Fulcrum Therapeutics Upgraded: BofA Debates Pivotal Study Success For Losmapimod In Rare Genetic Muscle Disease [Yahoo! Finance]
Avidity Biosciences, Inc. (RNA)
Company Research
Source: Yahoo! Finance
FSHD is a rare genetic muscle disease affecting a child's facial muscles, shoulders, upper arms, and lower legs. These muscles weaken and shrink (atrophy). The analyst notes that although a previous pivotal study failed using the DUX4 expression endpoint, the company conducted a post-hoc analysis and designed a new study with the endpoint “reachable workspace” (RWS), which has sparked debate in the investment community. Two key opinion leaders (KOLs) expressed optimism about the upcoming phase 3 results, citing the functional benefits seen in phase 2 using RWS, which measures upper extremity function. Despite being a newer endpoint, the KOLs believe RWS accurately tracks disease progression. They view the stabilization of RWS in phase 3 as clinically meaningful and believe replicating the phase 2 results—showing a 10% placebo-adjusted change at 48 weeks—would be a success. However, the analyst remains uncertain about the study's likelihood of success due to the unpredictabili
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News
- Avidity Biosciences to Participate in Upcoming Investor ConferencePR Newswire
- Avidity Biosciences, Inc. (NASDAQ: RNA) is now covered by analysts at The Goldman Sachs Group, Inc.. They set a "buy" rating and a $59.00 price target on the stock.MarketBeat
- Avidity Biosciences Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)PR Newswire
- Avidity Biosciences, Inc. (NASDAQ: RNA) had its "overweight" rating re-affirmed by analysts at Cantor Fitzgerald. They now have a $96.00 price target on the stock.MarketBeat
- Avidity Biosciences, Inc. (NASDAQ: RNA) had its "overweight" rating re-affirmed by analysts at Cantor Fitzgerald. They now have a $96.00 price target on the stock.MarketBeat
RNA
Earnings
- 8/9/24 - Beat
RNA
Sec Filings
- 9/23/24 - Form 4
- 9/23/24 - Form 4
- 9/23/24 - Form 4
- RNA's page on the SEC website