Leber's Congenital Amaurosis (LCA) - Genetic blindness

Phase 2/3 Phase 2/3 data due 1H 2021. Phase 1/2 data presented October 10, 2019 noted statistically significant improvement in vision.
$242 million

PTCT – PTC Therapeutics Inc.
GT-AADC
AADC deficiency

BLA Filing BLA filing due 4Q 2019.
$2.2 billion

XENT – Intersect ENT Inc.
ASCEND
Sinus Balloon

Phase 3 Data released October 7, 2019 did not meet primary endpoint.
$545.7 million