Savara Provides Positive Update on Development and Commercial Preparations of Molgradex, Including Expansion into NTM In CF
Acquires assets of Cardeas and Announces Appointment of
Molgradex Program Update
IMPALA and IMPALA-X Clinical Studies
Savara’s pivotal Phase 3 IMPALA study is evaluating Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis, or aPAP. Completion of patient enrollment remains on track for the third quarter of 2018 and topline data are anticipated in the second quarter of 2019. The IMPALA study includes a six-month placebo-controlled period with 2:1 randomization on Molgradex or placebo, followed by a six-month open-label period with all subjects receiving Molgradex. Earlier this year, the Company launched the IMPALA-X study, an open-label, multicenter study designed to evaluate the long-term use of Molgradex in patients with aPAP. The IMPALA-X study offers patients completing the IMPALA study the opportunity to continue treatment with Molgradex for up to three additional years.
Savara is encouraged by positive investigator feedback on treatment with Molgradex in the IMPALA study as well as the high interest in participation in the IMPALA-X study. The Company believes high enrollment into the IMPALA-X study gives important insight into the level of satisfaction with Molgradex. Of the 14 subjects eligible to enroll in the IMPALA-X study by the end of the second quarter of 2018, ten subjects have enrolled to date, while the remaining four subjects are expected to be enrolled upon delivery of the study drug to their clinical sites.
Driven by its confidence in the outcome of the IMPALA study, the Company will expedite its preparation for potential commercial launch by starting investments into core commercial staff and external activities required for timely launch, subject to product approval by the
“This business update on Molgradex emphasizes the gradual inclusion of a strong commercial focus within Savara in addition to our activities in product development, and it represents an important step in our evolution towards a fully integrated pharmaceutical company with our own product revenues,” stated
Phase 2a Open-Label Clinical Study in Cystic Fibrosis Affected Individuals With NTM
Savara is currently conducting a Phase 2a open-label clinical study,
The Company is now preparing to initiate a new study in the U.S. in CF affected individuals with chronic NTM infection. This Phase 2a open-label study will enroll 30 subjects with CF and chronic NTM lung infection, and is planned to begin in the first quarter of 2019. The study will enroll subjects with chronic Mycobacterium abscessus (M. abscessus) or Mycobacterium avium complex (MAC) infection. The study will comprise a 48-week treatment period and a 24-week follow-up period, and three subgroups of subjects will be recruited into the study. Group 1 will consist of subjects who have been on antimycobacterial treatment for at least six months prior to the baseline visit. Group 2 will consist of subjects who have stopped antimycobacterial treatment due to intolerance or lack of effect prior to the baseline visit. Group 3 will consist of subjects who have not been treated with antimycobacterial treatment but are clinically stable prior to the baseline visit.
The primary endpoint in the study will be NTM sputum culture conversion to negative (3 negative samples 4 weeks apart). Secondary endpoints include: (i) consistent NTM sputum culture conversion until end of treatment; (ii) sputum smear conversion/reduction in count of acid-fast bacilli (AFB); (iii) durable conversion (culture/smear) after end of treatment; (iv) change in pulmonary function; (v) change in respiratory symptom score; (vi) change in body mass index.
Exploratory Product Pipeline
As part of its growth strategy, Savara has launched an exploratory product pipeline comprising pre-proof-of-concept, high-potential programs focused on difficult-to-treat lung diseases. As the first new product in the exploratory pipeline, Savara acquired the rights to amikacin/fosfomycin inhalation solution, a Phase 2 ready product candidate that had been in development by
Following the acquisition, Savara appointed
“I am excited to become an advisor to Savara at such an important time in the Company’s development,” said Dr. Montgomery. “I believe the results of the planned and underway clinical trials in multiple rare lung diseases will go far to meet Savara’s vision of becoming “The Orphan Lung Disease Company”. I have provided input on the development of Molgradex and AeroVanc for many years and look forward to assisting Savara’s team with these programs that address unmet medical needs. In addition, I look forward to assisting the Company in identifying and evaluating new impactful products to help fuel the Company’s growth.”
Aerosolized amikacin/fosfomycin is a proprietary combination antibiotic that has demonstrated potent and broad-spectrum antibacterial activity in highly drug resistant pathogens, with promising applications in the intensive care unit (ICU) setting. In particular, multi-drug resistant Gram-negative infections in the ICU continue to be associated with significant mortality and require new innovative treatments. In this context, the potential to combine locally administered antibiotics with Molgradex’s localized stimulation of the innate immune system—an anti-infective immunotherapy—is a new approach that could represent the beginning of a paradigm shift in the treatment of refractory lung infection.
“The expansion of our NTM program, and the launch of our exploratory pipeline underline our commitment to growth through innovation and acquisition,” said Dr.
Savara is an orphan lung disease company. Savara's pipeline comprises: Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP and in Phase 2a development for nontuberculous mycobacteria, or NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in cystic fibrosis, or CF. Savara's strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. Savara's management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization. More information can be found at www.savarapharma.com. (Twitter: @SavaraPharma)
Dr. Montgomery is a board-certified internist and pulmonologist with more than 35 years of drug development and general management experience in the pharmaceutical industry, including positions at
Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as "expect," "intend," "plan," "anticipate," "believe," and "will," among others. Such statements include, but are not limited to, statements relating to being encouraged by positive investigator feedback for treatment with Molgradex in the IMPALA study as well as the high interest in participation in the IMPALA-X study, our belief that high enrollment into the IMPALA-X study gives important insight into the level of satisfaction with Molgradex, that the remaining four subjects are expected to be enrolled upon delivery of the study drug to their clinical sites, our confidence in the outcome of the IMPALA study, that we will expedite our preparation for potential commercial launch, our belief that robust results in the IMPALA study would make Molgradex eligible for designation as a breakthrough therapy, and facilitate the submission of the Molgradex BLA, in the first half of 2020 with an anticipated commercial launch in late 2020 or early 2021, that this business update on Molgradex emphasizes the gradual inclusion of a strong commercial focus within Savara and represents an important step in our evolution towards a fully integrated pharmaceutical company with our own product revenues, that the Phase 2a open-label study will enroll 30 subjects with CF and chronic NTM lung infection, and is planned to begin in the first quarter of 2019, that interim results are expected in the fourth quarter of 2018 with top line results in the second quarter of 2019, anticipating investing initially up to
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