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SAVARA REPORTS FOURTH QUARTER / YEAR-END 2019 FINANCIAL RESULTS AND PROVIDES BUSINESS UPDATE Announced Positive Results from IMPALA Open-Label Follow-Up Period That Demonstrate Continued Improvement After Longer Term Exp

Key Takeaway: SAVARA REPORTS FOURTH QUARTER / YEAR-END 2019 FINANCIAL RESULTS AND PROVIDES BUSINESS UPDATE Announced Positive Results from IMPALA Open-Label Follow-Up Period That Demonstrate Continued Improvement After Longer Term Exposure to Molgradex Company Planning an Additional Phase

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SAVARA REPORTS FOURTH QUARTER / YEAR-END 2019 FINANCIAL RESULTS
AND PROVIDES BUSINESS UPDATE
Announced Positive Results from IMPALA Open-Label Follow-Up Period That Demonstrate Continued
Improvement After Longer Term Exposure to Molgradex
Company Planning an Additional Phase 3 Study of Molgradex for the Treatment of Autoimmune Pulmonary
Alveolar Proteinosis (aPAP)
Announced Top Line Microbiology Data from OPTIMA, a Phase 2a Clinical Study in Nontuberculous
Mycobacterial (NTM) Lung Infection
AUSTIN, TX March 12, 2020 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, today reported financial results for the
fourth quarter and full year ending December 31, 2019 and provided a business update.
Results from the open-label period of IMPALA reaffirm
our strong belief that Molgradex could improve outcomes for patients with aPAP, a rare and debilitating lung disease, said Rob Neville, Chief Executive Officer, Savara. We continue our conversations with the FDA, as our highest priority
is to get clarity from the agency regarding the design and endpoints of a second Phase 3 study in aPAP.
The Company today announced results from the open-label follow-up period of IMPALA, a Phase 3 study that evaluated
Molgradex for the treatment of aPAP. The results, found below, will be discussed on today s conference call/webcast. Data from the double-blind period of IMPALA were presented at the 2019 European Respiratory Society (ERS) International
Congress and can be found on the Articles and Publications page of Savara s website.
IMPALA was a randomized, double-blind, placebo-controlled clinical study designed to compare the efficacy and safety of Molgradex with placebo in patients with
aPAP. Patients were randomized to receive treatment for 24 weeks in one of three arms: 1) Molgradex 300 g administered once daily continuously over 24 weeks, 2) Molgradex 300 g, and matching placebo, each administered once daily every
other week, or 3) inhaled placebo administered once daily continuously over 24 weeks. At the end of the 24-week double-blind period, all treatment arms rolled into a
24-week open-label follow-up period and received Molgradex 300 g administered once daily every other week.
The primary endpoint was change from baseline in alveolar-arterial oxygen gradient, or (A-a)DO2, to week 24. In addition, three key secondary endpoints St. George s Respiratory Questionnaire (SGRQ), six-minute walk distance (6MWD), and time
to requirement for whole lung lavage (WLL) along with multiple other secondary and exploratory endpoints were assessed.
IMPALA Open-Label Period
A total of 128 patients completed the open-label period of IMPALA. Sixty-eight patients enrolled in the open-label period through week 48. The other 60
patients were enrolled under an early protocol version of the study through week 72.
During the double-blind period, a dose frequency dependency was
observed in most endpoints with continuous administration of Molgradex showing higher efficacy than placebo and continuous administration appearing to result in higher efficacy than once daily dosing every other week. Open-label results presented
below focus on the group that had received a once daily continuous dose (OD) of Molgradex during the double-blind period versus those that had received once daily placebo (PBO) during the double-blind period both of which received once daily
dosing every other week during the open-label period.
Improvements in Two Independent Measures of Gas Exchange
In addition to (A-a)DO2, diffusing capacity of the lungs for
carbon monoxide (DLCO) is another way to assess gas exchange. As with (A-a)DO2, DLCO is impaired in patients with aPAP and was assessed as an additional
secondary endpoint in the study.
Patients who had been in the OD treatment group during the double-blind period:
Improvement from baseline was maintained or progressively increased in these patients during the open-label period of the study. The average (A-a)DO2 improvement in the double-blind period was -12.1 mmHg at week 24, compared to an average improvement from
baseline in the open-label period of -12.2 mmHg and -18.8 mmHg at weeks 48 and 72, respectively. The mean absolute improvement in DLCO during the double-blind period was
11.6% predicted at week 24, compared to a mean absolute improvement from baseline in the open-label period of 13.7% and 19.8% predicted at weeks 48 and 72, respectively.
Patients who had been in the PBO group during the double-blind period:
PBO patients that transitioned to active drug in the open-label period showed similar average improvements to those who had been in the double-blind OD group.
The average (A-a)DO2 improvement in the double-blind period was -8.8 mmHg at week 24, compared to an average improvement from baseline in the open-label
period of -14.6 mmHg and -17.0 mmHg at weeks 48 and 72, respectively. The mean absolute improvement in DLCO during the double-blind period was 3.9% predicted at week 24,
compared to a mean absolute improvement from baseline in the open-label period of 14.2% and 18.0% predicted at weeks 48 and 72, respectively.
Improvements in SGRQ
Patients who had been in the OD treatment group during the double-blind period:
Changes from baseline in the SGRQ were maintained and further increased with longer exposure to Molgradex. The average improvement during the double-blind
period was -12.3 points at week 24, compared to an average improvement from baseline in the open-label period of -13.1 and -17.4
points at weeks 48 and 72, respectively.
Patients who had been in the PBO group during the double-blind period:
At the end of the open-label period, PBO patients that transitioned to active drug showed similar average improvements in the SGRQ to those who received OD in
the double-blind period. The average improvement during the double-blind period was -4.7 points at week 24, compared to an average improvement from baseline in the open-label period of -14.7 and -14.9 points at weeks 48 and 72, respectively.
