BPC January 17 update

Phase 1 and 2 events to watch for the first quarter of 2020; Biotech week in Review

Weekly watchlist

Last week we previewed key Phase 3 data readouts slated for release during the first quarter of 2020. This week our focus turns to Phase 1 and 2 data that is scheduled to be released this quarter. First, let's review price-moving events from the week that was.

The annual J.P.Morgan Healthcare Conference was held over the past week. Notable movers who presented at the conference included:

Adaptimmune Therapeutics plc (ADAP): $4.52; +240%.

NantKwest, Inc. (NK): $7.35; +106%.

Momenta Pharmaceuticals, Inc. (MNTA): $30.60; +61%.

Nektar Therapeutics (NASDAQ:NKTR) shares closed the week down 16% to $22.48 following news a FDA Advisory Committee voted 27-0 against recommending approval of oxycodegol for the management of chronic low back pain in adults.

Durect Corporation (NASDAQ: DRRX) also appeared before an Advisory Committee for its painkiller, Posimir, to manage post-surgical pain. However, unlike Nektar, the company received a split 6-6 vote, and shares reacted accordingly, closing down just 1% to $2.51 on Friday.

BioNTech (Nasdaq: BNTX) announced it will acquire Neon Therapeutics, Inc. (Nasdaq: NTGN) for approximately $67m. Shares of Neon closed the week up 43% to $1.69.

Acasti Pharma Inc. (NASDAQ: ACST) shares slumped to close the week down 64% to $0.78 on news its Phase 3 TRILOGY 1 trial of CaPre for the treatment of severe hypertriglyceridemia did not show a statistically significant decrease in triglycerides compared with placebo. Data from its second trial are due in February.

Aquestive Therapeutics, Inc. (NASDAQ:AQST) shares fell 38% to $4.10 following news the FDA denied the company’s Citizen's Petition requesting the FDA stay approval of a New Drug Application (NDA) for Valtoco (diazepam nasal spray) submitted by Neurelis, Inc for the treatment of epilepsy cluster seizures. The FDA noted it had approved Neurelis's NDA on January 10, 2020.

Dare Bioscience, Inc. (NASDAQ: DARE) shares closed the week up 98% to $1.66. The company announced a license agreement with Bayer, where it may commercialize its contraceptive product, Ovaprene. The deal includes up to $310m in milestone payments. The upfront payment was $1m.

Stemline Therapeutics, Inc. (Nasdaq: STML) noted disappointing net revenues for Elzonris of $11.8m for the quarter, down from $13.1m reported the previous quarter. Shares closed the week down 28% to $7.03.

Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) shares closed the week down 41% to $4.47 following mixed data released from its Phase 2 trial of ELX-02 in the treatment of patients with nonsense mutation-mediated nephropathic cystinosis.

Biotech Phase 1 and 2 catalysts for 1Q 2020:

Drug Stage Catalyst Market Cap

AGTC – Applied Genetic Technologies Corporation
AAV Gene Therapy
Achromatopsia

Phase 1/2 Phase 1/2 interim data from pediatric patients due 2H 2020.
$92.7 million

AKCA – Akcea Therapeutics Inc.
AKCEA-ANGPTL3-LRx
Non-alcoholic fatty liver disease (NAFLD), Hypertriglyceridemia and Type 2 Diabetes Mellitus

Phase 2b Phase 2b data met primary endpoint - January 28, 2020.
$1.7 billion

AKRO – Akero Therapeutics Inc.
AKR-001
Nonalcoholic steatohepatitis (NASH)

Phase 2a Phase 2a data due 1Q 2020 (MRI‐PDFF).
$689.7 million

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 2 top-line data due around the end of March 2020.
$6.5 billion

AXGT – Axovant Gene Therapies Ltd.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

Phase 1/2 Phase 1/2 6-month data from the first two patients in the second cohort due 1Q 2020.
$94.4 million

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Namodenoson (CF102)
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data due 1Q 2020.
$10.9 million

CRVS – Corvus Pharmaceuticals Inc.
CPI-444
Solid tumors - cancer

Phase 1b Phase 1b/2 updated data at the ASCO Genitourinary Cancers Symposium (GU) February 13, 2020 noted 1/35 partial responses.
$121.6 million

IMMP – Immutep Limited
Eftilagimod Alpha - Active Immunotherapy PAClitaxel (AIPAC)
Metastatic breast cancer (MBC)

Phase 2b Phase 2b PFS data due March 2020.
$98.3 million

INSM – Insmed Inc.
INS1007
Bronchiectasis

Phase 2 Phase 2 trial met primary endpoint - February 3, 2020.
$2.7 billion

KDMN – Kadmon Holdings Inc.
KD025 - ROCKstar (KD025-213)
Chronic graft-versus-host disease (cGVHD)

Phase 2 Phase 2 top-line data of primary analysis to be announced in 2Q 2020
$732.5 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.
$1.3 billion

OPK – Opko Health Inc.
RAYALDEE
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) - Stage 5

Phase 2 Phase 2 initial open label data due 1Q 2020.
$998.4 million

PRQR – ProQR Therapeutics N.V.
QR-421a - STELLAR
Usher Syndrome Type 2

Phase 1/2 Phase 1/2 interim data due 1Q 2020.
$417.7 million

XLRN – Acceleron Pharma Inc.
Sotatercept - PULSAR
Pulmonary arterial hypertension (PAH)

Phase 2 Phase 2 trial met primary endpoint - January 27, 2020.
$4.8 billion