Ann: Positive Top-Line Results in FSGS Phase 2a Clinical Study, page-18

For Immediate Release
DIMERIX ANNOUNCES POSITIVE TOP-LINE RESULTS IN
PHASE 2a CLINICAL STUDY OF DMX-200 IN FSGS
Highlights
• Primary and secondary endpoints met in the Phase 2a study of DMX-200 in FSGS patients
• DMX-200 was found to be generally safe and well-tolerated in FSGS patients
• 86% of patients demonstrated a reduction of proteinuria with DMX-200 versus placebo
• A 29% reduction in proteinuria was observed across all patients receiving DMX-200 compared to
placebo
• 29% of patients achieved a >40% reduction in proteinuria on DMX-200 compared to placebo
• Multiple patients from both FSGS and diabetic kidney disease studies continue on DMX-200 via
TGA Special Access Scheme
• Statistically powered Phase 2 study in diabetic kidney disease results due in 4 - 6 weeks
MELBOURNE, Australia, 29 July 2020: Dimerix Limited (ASX: DXB), a clinical-stage drug development
company, today announced positive top-line results from the Phase 2a ACTION study of DMX-200 for
the treatment of focal segmental glomerulosclerosis (FSGS), a rare kidney disorder without an
approved pharmacologic treatment that often leads to end-stage kidney failure. The study
administered 240mg of oral DMX-200 to patients with FSGS already taking a stable 300 mg dose of
the angiotensin receptor blocker irbesartan. The 240mg daily dose was administered as a 120mg
capsule twice daily, which is an improved, more convenient transition from an 80mg capsule
administered three times a day in the previous Phase 2a study in patients with chronic kidney disease.
Teleconference at 11:00 (AEST) Wednesday 29 July
Conference ID: 10009009
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Ten patients were enrolled in the study, with seven patients meeting all pre-defined criteria required
for inclusion in the final analysis in accordance with the protocol. The primary endpoint for the study
was safety, as measured by the number and severity of adverse events and clinically significant
changes in the patient safety profile with the use of DMX-200 compared to placebo in participants
with FSGS who are receiving irbesartan. The preliminary safety findings show DMX-200 was generally
safe and well-tolerated, with no variation in the incidence or severity of adverse events between
treatment with DMX-200 or placebo. There were no patient withdrawals from the study and there
were no serious adverse events related to the drug reported.
The secondary endpoints were the proportion of patients who achieved a response during treatment
with DMX-200 compared to placebo as well as the percent change from baseline in 24-hour
proteinuria after 16 weeks of treatment with DMX-200 as compared to placebo. Importantly, 86%
(6/7) of patients demonstrated a reduction in proteinuria on treatment versus placebo, with an
average 29% change from baseline in 24-hour proteinuria compared to placebo following treatment
with DMX-200 based on protein/creatinine ratio (PCR) grouped analysis (an average reduction of
119mg/mmol (1052mg/g) proteinuria on DMX-200 versus an average reduction of 1mg/mmol
(8.84mg/g) proteinuria on placebo). Furthermore, 29% (2/7) of the patients achieved a >40%
reduction in proteinuria during treatment with DMX-200 compared to placebo.
Unlike other investigational drugs currently in development for FSGS, patients stayed on the standard
of care angiotensin receptor blockade. As a result, the reduction in proteinuria observed from DMX200 is in addition to any reduction in proteinuria expected from background therapy that would have
occurred prior to starting on DMX-200.
The Phase 2a study was a double-blind, randomised, placebo-controlled, crossover study designed to
evaluate the safety and preliminary signs of efficacy of DMX-200 in patients with FSGS who are
receiving a stable dose of standard of care, irbesartan. Patients must have been receiving irbesartan
for at least 12 weeks prior to being included in the trial. As previously announced, each participant
in the study received 16 weeks DMX-200 preceded or followed by 16 weeks placebo, separated by a
6-week washout period.
