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

$19.92
-1.36  -6%
Phase 3 Phase 3 commencement of dosing announced July 24, 2020.
$1.6 billion

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

$25.76
-0.10  0%
Phase 2a Phase 2a data showed improvements in cognition in AD patients by 4.4 points compared to placebo at 3.3. WAIS coding in AD patients show a statistically-significant 23% improvement and PD patients a statistically-significant 24% improvement compared with placebo, noted September 2021. Phase 2a of PD patients noted statistically significant improvements in WAIS coding scores, in the UPDRS 2, 3, 4, and in total MDS-UPDRS test compared to baseline, noted October 5, 2021. Additional Phase 2a data reported reduced neurotoxic proteins in both AD and PD patients, with decreased inflammation and improved axonal integrity and synaptic functions in both AD and PD patients, noted November 12, 2021. Phase 3 trials to be initiated 1Q 2022.
$208.7 million

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

$13.94
-0.52  -4%
Phase 2 Phase 2 completed enrollment, noted October 22, 2021. Phase 2 top-line results expected 1H 2022.
$520.7 million

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

$242.90
-7.23  -3%
Approved FDA approval announced June 7, 2021.
$35.7 billion

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

$2.45
-0.20  -8%
Phase 2 Phase 2 trial has been initiated.
$106.1 million

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

$5.28
-0.02  0%
Phase 2 Phase 2 IND filing submitted November 16, 2021.
$44.4 million

INMB – INmune Bio Inc.
XPro (pegipanermin)
Alzheimer’s disease

$13.33
-0.23  -2%
Phase 2 Phase 2 trial planned with data due 2H 2023.
$237.9 million

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

$30.67
-11.63  -27%
Phase 2 Phase 2 trial to be initiated by 1Q 2022.
$586.4 million

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

$217.97
+17.90  +9%
BLA Filing BLA due in US by year-end 2021.
$16.5 billion

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

$39.32
-0.69  -2%
NDA Filing Phase 3 study primary endpoint met at Day 15 but not significant at Day 42 - June 15 2021. New data showed improvement in measurements of overall quality of life, functioning and general well-being achieved at Day 15 and sustained at Day 42 - October 4, 2021. NDA filing 2H 2022 with rolling submission in early 2022.
$2.3 billion

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

$55.96
-2.09  -4%
Phase 3 First Phase 3 trial safety and efficacy 52-week study initiated in 750 Patients with AD - October 6, 2021. Second Phase 3 trial initiated November 18, 2021.
$2.2 billion

SNPX – Synaptogenix Inc.
Bryostatin-1
Alzheimer's disease

$9.52
-0.36  -4%
Phase 2b Phase 2b trial ongoing.
$63.5 million

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

$1.70
+0.04  +2%
Phase 1/2 Phase 1/2 topline data due late 2022 or early 2023.
$52.4 million

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

$183.59
-2.65  -1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$46.7 billion