BPC June 04 update

Binary event of the year - BIIB, potential sympathy plays + other key catalysts for June

Weekly watchlist

All eyes next week will be on the expected FDA decision regarding the regulatory application for aducanumab in patients with Alzheimer’s disease. The decision is seen as the binary event of the year, with potential sympathy moves by other companies developing drugs for Alzheimer’s, while the broader biotech market is likely to react to the outcome. The decision by the FDA is likely Monday.

We list below stocks which might move on sympathy plays depending on the outcome of the FDA decision, while also including THREE eagerly awaited clinical data readouts from Sage (SAGE), Vertex (VRTX) and Novavax (NVAX).

First, let’s review the week that was with price-moving and other notable events.

REGULATORY:

Liminal BioSciences Inc. (Nasdaq: LMNL) announced late-Friday the FDA approved Ryplazim (plasminogen) for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenia). Ryplazim becomes the first FDA approved therapy for this rare genetic disorder. Shares were trading up after hours Friday 55% to $6.27.

Scynexis, Inc. (NASDAQ: SCYX) announced that the FDA approved BREXAFEMME (ibrexafungerp tablets), for oral use in patients with vulvovaginal candidiasis (VVC), also known as vaginal yeast infection. Shares sold off on the news following strong gains through May, closing down 16% to $8.04.

Paratek Pharmaceuticals, Inc. (NASDAQ:PRTK) shares closed the week up 17% to $9.60 following news the FDA approved its supplemental New Drug Application (sNDA) for the oral-only dosing regimen of NUZYRA for the treatment of adults with Community-Acquired Bacterial Pneumonia (CABP).

Other companies to receive FDA approval included Alkermes plc (Nasdaq: ALKS), Chimerix (NASDAQ:CMRX) and BridgeBio Pharma, Inc. (Nasdaq: BBIO)

CLINICAL DATA:

Protalix BioTherapeutics (NYSE:PLX) shares closed the week down 30% to $2.14. The company reported data from their Phase 3 BALANCE trial of pegunigalsidase alfa (PRX–102) for the proposed treatment of Fabry disease. The initial data showed changes in eGFR when compared with agalsidase beta (Fabrazyme), were below the non-inferiority margin pre-specified for the interim analysis in the intention to treat population. eGFR, or Estimated glomerular filtration rate, measures the level of kidney function. The company also noted that one patient discontinued due a related treatment emergent adverse event.

OTHER:

MorphoSys AG (NASDAQ:MOR) announced it will acquire Constellation Pharmaceuticals, Inc., (NASDAQ:CNST) for $34.00 per share in cash, which represents a total equity value of $1.7 billion. Constellation shares closed the week up 70% to $33.84, while MorphoSys shares fell 6% to $20.83.

Key catalysts to watch BIIB NVAX SAGE VRTX + potential sympathy moves in play off BIIB outcome (Alzheimer’s):

Drug Price Stage Catalyst Market Cap

ALEC – Alector Inc.
AL001 (INFRONT-3)
Frontotemporal Dementia

$21.30
-0.85  -4%
Phase 3 Phase 3 commencement of dosing announced July 24, 2020.
$1.7 billion

ANVS – Annovis Bio Inc.
ANVS401
Alzheimer’s disease (AD) and Parkinson’s disease (PD)

$84.25
-11.85  -12%
Phase 2 Phase 2 full data July / August 2021. Biomarker data released May 21, 2021.
$585.3 million

ATHA – Athira Pharma Inc.
ATH-1017 (ACT-AD)
Mild-to-moderate Alzheimer's disease

$10.34
-0.41  -4%
Phase 2 Phase 2 top-line data due early-2022.
$384.9 million

BIIB – Biogen Inc.
ADUHELM (Aducanumab)
Alzheimer’s disease

$374.40
-6.51  -2%
Approved FDA approval announced June 7, 2021.
$56.4 billion

CYCN – Cyclerion Therapeutics Inc.
CY6463
Alzheimer's disease with vascular features (ADv)

$3.70
0.00  0%
Phase 2 Phase 2 trial to be initiated mid-2021.
$149.6 million

CYTH – Cyclo Therapeutics Inc.
Trappsol Cyclo
Alzheimer’s Disease

$9.87
-0.38  -4%
Phase 2 Phase 2 IND filing due 2H 2021.
$62.8 million

INMB – INmune Bio Inc.
XPro1595
Alzheimer’s disease

$17.12
-0.37  -2%
Phase 1b Phase 1b trial ongoing. Further data due summer 2021. Phase 2 trial to commence by end of 2021.
$258.9 million

LGVN – Longeveron Inc.
Lomecel-B
Alzheimer's Disease

$7.42
+0.08  +1%
Phase 2 Phase 2 trial to be initiated 2H 2021.
$140.9 million

NVAX – Novavax Inc.
NVX-CoV2373
COVID-19 vaccine

$194.37
+13.38  +7%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease.
$14.4 billion

PTPI – Petros Pharmaceuticals Inc.
Bryostatin-1
Alzheimer's disease

$3.11
-0.40  -11%
Phase 2 Phase 2 commencement of dosing announced October 6, 2020.
$30.5 million

SAGE – Sage Therapeutics Inc.
Zuranolone (SAGE-217) - WATERFALL Study (MDD-301B)
Major depressive disorder (MDD)

$55.69
-1.75  -3%
Phase 3 Phase 3 data released June 15, 2021. Primary endpoint met at Day 15 but not significant at Day 42.
$3.3 billion

SAVA – Cassava Sciences Inc.
Simufilam (PTI-125)
Alzheimer’s disease

$77.63
-12.09  -13%
Phase 2 Phase 2b trial did not meet primary endpoint - May 15, 2020. Noted improvements in biomarkers of disease pathology September 14, 2020. Phase 2 open-label data announced February 2, 2021. 9-month and SavaDx diagnostic data to be presented July 26-29, 2021 at AAIC meeting. Separate Cognition Maintenance Study (CMS) trial ongoing. Phase 3 trial to be initiated 2H 2021.
$3.1 billion

VCNX – Vaccinex Inc.
Pepinemab
Alzheimer’s Disease (AD)

$2.68
+0.02  +1%
Phase 1/2 Phase 1/2 trial first clinical sites have been activated - June 8, 2021.
$76.2 million

VRTX – Vertex Pharmaceuticals Incorporated
VX-864
Alpha-1 antitrypsin (AAT) deficiency

$188.87
+1.17  +1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$48.9 billion