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 due 2H 2020.
$150.5 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.
$1.3 billion

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

Phase 2a Phase 2a dose groups all met primary endpoint - March 31, 2020. Further data released June 30, 2020 noted 48% of patients achieved at least a one-stage improvement in fibrosis without worsening of NAFLD activity score (NAS) and 28% achieved at least a two-stage improvement in fibrosis.
$1.1 billion

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 2 six-month data from the open-label extension portion due 3Q 2020. Phase 3 trial to commence 4Q 2020.
$6.6 billion

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

Phase 1/2 Phase 1/2 6-month data from second dose cohort due 4Q 2020
$116.2 million

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

Phase 2 Phase 2 endpoints met - April 7, 2020.
$25.2 million

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

Phase 1b Phase 1b/2 initial data from the RCC cohort at ASCO May 29, 2020. AdenoSig positive group -ORR of 17%. AdenoSig negative group - no partial responses.
$123 million

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

Phase 2b Phase 2b overall survival data due late-2020. 63% of patients were progression-free at 6 months compared with 54% on placebo (HR 0.93)
$42.8 million

INSM – Insmed Inc.
Brensocatib (INS1007)
Bronchiectasis

Phase 2 Phase 2 full data June 24, 2020 noted the risk of exacerbation was reduced by 42% low dose vs placebo (HR 0.58, p=0.029) and by 38% for high dose (HR 0.62, p=0.046). Phase 3 trial to be initiated 2H 2020.
$2.8 billion

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

NDA Filing Phase 2 top-line data noted statistically significant Overall Response Rates (ORRs) of 73% with 200 mg once daily. NDA filing 4Q 2020.
$673.7 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 noted trial was well tolerated - March 25, 2020. Final top-line data 1Q 2021.
$2.7 billion

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

Phase 1/2 Phase 1/2 interim data March 31, 2020. 2/8 patients demonstrated benefit across multiple concordant outcome measures.
$249.3 million

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

Phase 2 Phase 2 trial met primary endpoint - January 27, 2020. Data presented at American Thoracic Society (ATS) June 24, 2020 noted PVR reductions of 21-34% compared with 2% for placebo.
$5.9 billion