BPC November 20 update

PDUFA dates to round out 2020; Biotech week in review

Weekly watchlist

With a shortened holiday week ahead to round out November, we now turn our focus to events slated for the final month of the year. This week we look at regulatory events and will follow up the weekend following Thanksgiving with clinical data readouts to watch. There will be no weekly review over the Thanksgiving holiday weekend. First, let's review the week that was with notable healthcare news.

COVID-19 VACCINE:

Pfizer Inc. (NYSE:PFE) and BioNTech SE (NASDAQ:BNTX) announced the submission to the FDA for Emergency Use Authorization (EUA) of their COVID-19 vaccine candidate, BNT162b2, which will potentially enable use of the vaccine in the U.S. by mid/late-December 2020. Final efficacy analysis from its Phase 3 trial showed a vaccine efficacy rate of 95%. The FDA Vaccines and Related Biological Products Advisory Committee (VRBPAC) will meet on December 10 to discuss the EUA.

Moderna, Inc. (NASDAQ:MRNA) shares closed the week up 9% to $97.61 on news data from its Phase 3 trial of mRNA-1273, its vaccine candidate against COVID-19, exhibited an efficacy rate of 94.5%. Moderna will also submit a EUA in the coming weeks.

REGULATORY:

Eiger BioPharmaceuticals, Inc. (NASDAQ:EIGR) received approval from the FDA for Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome. Shares closed the Friday's after hours session up 12% to $11.25.

Bristol Myers Squibb (NYSE:BMY) announced the review of its Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel) for the treatment of adults with relapsed or refractory (R/R) large B-cell lymphoma, has been delayed, with the FDA unable to conduct an inspection of a manufacturing facility in Texas due to travel restrictions related to the COVID-19 pandemic. A revised PDUFA date was not announced.

Adamis Pharmaceuticals Corporation (NASDAQ:ADMP) announced that it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for ZIMHI high dose naloxone injection product for the treatment of opioid overdose. Shares closed the week down 40% to $0.41.

CLINICAL DATA:

RAPT Therapeutics, Inc. (Nasdaq: RAPT) shares closed Monday down 46% to $16.41 on news its trial of FLX475 in various cancers, showed across the three Phase 2 monotherapy cohorts, just 1 out of 22 patients exhibited a partial response.

uniQure N.V. (NASDAQ: QURE) announced its Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B, met the primary endpoint with Factor IX (FIX) activity increasing after dosing from ≤2% to a mean of 37.2 percent at 26 weeks. Shares closed the week up 7% to $46.11.

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) shares slid to close Tuesday down 66% to $4.02 on news its Phase 3 trial evaluating NurOwn as a treatment for Amyotrophic lateral sclerosis (ALS) did not meet the primary endpoint of the trial.

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PDUFA dates and one COVID-19 FDA Advisory Committee Meeting to round out 2020:

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran
Primary Hyperoxaluria Type 1 (PH1)

Approved FDA approval announced November 24, 2020.
$15.1 billion

ATNX – Athenex Inc.
Tirbanibulin ointment
Actinic keratosis

PDUFA PDUFA date December 30, 2020.
$1.3 billion

BCRX – BioCryst Pharmaceuticals Inc.
Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients

PDUFA PDUFA date December 3, 2020
$902.3 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

PDUFA PDUFA date December 20, 2020.
$3.8 billion

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

PDUFA PDUFA date originally set for August 28, 2020 delayed. New date on or about the week of November 30, 2020.
$105.1 million

LQDA – Liquidia Corporation
LIQ861
Pulmonary arterial hypertension

CRL CRL announced November 25, 2020.
$120.8 million

MGNX – MacroGenics Inc.
Margetuximab
Metastatic breast cancer

PDUFA PDUFA date December 18, 2020.
$1.3 billion

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

PDUFA priority review PDUFA date under priority review December 20, 2020. Phase 3 data meet primary endpoint - November 19, 2019. Castration Resistance Free Survival data released September 29, 2020 did not achieve statistical superiority (p = 0.84).
$2.2 billion

OSMT – Osmotica Pharmaceuticals plc
Ontinua ER (arbaclofen)
Spasticity resulting from multiple sclerosis

PDUFA PDUFA date December 29, 2020.
$383 million

PFE – Pfizer Inc.
BNT162b2
COVID-19 vaccine

Phase 3 Phase 3 final efficacy analysis noted vaccine was 95% effective in preventing COVID-19. EUA filing announced November 20, 2020. Advisory Committee meeting December 10, 2020.
$212.9 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity / Leptin Receptor Deficiency Obesity

Approved FDA approval announced November 27, 2020.
$1.4 billion

SCPH – scPharmaceuticals Inc.
Furoscix
Heart failure

PDUFA PDUFA date December 30, 2020.
$251.9 million

VNDA – Vanda Pharmaceuticals Inc.
HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

PDUFA priority review PDUFA date under priority review December 1, 2020.
$668.3 million

YMAB – Y-mAbs Therapeutics Inc.
Naxitamab
Neuroblastoma

Approved FDA approval announced November 25, 2020.
$2.1 billion