BPC September 03 update

Ardelyx ARDX positive late-stage data +71%; ​Cidara CDTX partnership +40%

Price and Volume Movers

Cidara Therapeutics, Inc. (Nasdaq: CDTX) shares closed up 40% to $2.35 on news of a partnership with Mundipharma to develop rezafungin for the treatment and prevention of invasive fungal infections. Mundipharma will be granted rights to rezafungin outside the U.S. and Japan. Cidara will receive a $30m upfront payment and Mundipharma will make a $9m equity investment in Cidara. The company will also receive an additional $42m in near-term funding clinical trials and is eligible to receive milestone payments which potentially will value the deal at $568m if all milestones are met.

Ardelyx, Inc. (Nasdaq: ARDX) announced that it met the primary endpoint in its Phase 3 AMPLIFY trial of tenapanor in combination with phosphate binders in patients with chronic kidney disease (CKD) on dialysis whose hyperphosphatemia was not previously controlled with binders alone. The data showed a statistically significant (p=0.0004) reduction in serum phosphorus levels for patients treated with tenapanor and phosphate binders compared to phosphate binders alone. Shares closed up 71% to $5.82.

Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS) announced that its Phase 2 trial evaluating AR-105 for the treatment of ventilator-associated pneumonia (VAP) caused by gram-negative Pseudomonas aeruginosa, did not meet the primary endpoint. Shares closed down 8% to $10.80.

Roche (OTCQX: RHHBY) announced a further extension for the offering period of its pending acquisition of Spark Therapeutics, Inc. (NASDAQ: ONCE) to October 1, 2019.

Abeona Therapeutics Inc. (NASDAQ:ABEO) shares rallied to close up 91% to $2.94. The company announced that it has retained Jefferies LLC as its financial advisor to assist with the review of strategic alternatives, including potential partnership or sale/merger options.

Emergent BioSolutions Inc. (NYSE:EBS) announced a contract award by the U.S. Department of Health and Human Services (HHS) valued at approximately $2b over 10 years for the continued supply of ACAM2000, (Smallpox Vaccine, Live) into the U.S. Strategic National Stockpile (SNS). Shares closed up 14% to $50.14.

The Medicines Company (NASDAQ: MDCO) shares closed up 10% to $46.31 following the presentation of data from its Phase 3 ORION-11 trial of inclisiran, a treatment to reduce low-density lipoprotein cholesterol (LDL-C). For the primary endpoints, inclisiran delivered placebo-adjusted LDL-C reductions of 54% (p<0.0001) at day 510 and demonstrated time-averaged placebo-adjusted LDL-C reductions of 50% (p<0.0001) from days 90 through 540.

Cassava Sciences, Inc. (Nasdaq: SAVA) shares closed up 18% to $1.32. The company noted today that it will present a corporate and clinical overview at the H.C. Wainwright Global Investment Conference on September 9. Phase 2a data of PTI-125 in patients with Alzheimer’s disease are also due this quarter.

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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:

Kadmon Holdings, Inc. (KDMN): $2.46; +15%.

Ritter Pharmaceuticals, Inc. (RTTR): $1.14; +11%.

Dynavax Technologies Corporation (DVAX): $4.58; +11%.

Marinus Pharmaceuticals, Inc. (MRNS): $1.27; +10%.

DECLINERS:

Mirum Pharmaceuticals, Inc. (MIRM): $10.06; -21%.

Liquidia Technologies Inc. (NASDAQ: LQDA): $3.29; -19%.

Neoleukin Therapeutics, Inc. (NLTX): $2.80; -16%.

PhaseBio Pharmaceuticals, Inc. (PHAS): $7.38; -15%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGN – Allergan plc
Juvéderm VOLUMA
Mid-Face Injection Via Cannula

Approved FDA Approval announced September 3, 2019.
$54.5 billion

AKBA – Akebia Therapeutics Inc.
Vadadustat - PRO2TECT
Non-dialysis patients with anemia related to CKD (NDD-CKD)

Phase 3 Phase 3 data due mid-2020.
$537.3 million

APLS – Apellis Pharmaceuticals Inc.
APL-2 PRINCE
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3 Phase 3 commencement of dosing announced September 3, 2019.
$1.7 billion

ARDS – Aridis Pharmaceuticals Inc.
AR-105 (Aerucin)
Pseudomonas aeruginosa

Phase 2 Phase 2 trial did not met primary endpoint - September 3, 2019.
$67.7 million

ARDX – Ardelyx Inc.
Tenapanor - AMPLIFY
Hyperphosphatemia - adjunctive therapy to phosphate binders

Phase 3 Phase 3 trial met primary endpoint.
$297.7 million

ATOS – Atossa Genetics Inc.
Endoxifen
Healthy volunteers

Phase 1 Phase 1 data due within 30 days - noted September 3, 2019.
$19.1 million

BLUE – bluebird bio Inc.
Lenti-D - Starbeam
Childhood cerebral adrenoleukodystrophy (CCALD)

Phase 2/3 Phase 2/3 updated data September 18, 2019 noted 88% (N=15/17) continue to be alive and MFD-free.
$5.8 billion

CNCE – Concert Pharmaceuticals Inc.
CTP-543
Alopecia areata

Phase 2a Phase 2 data to be presented at EADV October 12, 2019 at 9:15am GMT.
$260.6 million

GNFT – GENFIT S.A.
Elafibranor
Primary biliary cholangitis (PBC)

Phase 3 Phase 3 trial to commence 2H 2019.
$696.5 million

INCY – Incyte Corporation
Pemigatinib (FIGHT-202)
Cholangiocarcinoma

Phase 2 NDA filing to be submitted 2H 2019. Phase 2 data to be presented at ESMO September 27, 2019, 9am ET.
$16.8 billion

INCY – Incyte Corporation
INCB001158
Solid tumors

Phase 1/2 Phase 1/2 data due at ESMO September 29, 2019.
$16.8 billion

QURE – uniQure N.V.
AMT-061 (HOPE-B)
Hemophilia B

Phase 3 Phase 3 enrolment to be completed September 2019 with top-line data due in 2020.
$1.8 billion

TPTX – Turning Point Therapeutics Inc.
Repotrectinib (TPX-0005) - TRIDENT-1
Non-small-cell lung cancer (NSCLC) and solid tumors

Phase 1/2 Phase 2 interim data due 2H 2020. Phase 1 updated data September 3, 2019 noted ORR 91% In TKI-naïve ROS1+ NSCLC patients.
$1.3 billion