Jasmine Fels

Jasmine Fels from UBS Group AG asked for details on what Altimmune aims to discuss with the FDA during its end-of-Phase 2 meeting, particularly regarding the potential for a non-invasive test (NIT) based Phase 3 trial design, and whether an update would be provided post-meeting.

Answer

Chief Medical Officer Dr. M. Scott Harris responded that while specific proposals could not be shared pre-meeting, they anticipate a 'rich conversation' with the FDA, acknowledging that regulatory opinions are evolving. He confirmed that the company will provide an update to the market after the meeting concludes.

Jasmine Fels of UBS, on behalf of Ellie Merle, inquired about plans to discuss NASH F4 cirrhosis with the FDA and sought confirmation on the company's cash runway following the new credit facility.

Answer

An executive, likely CSO Dr. Scot Roberts, confirmed Altimmune is 'extremely interested' in F4 cirrhosis and intends to discuss a trial design with the FDA, believing pemvidutide is well-suited for the full spectrum of MASH. CFO Gregory Weaver clarified that the current cash position provides runway through Q3 2026, which could be extended by another year with the optional draws from the new Hercules Capital facility.

Jasmine Fels, on for Ellie Merle at UBS Group, asked what a 3x greater AUC for BMN 333 might translate to in terms of growth based on preclinical data, and how this impacts the prioritization of BMN 333 versus VOXZOGO for other disorders.

Answer

Gregory Friberg, EVP and Chief R&D Officer, referenced preclinical models where a similar increase in exposure unlocked additional growth beyond that achieved by VOXZOGO. He stated that the exact amount in humans is the key question to be tested. He confirmed the company is excited about BMN 333's potential as a best-in-class agent for other indications, like hypochondroplasia, and is actively planning for its development in those areas.

Jasmine Fels, on for Ellie Merle at UBS, asked what the '3x greater AUC' for BMN-333 might translate to in terms of growth based on preclinical data, and how this affects its prioritization versus VOXZOGO for other indications.

Answer

Gregory Friberg, EVP and Chief R&D Officer, referenced preclinical models where increased exposure unlocked additional growth beyond what VOXZOGO delivered. He stated that having BMN-333, a potential best-in-class agent, allows them to test this hypothesis in humans. He confirmed they are already planning for BMN-333 in hypochondroplasia and other indications, as a dose-ranging study is required for any new use, making it a key asset for future growth disorder treatments.

Jasmine, on behalf of Eliana Merle at UBS, asked about the potential impact of a competitor's donation to co-pay foundations on Apellis's free drug usage and inquired about the latest compliance and adherence rates for Cyfovri.

Answer

CEO Dr. Cedric Francois reiterated that Apellis is independent of foundation management. CMO Dr. Caroline Baumal reported that real-world data from large physician groups shows Cyfovri compliance is around 80%, driven by motivated patients seeking to treat their persistent vision loss.

Jasmine, on behalf of Eliana Merle at UBS, asked about the potential impact of a competitor's donation to co-pay foundations on Apellis's free drug usage and inquired about the latest compliance and adherence rates for Cyfovri.

Answer

CEO Dr. Cedric Francois reiterated that foundation funding is independent of Apellis. CMO Dr. Caroline Baumal stated that real-world data from large physician groups indicates Cyfovri compliance is around 80%, driven by motivated patients seeking to treat their persistent vision loss.

Jasmine Fels from UBS, on behalf of Ellie Merle, asked about the expected baseline rate of acute pancreatitis in the SHASTA-3 and 4 studies and the potential for expanding zodasiran's use beyond HoFH.

Answer

President and CEO, Dr. Christopher Anzalone, clarified that Arrowhead will focus zodasiran only on HoFH, as expanding to HeFH would require a large, expensive program. For the plozasiran SHTG studies, he expects baseline triglycerides to be similar to Phase II (around 850-900 mg/dL), while the dedicated pancreatitis study baseline could be around 2,000 mg/dL.