PPMD’s 2021 Virtual Annual Conference: Recently Reported Updates

As a new addition to PPMD’s 2021 Virtual Conference, PPMD invited industry partners to share new data with the community live during the Conference in a series called Recently Reported Updates.

These updates served as late-breaking sessions where we could provide more time for new data to be presented to the community outside of our shorter In the Pipeline series. Five industry partners had data that was presented to the community in this format: NS Pharma, PTC Therapeutics, FibroGen, Italfarmaco, and ReveraGen (partnership with Santhera).

You can find all of the presentations and accompanying posters where applicable below, with a brief summary of the newly reported data.

NS Pharma

Viltepso

• Dr. Paula Clemens, University of Pittsburgh, presented functional data from the Open Label Extension of NS Pharma’s Phase 2 study of Viltolarsen.
• At week 109 patients on Viltepso had a significant increase in performance of functional measures, such as time to rise and 10 meter run/walk, as compared to a matched historical control group from the CINRG natural history study that show a decline over that time period.
• View poster.

PTC Therapeutics

Deflazacort

• Dr. Craig McDonald, UC Davis, presented a comparison of performance on functional outcome measures in patients on deflazacort or prednisone from the placebo arms, those patients who did not receive investigational product, from a number of clinical trials (PTC’s Phase 2b and 3 trials of Ataluren and the Eli Lilly Phase 3 trial of tadalifil).
• The change in baseline function at 48 weeks (as measured by 6MWT, time to climb 4 stairs, time to descend 4 stairs, time to stand, and NSAA) favored deflazacort.
• Greater differences were observed in patients who were older than 8, had more advanced disease progression, or had been on steroids for over 3 years.
• This data reflects the potential benefit of deflazacort compared to prednisone for functional outcome measures.
• View poster.

FibroGen

Pamrevlumab

• Dr. Bassem Elmankabadi, FibroGen, presented data from the Phase 2 trial of Pamrevlumab, an anti-fibrotic monoclonal antibody.
• Pamrevlumab is currently being studied in Phase 3 trials for ambulatory and non-ambulatory patients.
• Data from the phase 2 trial showed potential benefit of Pamrevlumab as compared to natural history controls across a number of outcome measures, such as Performance of Upper Limb (PUL), percent predicted Force Vital Capacity (%pFVC), Left Ventricular Ejection Fraction.
• As Dr. Elmankabadi noted this is a small trial (n=21) without a placebo group, we look forward to future results from the two pivotal Phase 3 trials with Pamrevlumab.
• View poster.

Italfarmaco

Givinostat

• Dr. Paolo Bettica, Italfarmaco, presented top-line results from the Phase 2 study with Givinostat in Becker muscular dystrophy patients.
• The primary endpoint of change in total muscle fibrosis as measured from biopsies was selected based of experience in Duchenne.
• Unfortunately, over 12 months there was no change seen in total fibrosis, with no difference between placebo or treated groups.
• Positive trends in response to treatment were seen in secondary endpoints such as fat fraction which measures the infiltration of fat in the thigh and quadriceps, indicating there may be treatment benefit for slowing infiltration of fat in Becker muscular dystrophy.
• Italfarmaco is continuing to evaluate the data and speaking with regulators as the top-line results do support continued further development of Givinostat in adult BMD patients.

ReveraGen (partnership with Santhera)

Vamorolone

• Dr. Eric Hoffman, ReveraGen, presented top-line results along with new safety data on Vamorolone from the Phase 2b pivotal study VISION-DMD.
• The study met its primary endpoint of change in time to stand velocity between 6mg/kg of Vamorolone compared to placebo over 24 weeks.
• Similar statistically significant differences were seen in secondary endpoints comparing the 6mg or 2mg doses to placebo.
• Finally, the safety data presented showed Vamorolone preserves growth trajectory and had no negative impact on bone biomarkers at 24 weeks compared to prednisone.

Additional Research Highlights

In addition to these Recently Reported Updates sessions, PPMD’s 2021 Virtual Annual Conference featured dedicated updates from 20 industry partners currently in clinical trials who presented clear and concise information regarding various therapeutic approaches during our In the Pipeline Series. Our full Conference live stream, including In the Pipeline sessions, can be viewed here.