Andy Biotech

#Biotech Investor | Recovering Scientist | May have L/S position in names mentioned | Opinions are strictly my own and not intended as investment advice

[Andy Biotech @AndyBiotech]

Amazing to see translational science is moving at a much faster pace in #biotech these days from bench to bedside!

#CRISPR 11yrs
2012 Doudna/Charpentier paper
2020 Nobel
2023 First FDA approval $CRSP

#RNAi 20yrs
1998 Fire/Mello paper
2006 Nobel
2018 First FDA approval $ALNY

[Andy Biotech @AndyBiotech]

End of an era!

As a long-time follower of global #biotech/healthcare, I have learned a lot from the many good analysts at @CreditSuisse esp for their ex-US coverage, which was often more critical than typical US sell-side coverage. Best wishes to everyone on their new endeavor!

[Andy Biotech @AndyBiotech]

$SRPT ELEVIDYS priced at $3.2M

This is probably the biggest problem w/ accelerated approvals for the healthcare system.

How does it make sense that a drug under AA w/ unproven benefits priced 50% higher than $NVS Zolgensma at $2.1M w/ full approval and established efficacy?

[Andy Biotech @AndyBiotech]

"The exception is more interesting than the rule."
#Biotech reverse merger occasionally turn into >$1B buyout

Aduro➡️ $KDNY ➡️$3.2B $NVS
shell➡️Medivation➡️$14B $PFE
Trimeris➡️Synageva➡️$8.4B ALXN
CancerVax➡️Micromet➡️$1.2B $AMGN
Biodel➡️Albireo➡️$1B Ipsen
shell➡️Cougar➡️$1B JNJ

[Andy Biotech @AndyBiotech]

As expected, FDA AdCom on $BIIB/Eisai lecanemab is setting up to be a "clearing the air" event after the whole Aduhelm saga.

More importantly, FDA reviewer made it very clear the changes observed on the CDR-SB is considered "clinically meaningful".

[Andy Biotech @AndyBiotech]

$BIIB My thought on lecanemab "clinical meaningfulness" debate:

18-mo rx slowed disease progression by 6~8mo.
Is it not meaningful to pts and families to have 6~8 more months of your memory and freedom?

An oncology drug w/ 6~8mo of PFS benefit would be heralded as breakthrough!

[Andy Biotech @AndyBiotech]

Friendly reminder: "Don't talk about something you don't know much about as if you understand it. People in the field can tell."

[MuddyWatersResearch @muddywatersre]

Making it even harder for $KDNY atrasentan to get FDA approval is a competing drug, sparsentan, has exclusive orphan drug approval. Atrasentan would have to show it is more efficacious and/or safer. Comparing trial data, atrasentan seems to be inferior https://t.co/j2TYNV7ZGz

[Andy Biotech @AndyBiotech]

$SRPT FDA summary slides today emphasizes the key point that "Patients likely have only one chance to receive an AAV vector-based gene therapy for DMD", which could drive the panel discussion today to wait for Ph3 data that will be available later this year.

[Andy Biotech @AndyBiotech]

$SRPT Apparently FDA placed SRP-9001 on clinical hold in Aug 2021 due to an unexpected SAE of asthenia requiring hospitalization and respiratory support, but the company never disclosed it to investors AFAIK.

Did it not count as a material event for most important pipeline drug?

[Andy Biotech @AndyBiotech]

Overall great news for #Alzheimer pts! Another unequivocally positive Ph3 should further validate Aβ MoA, bolster doctor confidence and derisk CMS reimbursement.