Patients who had been in the OD treatment group during the double-blind period:
The average improvement during the double-blind period was 39.6 meters at week 24, compared to an average improvement from baseline in the open-label period of
48.9 meters and 71.7 meters at weeks 48 and 72, respectively.
Patients who had been in the PBO group during the double-blind period:
The average improvement during the double-blind period was 6.0 meters at week 24, compared to an average improvement from baseline in the open-label period of
40.4 meters and 29.9 meters at weeks 48 and 72, respectively.
During the double-blind period of the study, 33 whole lung lavage procedures were required, including 9 in the continuous group compared to 17 in the placebo
group. During the open-label period of the study only 5 whole lung lavage procedures were conducted. The pre-study rate of WLL was 0.8 procedures per patient year, compared to 0.055 procedures per patient year
during the open-label period of the study.
The Company intends to present more comprehensive data from the IMPALA study at an upcoming scientific conference and/or
submit it for consideration in a peer-reviewed journal.
Molgradex for NTM Lung Infection
The Company today announced top line microbiology results from OPTIMA, a Phase 2a, exploratory, open-label,
non-controlled clinical study evaluating Molgradex for the treatment of NTM lung infection in patients not affected by cystic fibrosis (CF). The results, found below, will be discussed on today s
conference call/webcast.
OPTIMA study, comprised of a 48-week treatment period and a 12-week follow-up period, enrolled patients with either
Mycobacterium avium complex (MAC) infection, or the more difficult-to-treat Mycobacterium abscessus (MABSC) infection. Prior to enrolling, all patients
were diagnosed with bronchiectasis, with all except one having radiological evidence of nodular multifocal and/or cavitary bronchiectasis. Two treatment groups of patients were recruited into the study, both of which received Molgradex 300 g
administered once daily. Treatment group one consisted of patients who remained sputum culture positive while currently on a multidrug NTM guideline-based anti-mycobacterial regimen, which had been ongoing for at least
six-months prior to the baseline visit. Treatment group two consisted of patients who remained sputum culture positive, but either stopped a multidrug NTM guideline-based anti-mycobacterial regimen at least 28
days prior to screening due to lack of response or intolerance, or never started such treatment.
Summary of Efficacy and Safety Results
Thirty-two patients were treated in the study, of which 24 had MAC infection and 8 had MABSC infection. Of the patients
with MAC infection, 11 were in treatment group one and 13 were in treatment group two. Of the patients with MABSC infection, 3 were in treatment group one and 5 were in treatment group two.
Results from the Intention-to-Treat (ITT) population showed that 5 out of 24
patients (21%) with MAC infection achieved a sputum culture conversion, defined as at least three consecutive sputum samples without growth of nontuberculous mycobacteria during the treatment period. Two of these patients, one from each treatment
group, remained culture negative through the 12-week follow-up period. Sputum culture conversions were not observed in patients with MABSC infection.
Among the 32 treated patients in the safety population, 14 experienced serious adverse events (SAEs), one of
which was considered potentially treatment related. The most common SAE was infective exacerbation of bronchiectasis. Three patients died during the study, with all deaths unlikely related to the study treatment.
The Company will continue to assess data from the OPTIMA study to better understand the clinical outcomes seen in some patients. Next steps for the NTM
program will be determined once results from ENCORE, an ongoing Phase 2a, open-label, non-controlled study evaluating Molgradex for the treatment of NTM in patients living with CF, are available.
The Company intends to submit the full data set from OPTIMA for consideration at an upcoming scientific conference.
Enrollment continues in AVAIL, a pivotal Phase
3 clinical study of AeroVanc for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in CF. Total target enrollment is 200 patients. The adult population is fully enrolled, and the Company has
enrolled 133 patients out of a target of 150 in the primary analysis population (younger patients between 6-21 years of age). The Company will continue enrollment until Q2 2020 and anticipates enrolling a
total of ~140 younger patients. Top line results are expected in early 2021.
Fourth Quarter Financial Results (Unaudited)
Savara s net loss for the fourth quarter of 2019 was $31.7 million, or $(0.72) per share, compared with a net loss
of $10.5 million, or $(0.29) per share, for the fourth quarter of 2018. Research and development expenses were $8.7 million for the fourth quarter of 2019, compared with $9.9 million for the fourth quarter of
General and administrative expenses for the fourth quarter of 2019 and 2018 were $3.3 million.
As of December 31, 2019, the Company had cash of $49.8 million and short-term investments of $72.0 million. Operating expenses for
the fourth quarter of 2019 were approximately $31.5 million which included $19.4 million in noncash goodwill impairment charges. The Company ended the fourth quarter of 2019 with approximately $25.1 million outstanding in
short- and long-term debt on its balance sheet, which was used and will continue to be used to bolster operations and advance the development of drug candidates.
Fiscal Year 2019 Financial Results
loss for the year ended December 31, 2019 was $78.2 million, or $(1.95) per share, compared with a net loss of $61.5 million, or $(1.85) per share for the year ended December 31, 2018.
Research and development expenses increased by $1.6 million, or 4.3%, to $38.8 million for the year ended December 31, 2019 from
$37.2 million for the year ended December 31, 2018. The increase was primarily due to increased development and regulatory costs associated with the development of Molgradex for the treatment of aPAP, NTM, and NTM in patients with CF as
well as development costs associated with the enrollment and other Phase 3 study activities of the AeroVanc program.
General and administrative expenses
increased by $2.4 million, or 22.8%, to $13.1 million for the year ended December 31, 2019 from $10.7 million for the year ended December 31, 2018. The increase was primarily due to increased commercial costs related to
Last updated: Mar 12, 2020