BPC March 07 update

Updated biotech stock events to watch - March; Biotech week in review

Weekly watchlist

The latest week saw much price-moving news, with clinical readouts and regulatory news followed by later in the week, speculative price action among nano-cap stocks, initially triggered by moves from Bio-Path Holdings Inc (BPTH: NASDAQ), which closed the week up 172%. Other nano-caps such as Seelos Therapeutics, Inc. (SEEL) also joined in for the ride.

ArQule, Inc. (NASDAQ: ARQL) shares closed Thursday up 67% to $5.44 following the release of data from its Phase 1 trial of ARQ 531 in B-Cell Malignancies. ARQ 531 is a Bruton’s tyrosine kinase (BTK) inhibitor designed to inhibit both wild type and C481S-mutant BTK. The data on one patient with a C481S resistance mutation noted an 88% tumor burden reduction after the first scan.

Ascendis Pharma A/S (Nasdaq: ASND) announced Monday that it met the primary endpoint in its Phase 3 theiGHt Trial, of once-weekly TransCon Growth Hormone (hGH) compared to a daily growth hormone (Genotropin) in children with pediatric growth hormone deficiency (GHD). Shares closed the week up 74% to $120.61.

Nightstar Therapeutics plc (NASDAQ: NITE) shares surged to close Monday up 66% to $25.18 on news that it will be acquired by Biogen Inc for $25.50 per share, or around $800m in cash.

Evoke Pharma, Inc. (NASDAQ: EVOK) shares closed the week down 58% to $1.27 following receipt of a multi-disciplinary review (DR) letter from the FDA regarding its New Drug Application (NDA) for Gimoti. The letter described chemistry, clinical and clinical pharmacology concerns. The PDUFA date of April 1, 2019 remains. However, given this week’s events an approval decision is unlikely.

AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) shares closed Friday down 18% to $11.29 following news that it did not meet the co-primary endpoints in its Phase 3b Prolong trial evaluating Makena for the prevention of pre-term birth. The confirmatory trial was conducted as part of its accelerated approval process after receiving approval from the FDA in February 2011. The company noted a change in demographics as a possible cause for the trial failure and plans to conduct a sub-group analyses of the data.

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Biotech Stock earnings snapshot for next week (10):

M: DRNA ZFGN

T: NERV BLCM VSTM

W: TRVN MOR

T: PGNX AXSM RMTI

This week’s biotech stock watch list continues to focus on biotech events slated for March. We have removed a couple that saw readouts earlier in the week and have added other stocks to watch for a total of 15 events.

Drug Stage Catalyst Market Cap

AERI – Aerie Pharmaceuticals Inc.
Roclatan
Glaucoma

Approved FDA Approval announced March 12, 2019.
$705.4 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - INVIGORATE
Allergic conjunctivitis

Phase 3 Phase 3 data due 1H 2021.
$138.2 million

ARPO – Aerpio Pharmaceuticals Inc.
AKB-9778 - TIME 2B
Non-Proliferative Diabetic Retinopathy

Phase 2 Phase 2 data released March 18, 2019 did not meet primary endpoint.
$47.9 million

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

Phase 3 Phase 3 data did not meet primary endpoint. Secondary endpoints met. Second Phase 3 trial to commence 3Q 2020.
$2.8 billion

BXRX – Baudax Bio Inc.
Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery

Approved FDA Approval announced February 20, 2020.
$70.6 million

GLPG – Galapagos NV
Filgotinib
Rheumatoid arthritis (RA)

PDUFA NDA filing announced December 19, 2019. PDUFA estimate under priority review August 19, 2020.
$12.9 billion

LLY – Eli Lilly and Company
Lebrikizumab
Atopic Dermatitis

Phase 3 Phase 3 trial initiation announced October 9, 2019. Top-line data due 1H 2021.
$145.9 billion

LXRX – Lexicon Pharmaceuticals Inc.
Sotagliflozin
Type 1 Diabetes

CRL CRL issued March 22, 2019. Appeal rejected by FDA - December 2, 2019.
$202.2 million

NBIX – Neurocrine Biosciences Inc.
Crinecerfont (NBI-74788) - (ENDO)
Congenital Adrenal Hyperplasia (CAH) - adults

Phase 2 Phase 2 full data to be presented at ENDO Online 2020 meeting June 08, 2020.
$11.1 billion

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 negative data released December 17, 2019. Mean changes in ppFEV1 were not statistically significant in the heterozygous population. Phase 3 trial to be initiated in 2020.
$72 million

SAGE – Sage Therapeutics Inc.
Brexanolone - SAGE-547 (202C)
Postpartum Depression - moderate

Approved FDA approval announced March 19, 2018.
$1.9 billion

SGEN – Seattle Genetics Inc.
Enfortumab vedotin
Urothelial cancer

Approved FDA Approval announced December 18, 2019.
$26.8 billion

UROV – Urovant Sciences Ltd.
Vibegron
Overactive bladder (OAB)

PDUFA PDUFA date December 26, 2020.
$356.2 million

UTHR – United Therapeutics Corporation
Esuberaprost - BEAT
Pulmonary arterial hypertension (PAH)

Phase 3 Phase 3 data released April 8, 2019 did not meet primary endpoint.
$5.5 billion