
Alex Harding
@AlexHarding7
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Testing for lipoprotein(a) is becoming a lot more common The best options we have to lower it currently are PCSK9 mAbs (~30% lowering) and apheresis (specialist) But the first phase 3 results for targeted agents (antisense oligos, siRNAs, inhibitors) are expected next year...
Congrats to my mentee @MattRamsisMD for leading this paper examining trends in Lp(a) testing. https://t.co/XKCOWhFA3A Testing has increased, but it is still very low (less than 1% of the population). Per @nationallipid and @society_eas and @escardio guidelines everyone
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Twenty Years In Early Stage Biotech VC (Part 1) Here are some observations on the “Science” of startups from my vantage point as an early stage VC… · Science-first opportunism beats top-down strategy for deal selection · Don’t believe what you read in science papers at face
Twenty years ago in mid-October 2005, I joined @AtlasVenture. Hard to believe - time flies. A big thank you to my partners JF Formela and Peter Barrett, who took a bet on me. Both have been great mentors, partners, and friends. Since 2005, I've worked with hundreds of
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Should be required reading in biotech!
Twenty Years In Early Stage Biotech VC (Part 1) Here are some observations on the “Science” of startups from my vantage point as an early stage VC… · Science-first opportunism beats top-down strategy for deal selection · Don’t believe what you read in science papers at face
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https://t.co/LsgdwXEmFt Let's focus on how to increase speed and reduce cost of developing medicines -- there are lessons we can learn here from Chinese biotechs. @ldtimmerman
@NeilUdassi
timmermanreport.com
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $199 per year, you reward quality independent biotech reporting,...
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Very excited for this!
Our MYB RNA degrader, REM-422, will be featured in late-breaking oral and poster presentations and in a session on targeting RNA with small molecules at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics. #Targets25 ➡️ https://t.co/eoSx7LEb4n
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We’re proud to share new preclinical data from our novel SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation at the ESGCT 2025 Annual Congress: https://t.co/1UZpJhWN8J
#ESGCT2025
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Totally agree. Restricting access to inhalers in the name of reducing greenhouse gas emissions is ridiculous.
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This is the latest in a string of recent successes for PCSK9 inhibition 1. PCSK9 inhibition (with an injectable antibody) reduces cardiovascular risk as primary prevention in a phase 3 trial 2. PCSK9 siRNA FDA-approved for first-line use – as primary prevention 3. Oral PCSK9
🤑If VESALIUS-CV holds on full data, it’s a paradigm shift for Repatha. Blockbuster math below..... PCSK9 wins primary prevention, not just post-MI/stroke. This unlocks tens of millions of eligible patients before 1st event!!! 1️⃣Repatha today: ~$1.6B sales >Primary Prevention
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VESALIUS (Repatha for primary prevention) meets primary outcome! Big win for patients and hopefully good news for access. Can't wait to see the details and numbers at @American_Heart Scientific Sessions #AHA25. https://t.co/DVVZvTnxXZ
amgen.com
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Good news re orphan drugs in the IRA. Next step is to eliminate the 'pill penalty' (9 vs 13 years). https://t.co/Sagl0qlhDx
endpoints.news
CMS finalized guidance this week that the agency says will increase transparency in the third round of Medicare drug price negotiations and 'preserve critical incentives for rare disease research.'
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Great article that is worth a repost for $QURE AMT130 amazing data. How incredible is it that one of the patients not only remained stable but was able to go back to work? https://t.co/Z0rw9e2zCl
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This 2012 piece on the #placeboeffect by #DrugBaron (@sciencescanner) is a very topical today with the $MLTX news & "unexpected PBO" -you won't regret the time spent reading it promise! https://t.co/5FQ2B2oX3C
drugbaron.com
Nothing kills biotech companies like an active placebo. More often than not the press release after a failed trial points the finger at a "surprisingly large placebo response" against which the...
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Will companies with US based mfg take this as an opportunity to raise launch prices?
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As if q6month dosing of TSLP mAbs wouldn’t be possible without AI… What is the putative advantage of this molecule over the other q6month TSLP mAbs? AI is cool?
What if an antibody engineered with #AI could make six-month dosing possible for patients with severe asthma? Today, we announced that Phase 1 results for GB-0895 will be presented as a late-breaker at #ERS2025 in Amsterdam this Sunday. GB-0895 is an investigational anti-TSLP
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4) Targeting HTT exon 1 fragments (as QURE does) is likely very important, though this remains to be proven more definitively
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3) Intrathecal ASOs have class related toxicities, independent of target. Multiple such ASOs (including Spinraza) have reported ventricular enlargement similar to tominersen
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2) HTT is a good target. After tominersen, some people went to the extreme of questioning HTT as a target. Clearly, they just weren’t getting sufficient target engagement
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QURE’s data shoulders any doubt in that regard, as their product does not spare wt HTT and in fact likely produces greater wt HTT KD in some brain regions
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In 2021, the tominersen ASO trial was stopped early for futility and sent the HD field into a deep spiral. Today’s data should clear some things up and renew interest in HD drug development. 1) Tominersen’s failure was not due to wt HTT KD. That was pretty clear all along but…
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