Whilst this initial Phase 2a study in patients with FSGS was of a short duration and was not powered
for statistical significance, it was designed to derive maximum insight from a small number of
patients, while retaining the ability for a flexible number of patients to complete the study. As such,
the study delivered encouraging data, which supports further development of DMX-200 in FSGS.
“I believe that the results of this Phase 2a FSGS study, together with the positive outcomes of the
Company’s prior Phase 2a study in Chronic Kidney Disease, further validates Dimerix’ lead candidate,
DMX-200, in sclerotic kidney diseases. The positive signals suggest that treatment with DMX-200 may
indeed result in clinically meaningful improvements in kidney function when added to the standard
of care in patients with FSGS. To have 86% of patients see a benefit on DMX-200 versus placebo in a
disease is very impressive. I am very excited at what this may mean for future studies in patients with
FSGS," commented Dr Hiddo Heerspink, Chair of the Dimerix Medical Advisory Board.
Investigator on the study and Head of the Melbourne Renal Research Group, Associate Professor
David Packham, commented “These are tremendous top line results and certainly very encouraging
for all FSGS sufferers. I look forward to further analysis over the next several weeks, which I expect
will equally be fascinating and informative.”
"We are very pleased with the top-line results from the study, which suggest DMX-200 could be a
significant advancement in the treatment of FSGS," said Dr Nina Webster, CEO and Managing Director
of Dimerix. "FSGS patients today face poor outcomes with limited medical options, and we continue
to progress our proposed development pathway forward that could deliver the first approved
pharmacologic treatment to the FSGS community. In the meantime, we will soon be reporting on the
larger diabetic kidney disease Phase 2 study that we hope will further support the growing evidence
of DMX-200 efficacy in treating kidney diseases."
It is anticipated that additional analyses of these FSGS data will be available in due course, following
availability of additional data and review by the Medical Advisory Board.
In the meantime, multiple patients from both the FSGS and the diabetic kidney disease study
continue on treatment with DMX-200 through the TGA’s Special Access Scheme following respective
study completion.
Dimerix has received Orphan Drug Designation for DMX-200 in both the US and Europe for the
treatment of FSGS. Dimerix established with the respective regulatory agencies that “the intention
to treat FSGS with DMX-200 was justified based on preliminary non-clinical data which showed a
reduction in the number of podocytes lost and an improvement in proteinuria.” Furthermore, as
stated by the respective regulatory agencies, the orphan designation indicates that “Dimerix has
provided sufficient justification that if approved, [DMX-200] is likely to be of significant benefit to
those affected by the condition” and that “[DMX-200] would provide a clinically relevant advantage
as an alternative to any currently marketed products”. Orphan designation also provides regulatory
and financial benefits to help bring DMX-200 to market in the US and Europe faster, including reduced
fees during the product development phase, protocol assistance from the regulatory authorities, and
7-year (US) and 10-year (Europe) market exclusivity following product approval.
Dimerix is also awaiting the Phase 2 study results in diabetic kidney disease, which completed dosing
in late July and has an expected data read-out in 4 - 6 weeks. In addition to the ongoing Phase 2
diabetic kidney disease study, the study in patients with COVID-19, and DMX-700 in Chronic
Obstructive Pulmonary Disease, Dimerix continues to undertake planning for its proposed global
Phase 3 pivotal program in FSGS.
For further information, please visit our website at www.dimerix.com or contact:
Dr Nina Webster, Dimerix Limited
Chief Executive Officer & Managing Director
Tel: +61 1300 813 321
E: [email protected]
Rudi Michelson
Monsoon Communications
Tel: +61 3 9620 3333
Mob: +61 (0)411 402 737
Conference call details
• Time: 11:00 (AEST) Wednesday 29 July
• Conference ID: 10009009
Access the call by pre-registration (preferred option) or by direct dial-in (delays possible):
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Authorised for lodgement by the Board of the Company