5 Stocks Likely To Move Big This Quarter On Rare Genetic Disease Readouts
We used our BPIQ platform to identify biopharma stocks likely to move big this quarter on rare genetic disease readouts. We found 5 of them, including an upcoming approval decision! See the table and text below for more details, which are likely to be a big or a suspected mover, based on implied volatility data.
$MRNS
Ganaxolone / TSC (Tuberous Sclerosis Complex) / Phase 3 ✔️
Ganaxolone is a neuroactive steroid that binds to GABA-A receptors in the brain and is thought to help normalize over-excited neurons and provide a stabilizing effect. Ongoing Ph3 trial overview and prior results 📸
$SRRK
Apitegromab (SRK-015) / Spinal Muscular Atrophy (SMA) / Phase 3
Apitegromab is a selective muscle-targeted approach designed to improve motor function. It specifically and only inhibits myostatin and has the potential to build muscle and strength to improve patient outcomes.
$BHVN
Taldefgrobep alfa (BHV-2000) / Spinal Muscular Atrophy (SMA) / Phase 3
Taldefgrobep alfa inactivates free myostatin (GDF-8), an inhibitor of muscle growth. Also inhibits skeletal muscle ActRII receptor signaling, and thus, enhances muscle mass.
$AMLX
AMX0035 / Wolfram Syndrome / Phase 2
AMX0035 is shown to target two key neurodegenerative disease pathways by mitigating ER stress and mitochondrial dysfunction, reducing neuronal cell death. Phase 2 HELIOS study in Wolfram syndrome ongoing.
$SLNO
Diazoxide Choline Controlled-Release (DCCR) / Prader-Willi Syndrome (PWS) / PDUFA
DCCR is a proprietary extended-release formulation of diazoxide that can activate the KATP channel in the brain, pancreas and fat tissue, which may:
help regulate appetite and reduce hyperphagia
improve other difficult to manage behaviors
reduce resistance to insulin and leptin
help limit the accumulation of excess body fat
Footnote: ✔️ Confirmed by implied volatility data.
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#MRNS #SRRK #BHVN #AMLX #SLNO
This article is not investment, tax, or legal advice. Please do your own diligence and seek advice from professional advisors representing your interests.
Article history:
First published 09/26/24 JD, SK, AV & EJV