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

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!

$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?

"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

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".

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

$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.

$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?

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"

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

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

$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

#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)

$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

$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

$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!

$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.

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.

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)

#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?