BPC January 03 update

PDUFA and Advisory Committee meetings to watch for the first quarter

Weekly watchlist

A happy new year to all readers of BioPharmCatalyst.

Over the next three weeks we will be listing key biotech events to watch during the first quarter of 2020. We start this week looking at regulatory events (Advisory Committee meetings and PDUFA dates), before noting key Phase 1/2 and Phase 3 readouts over the following two weeks.

First, let’s review the week that was, which was unsurprisingly quiet in terms of newsflow due to the holiday season.

Sol-Gel Technologies, Ltd. (NASDAQ:SLGL) shares closed the week up 70% to $15.65 following news its two pivotal Phase 3 clinical trials for Twyneo demonstrated statistically significant improvement on all co-primary endpoints in the treatment of patients with acne.

Wave Life Sciences Ltd. (NASDAQ:WVE) shares closed the week down 52% to $7.60 following the release of top-line data from its Phase 1b/2a PRECISION-HD2 trial evaluating WVE-120102, for the treatment of Huntington's disease (HD). While a statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein was observed, the decrease was much lower than previously released data from Ionis and its Huntington’s disease candidate, IONIS-HTTRx (RG6042).

Savara Inc. (Nasdaq: SVRA) announced that the FDA granted Breakthrough Therapy designation for Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). The Breakthrough Therapy designation is based on data from IMPALA, a Phase 3 trial evaluating Molgradex for the treatment of aPAP, which failed to meet its primary endpoint in June last year. Shares closed the week up 133% to $4.07.

Novan, Inc. (NASDAQ:NOVN) shares closed Friday down 73% to $0.84 following news its Phase 3 B-SIMPLE program of SB206 for the treatment of molluscum contagiosum did not meet the primary endpoint in either of the two trials.

Durect Corporation (Nasdaq: DRRX) announced its Phase 2a clinical trial of DUR-928 in patients with mild to moderate plaque psoriasis did not demonstrate a benefit over vehicle, missing primary and secondary endpoints. Shares closed Thursday down 31% to $2.62.

Incyte Corporation (NASDAQ:INCY) shares closed Friday down 9% to $77.90 following its announcement that its Phase 3 GRAVITAS-301 trial evaluating itacitinib in combination with corticosteroids in patients with treatment-naïve acute graft-versus-host disease (GVHD), did not meet the primary endpoint of improving overall response rate (ORR) compared to placebo.

Drug Stage Catalyst Market Cap

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.
$215 million

AIMT – Aimmune Therapeutics Inc.
AR101
Peanut Allergy

PDUFA Advisory Committee meeting September 13, 2019 voted in favor of supporting approval. PDUFA date late-January 2020.
$2.1 billion

BMY – Bristol-Myers Squibb Company
Ozanimod - RADIANCE
Relapsing Multiple Sclerosis

PDUFA PDUFA date March 25, 2020.
$156.8 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285
PDGFRA exon 18 mutant Gastrointestinal stromal tumors (GIST)

Approved FDA Approval announced January 9, 2020.
$3.3 billion

DRRX – DURECT Corporation
POSIMIR
Post-operative pain relief

PDUFA CRL Feb 12 2014. Advisory Committee meeting voted 6-6 January 16, 2020 regarding approval. PDUFA date of December 27, 2019 was not met due to the meeting. No guidance announced regarding new timeline.
$404 million

EPZM – Epizyme Inc.
Tazemetostat
Epithelioid Sarcoma

Approved FDA approval announced January 23, 2020.
$2.6 billion

ESPR – Esperion Therapeutics Inc.
Bempedoic acid
Hyperlipidemia whose LDL-C is not adequately controlled with low- and moderate-dose statins

PDUFA PDUFA date February 21, 2020.
$1.4 billion

HRTX – Heron Therapeutics Inc.
HTX-011
Post operative pain

PDUFA PDUFA date March 26, 2020.
$2.1 billion

IGXT – Intelgenx Techs Corp
RIZAPORT (RHB-103)
Migraine

PDUFA PDUFA date March 26, 2020.
$44 million

IPCIF – Intellipharmaceutics International Inc - Ordinary Shares
Aximris XR (Oxycodone Hydrochloride extended release)
Pain relief

PDUFA Advisory Committee Meeting January 15, 2020 voted 24-2 against recommending approval.
$4.4 million

NKTR – Nektar Therapeutics
NKTR-181
Lower back pain

PDUFA FDA Advisory Committee voted 27-0 against recommending approval - January 14, 2020.
$3.8 billion

XXII – 22nd Century Group Inc.
VLN cigarettes
MRTP application

NDA Filing FDA Tobacco Products Scientific Advisory Committee (TPSAC) to meet February 14, 2020, regarding MRTP application. IGNORE the "NDA filing" stage. This is used as a placeholder by BioPharmCatalyst as no MRTP stage is available in our system.
$143.8 million