$LLY comment to STAT about curve widening over time provides further evidence for the promise of "disease modifying"

[Andy Biotech @AndyBiotech]

A new addition to #Biotech best turnaround M&A

$ISEE $1➡️40X $ALPMY BO in 4y
$FPRX $2➡️20X $AMGN BO in 1y
$IMMU $2➡️40X $GILD BO in 4y
$ARQL $1➡️20X $MRK BO in 2y
$ARRY $2➡️20X $PFE BO in 3y
$ECYT $1➡️20X $NVS BO in 1y
$MDVN $2➡️40X $PFE BO in 6y
$PCYC <$1➡️200X $ABBV BO in 6y

[Andy Biotech @AndyBiotech]

Two big FDA Adcom mtg dates just released on FDA agenda

May 12 -- $SRPT DMD gene therapy
https://t.co/iVVwoUCsrn

June 9 -- $BIIB/Eisai lecanemab
https://t.co/ITRSinWRf0

[Andy Biotech @AndyBiotech]

$SNY continues its quest as biotech shorties' worst enemy

$3.3B $PRVB at 273% premium ahead of first earning after launch

$2.5B $THOR at 172% premium before PoC -> IL2 discontinued + $1.7B impairment

$3.7B $PRNB -> clinical hold due to DILI

$3.2B $TBIO -> COVID19 discontinued

[Andy Biotech @AndyBiotech]

#Biotech another addition to the list of billion$ M&A for drug dumped by pharma

$SNY $3.3B $PRVB (LLY/MGNX)
$MRK $9.5B Cubist (LLY)
$GSK $5B Tesaro(MRK)
$GSK $1.9B $SRRA (GILD)
$PFE $1.9B Vicuron(LLY)
$GALE.SW $1.5B Relypsa(AMGN)
$LLY $1.1B Dermira (Roche)
$CELG >$1B Impact(SNY)

[Andy Biotech @AndyBiotech]

$BIIB/Eisai lecanemab KO $LLY Donanemab in Round 1 of #Alzheimer's new drug fight

FDA Issues Complete Response Letter for Accelerated Approval of Donanemab due to limited number of patients with 12-month drug exposure

[Andy Biotech @AndyBiotech]

$HZNP Cheers for an early Christmas gift!

$145M River Vision acq turned out to be one of the best deals in biotech history:
Pharmacyclics -> Celera for ibrutinib
Vertex -> $538M Aurora for CF franchise
Gilead -> $470M Triangle for HIV franchise
JAZZ -> $122M Orphan Med for Xyrem

[Andy Biotech @AndyBiotech]

$HZNP to acquire River Vision for $145M. Congrats @bmgallagherjr!
https://t.co/Y14H4lt0Nm

Nice data in NEJM last wk
https://t.co/jCEPdP8RO0

[Andy Biotech @AndyBiotech]

$BIIB My thought on lecanemab "clinical meaningfulness" debate:

18-mo rx slowed disease progression by 6~8mo.
Is it not meaningful to pts and families to have 6~8 more months of your memory and freedom?

An oncology drug w/ 6~8mo of PFS benefit would be heralded as breakthrough!

[Andy Biotech @AndyBiotech]

$BIIB/Eisai - money slide for Lecanemab at #CTAD22
https://t.co/ZU1jjCYhyE
The biggest promise of "disease modifying" (vs. symptomatic) treatments is probably the compounding effect over time.
The widening of curve separation out to 18mo provides an early hint for that promise.

[Andy Biotech @AndyBiotech]

While some debate on clinical meaningfulness, we have to start somewhere for a tough disease like #Alzheimer's and this is a solid first step!

The biggest Q into #CTAD22 is probably whether benefit seen in both ApoE4 carriers and non-carriers given trial enriched w/ 70% carrier.

[Andy Biotech @AndyBiotech]

Great day for #Alzheimer's patients with first unequivocally positive Ph3 for a disease modifying treatment!

Kudos to @EisaiUS for trial execution, believe one big factor for success is how they managed to get dropout rate to only 15% amid COVID19 pandemic (vs >20% historically)

[Andy Biotech @AndyBiotech]

Great day for #Alzheimer's patients with first unequivocally positive Ph3 for a disease modifying treatment!

Kudos to @EisaiUS for trial execution, believe one big factor for success is how they managed to get dropout rate to only 15% amid COVID19 pandemic (vs >20% historically)

[Andy Biotech @AndyBiotech]

#ESMO22 $AMGN Lumakras very underwhelming Codebreak-200 data on top of an already slow launch

How to justify $18K/mo price w/ ~1mo mPFS benefit and detrimental overall survival?

Double whammy day w/ $BMY Sotyktu clean label re. Otezla.
Silver lining: more need for SMID bio M&A?