Shares of Dyne Therapeutics, Inc. DYN, a clinical-stage muscle disease company, tumbled 30.7% on two key announcements.
The company announced that some of its senior executives have stepped down from their respective positions. Earlier, DYN reported mixed data from its ongoing phase I/II study, the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD).
Year to date, Dyne has surged 155.8% against the industry’s 2.9% decline.
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Dyne Reports New Data From DMD Trial
The DELIVER study is evaluating DYNE-251 in people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody that binds to the transferrin receptor 1, which is highly expressed on muscle.
Data reported includes six-month biomarker and functional data from 8 male patients enrolled in the 20 mg/kg (approximate PMO dose) cohort who were randomized to receive DYNE-251 or placebo once every four weeks, and 12-month functional data from six participants in the 10 mg/kg cohort.
DMD, a rare disease, is caused by mutations in the gene that encodes for dystrophin. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function.
Patients treated with 20 mg/kg of DYNE-251 once every four weeks reported a mean absolute dystrophin expression of 3.71% of normal levels (unadjusted for muscle content), more than 10-times higher than the 0.3% reported in a study of the weekly standard of care, eteplirsen. The DYNE-251 treated group reached 8.72% mean absolute dystrophin when muscle content was adjusted. This level was greater than that reported by peptide conjugate PMOs in clinical development.
Per Dyne, meaningful improvements in multiple functional endpoints were observed in both the cohorts.
Dyne stated that DYNE-251 demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. The majority of treatment-emergent adverse events were mild or moderate. However, two participants at the 40 mg/kg dose level experienced serious adverse events potentially related to the study drug. Per the company, both the participants recovered.
DYN’s Key Executives Quit
Post the data readout, Dyne announced that its chief medical officer (CMO), chief operating officer and chief business officer are leaving the company. Nonetheless, the CMO, Wildon Farwell, will remain through the end of 2024.
Dyne Fails to Impress Investors
Investors were not impressed with the data reported by the company and the exit decision of some of its top executives. Consequently, the stock lost 30.7% on Sept. 3.
Eteplirsen is Sarepta Therapeutics’ SRPT first DMD drug marketed under the brand name Exondys 51.
Sarepta is the market leader in the treatment of DMD. Apart from Exondys 51, SRPT markets Elevidys (SRP-9001), the first and currently the only gene therapy for DMD. Elevidys is approved for treating all DMD patients aged four years and older. It also has two more DMD drugs in its arsenal, namely Vyondys 53 and Amondys 45.
DYN’s Zacks Rank and Stocks to Consider
Dyne currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the drug/biotech sector are Krystal Biotech KRYS and Exelixis EXEL. While KRYS sports a Zacks Rank #1 (Strong Buy) at present, Exelixis carries a Zack Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Krystal Biotech’s earnings per share (EPS) estimate for 2024 has increased to $2.38 from $1.91 in the past 30 days. Shares of KRYS have surged 51.1% year to date.
In the past 30 days, EXEL’s EPS estimate for 2024 has moved north to $1.79 from $1.39. Year to date, shares of EXEL have risen 8.5%.
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