基因编辑：2025年5月月度回顾

Restricted - External U.S. Small & Mid Cap BiotechnologyPOSITIVEU.S. Small & Mid Cap BiotechnologyGena Wang, PhD, CFA+1 212 526 4252gena.wang@barclays.comBCI, USHang Hu, PhD+1 212 526 6364hang.hu@barclays.comBCI, USTony Deng, MD+1 212 526 0350tony.deng@barclays.comBCI, USJustin Kong, MD+1 212 526 1961justin.kong@barclays.comBCI, US •Gene therapies in DMD.Most investors believe that SRPT's Elevidys is unlikely to be pulled•offthe market. Key concerns were label restriction, long-term patient demand, and acompetitive landscape.•DM1 and FSHD.Overall investors were bearish on DYN's (not covered) DM1 program to•receive an accelerated approval path (AA), but slightly more positive on the possibility of AAfor RNA's FSHD program.•ATTR-CM panel.Investors were overall bullish on ALNY Amvuttra market oppt but were•largely split on near-term launch uptake and overall stock upside potential given valuation.New COVID-19 vaccine framework and read-through to drug approval paths under newCBER leadership.Most investors were somewhat relieved when the new framework for theCOVID vaccine was announced. Concerns for Director Vinayak Prasad making some drasticchanges were largely gone. See MRNA: FDA's Covid-19 vaccine framework removes near-termoverhang, 05/20/2025.Journavx scripts and VRTX from here.Some investors pushed back regarding our projectedscripts and gross revenue ($36M) for 2Q25. Key concern was continued high gross to net withfree drug, resulting in limited revenue. Investor expectations are low for 2Q25 and2025. See Weekly IQVIA Rx suggest Journavx gross revenue of ~$31-36M and tafamidis netrevenue of ~$1.1B in 2Q25, 05/23/2025.LEGN stock performance.Overall investors were surprised on the magnitude of the stock'smovement. Key bear thesis points were toxic drug profile and small market demand,difficultymoving community patients into academic centers to receive CAR T therapies. Investors wereskeptical on company-compiled 2025 consensus of $1.9B (Bloomberg consensus of ~$1.9B).See LEGN: 1Q25 incremental; eyes on ASGCT and ASCO data updates, 05/13/2025;see Takeaways of ASCO 2025 abstracts, 05/23/2025.LEGN stock move post earnings and EHA abstracts.LEGN was under pressure post bothearnings and release of EHA abstracts. On earnings, the main pushback was modest revenuegrowth in 2Q25 with accelerated growth largely implied in 2H25; and skepticism regardingdemand when supply reaches 10k doses by YE25. For EHA abstracts, the main pushback was theclean safety for anito-cel without any new events of late onset neuro-tox and some improvedefficacywith higher CR rate. Other investors emphasized thatdifferentpatient baseline andenrollment exclusion criteria would probably make comparison impossible. See LEGN:1Q25incremental; eyes on ASGCT and ASCO data updates, 05/13/2025.CYTK MAPLE data and next step.While positive data was largely expected, some investorsquestioned the limited information in the press release and were concerned about themagnitude of benefit, especially given prior experience of minimal communication frommanagement regarding REMS. Some other investors remained focused on the approval ofaficamten on Dec 26, and continued to believe in thedifferentiatedprofile vs. Camzyos.See CYTK: Positive MAPLE-HCM as expected; anticipating on time aficamten approval,05/13/2025.VRTX Journavx IQVIA scripts and 2Q25 revenue projections.Some investors were positivelysurprised by our 2Q25 revenue projection of $36M if linear growth was assumed. Investors wetalk to had lower revenue and gross to net expectations than our 2Q25 estimates. See VRTX:Journavx IQVIA Rx suggests ~$30-36M gross revenue in 2Q25, 05/16/2025.ALNY impact from MFN and Trump's drug pricing plan.Most investors we talk to agreed thatstock reaction was overdone and there should be limited impact from the anticipated Trump 2 announcement on drug pricing plans. Clarity is being sought on the following: 1) Despiteorphan designation, would Amvuttra be excluded from IRA price renegotiation given it has twoindications (ATTR-PN and ATTR-CM)? 2) Given increasing patient diagnosis, would ATTR-CM loseits orphan disease category? 3) Ex-US and US pricedifferences?4) Medicaid exposure? 5) WillAmvuttra receive extra scrutiny due to higher price compared to other drugs?See ALNY: Amvuttra - likely limited impact from anticipated Trump announcement for Medicaredrug price plan, 05/08/2025.PTCT HTT data and stock from here.Most investors we talk to were caught by surprise on thetiming of the data, which was earlier and somewhat more messy than expected. However, it wasa split view on accelerated approval path – bulls (minority) still see a chance given the overalldifficultdisease and the FDA's willingness to approve drugs in Huntington's disease. Bearsbelieve accelerated approval wasoffthe table given lack of clear trends on manymeasurements. Some even questioned the possibility of a positive Ph3. However, given the NVS(covered by Emily Field) deal, it was thought that development