BPC October 11 update

14 small/mid-cap PDUFA and Advisory Committee Meeting dates through to December; Biotech week in review

Weekly watchlist

Over the past two weeks we have highlighted key upcoming Phase 1/2 and Phase 3 clinical readouts slated for release this quarter. We complete our preview this week, highlighting regulatory events for small and mid-cap biotech stocks. Such events cover both Advisory Committee Meeting and PDUFA dates.

First, let’s review the week that was with a summary of price-moving events.

Pfenex Inc. (NYSE American: PFNX) announced Monday that the FDA approved the New Drug Application (NDA) for PF708 for the treatment of osteoporosis. Shares closed the week up 19% to $8.88.

Akcea Therapeutics, Inc. (NASDAQ: AKCA) shares closed the week up 31% to $19.75 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 its parent company, Ionis, will each receive $125m upfront, and will also be eligible to receive milestone payments of up to $1.3b.

Clinuvel Pharmaceuticals Limited (OTC: CLVLY) received FDA approval Tuesday for Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria. Shares closed the week up 37% to $24.80.

Ra Pharmaceuticals Inc. (NASDAQ: RARX, Ra Pharma) shares surged Thursday to close up 101% to $45.64 on news that it is set to be acquired by UCB for $48 per share, equivalent to approximately $2.1b.

OncoSec (NASDAQ: ONCS) shares closed Thursday up 35% to $2.21 following its announcement it will receive a $30m investment from China Grand Pharmaceutical (CGP) and its affiliate Sirtex at $2.50 per share, for a 53% share of the company and an option to acquire the remaining shares.

Reata Pharmaceuticals, Inc. (NASDAQ:RETA) announced it is reacquiring rights to bardoxolone methyl (bardoxolone) and omaveloxolone from Abbvie. AbbVie will receive a total of $330m in cash. Reata will make an upfront payment of $75m in 2019, with the remainder payable in 2020-21. Shares closed the week up 19% to $95.81.

Aclaris Therapeutics, Inc. (Nasdaq: ACRS) announced Thursday it has divested Rhofade, a topical treatment of persistent facial erythema (redness) associated with rosacea, to EPI Health. Aclaris will receive $35m upfront and potential sales milestone payments of up to $20m. Shares closed Friday up 19% to $1.42.

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14 small/mid-cap PDUFA and Advisory Committee Meeting dates through to December:

Drug Stage Catalyst Market Cap

ADMP – Adamis Pharmaceuticals Corporation
Higher Dose Naloxone Injection
Opioid overdose

PDUFA PDUFA date October 31, 2019. Company noted as of November 4, 2019 no communication has been received from the FDA.
$65.7 million

AGRX – Agile Therapeutics Inc.
Twirla
Contraceptive patch

PDUFA CRLs issued 2013 and 2017. New PDUFA extended by three months to February 16, 2020. Advisory Committee Meeting October 30, 2019 voted 14 to 1 in favor.
$103.2 million

AMRN – Amarin Corporation plc
Vascepa
High Triglycerides With Mixed Dyslipidemia

PDUFA priority review PDUFA date December 28, 2019. Advisory Committee meeting November 14, 2019 voted 16-0 in favor.
$7.4 billion

AQST – Aquestive Therapeutics Inc.
Exservan (riluzole) Oral Film
Amyotrophic Lateral Sclerosis

PDUFA PDUFA date November 30, 2019.
$155.3 million

ASRT – Assertio Therapeutics Inc.
Cosyntropin depot
Adrenocortical insufficiency screening

CRL CRL announced October 21, 2019.
$60 million

AVDL – Avadel Pharmaceuticals plc
AV001
Hospital product

PDUFA PDUFA date extended by three months to December 15, 2019.
$216.8 million

CORV – Correvio Pharma Corp.
Brinavess
Atrial fibrillation (AF)

PDUFA PDUFA date December 24, 2019. FDA Advisory Committee meeting December 10, 2019.
$111.1 million

ETON – Eton Pharmaceuticals Inc.
ET-202
Injectable hospital product

Approved FDA Approval announced October 22, 2019.
$107.5 million

FOMX – Foamix Pharmaceuticals Ltd.
FMX101
Acne

Approved FDA Approval announced October 18, 2019.
$202.5 million

ITCI – Intra-Cellular Therapies Inc.
Lumateperone
Schizophrenia

PDUFA PDUFA date extended by three months to December 27, 2019. No advisory committee meeting expected.
$523.9 million

LPCN – Lipocine Inc.
TLANDO - LPCN 1021
Men with low testosterone (Low T)

CRL CRL announced November 11, 2019.
$10.1 million

MLNT – Melinta Therapeutics Inc.
Baxdela
Community-acquired bacterial pneumonia (CABP)

Approved FDA Approval announced October 24, 2019.
$18.7 million

RDHL – Redhill Biopharma Ltd.
Talicia RHB-105
H. pylori

Approved FDA Approval announced November 4, 2019.
$203.2 million

XLRN – Acceleron Pharma Inc.
Luspatercept
Beta-thalassemia

Approved FDA Approval announced November 8, 2019.
$2.3 billion