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.


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

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


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%.


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 presentation at ASCO Genitourinary Cancers Symposium February 13, 2020 noted median overall survival (95% CI) of 33.7 months.
$48.8 million

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

Phase 2 Phase 2 interim data due around 2Q 2020.
$16.9 million

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

Phase 1/2 Phase 1 interim data to be presented 1Q 2020.
$16.9 million

DRRX – DURECT Corporation
Nonalcoholic steatohepatitis (NASH)

Phase 1b Phase 2 trial to be completed 1H 2020 with data due following completion of trial.
$340.5 million

DRRX – DURECT Corporation

Phase 2a Phase 2 top-line data did not meet endpoints - January 2, 2020.
$340.5 million

JNJ – Johnson & Johnson
Plaque psoriasis - pediatric

sNDA Filing sBLA filing announced October 7, 2019.
$354.3 billion

KOD – Kodiak Sciences Inc
Diabetic macular edema (DME)

Phase 1b Phase 1b data February 8, 2020 - 55% of wet AMD eyes extended to six months and 64% of DME eyes extended to six months or longer before first retreatment.
$2.8 billion

MITO – Stealth BioTherapeutics Corp.
Rare neurodegenerative diseases

Phase 1 Phase 1 trial initiation announced January 21, 2020.
$64.8 million

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

Phase 2 Phase 2 preliminary top-line results from the 24-week cohort (Cohort 4) met primary endpoint - February 24, 2020.
$1.2 billion

NKTR – Nektar Therapeutics
Atopic Dermatitis

Phase 1b Phase 1b trial initiation announced October 7, 2019.
$3.7 billion

PFNX – Pfenex Inc.

Approved FDA approval announced October 7, 2019.
$367.4 million

PRQR – ProQR Therapeutics N.V.
Sepofarsen (QR-110 ) ILLUMINATE
Leber's Congenital Amaurosis (LCA) - Genetic blindness

Phase 2/3 Phase 2/3 data due 1H 2021.
$352.7 million

PTCT – PTC Therapeutics Inc.
AADC deficiency

BLA Filing BLA filing due 2Q 2020.
$3.4 billion

XENT – Intersect ENT Inc.
Sinus Balloon

Phase 3 Data released October 7, 2019 did not meet primary endpoint.
$751.9 million