STOCKHOLM, June 30, 2020 /PRNewswire/ -- Oncopeptides AB
(publ) (Nasdaq Stockholm: ONCO) today announces that the Company
submits a New Drug Application (NDA) to the U.S. Food and Drug
Administration, FDA, for accelerated approval of melflufen (INN
melphalan flufenamide) in combination with dexamethasone for the
treatment of adult patients with multiple myeloma whose disease is
refractory to at least one proteasome inhibitor, one
immunomodulatory agent and one anti-CD38 monoclonal antibody (i.e.,
triple-class refractory multiple myeloma patients).
Melflufen is the lead candidate coming out of the Oncopeptides'
proprietary PDC-platform. The product is a first-in-class
aminopeptidase-targeting peptide-drug conjugate that rapidly
delivers an alkylating payload into tumor cells. The
submission is based on the results from the pivotal phase 2 study
HORIZON, evaluating intravenous melflufen in combination with
dexamethasone in patients with relapsed refractory multiple myeloma
(RRMM).
The results from the HORIZON study demonstrates that melflufen
in combination with dexamethasone, has a potential to provide a
therapeutic option for patients with RRMM that are hard to treat
and have a poor prognosis, including patients with triple class
refractory myeloma and patients with extramedullary disease (EMD).
The responses in the HORIZON study were durable and often deepened
with prolonged treatment, suggesting that patients could benefit
from staying on treatment for as long as possible.
"I am very proud and humbled by the organizations ability to
timely submit the NDA for accelerated approval of melflufen. This
is a major milestone for Oncopeptides and is a result of dedicated
research and development activities throughout the last decade",
says Jakob Lindberg CEO of Oncopeptides. "I would like to express
my sincere gratitude to all patients, co-workers, investigators and
shareholders who have provided relentless support to enable a novel
treatment option for a fast-growing patient population with a
significant unmet medical need".
Following the submission to the FDA Oncopeptides will initiate
an Expanded Access Program (EAP) in the U.S. to enable melflufen
treatment for patients with a significant unmet medical need.
The information in the press release is information that
Oncopeptides is obliged to make public pursuant to the EU Market
Abuse Regulation. The information was submitted for publication,
through the agency of the contact person above, on June 30, 2020 at 08.00 (CET).
About the HORIZON study
In total 157 multiple myeloma patients have been enrolled and
evaluated in the pivotal phase 2 HORIZON study. The study was fully
recruited in October 2019, the final
data cut was made on January
14th and the final data was presented at the
EHA meeting in June. The patients in the study were refractory to
pomalidomide and/or daratumumab after failing on immunomodulatory
drugs (IMiDs) and proteasome inhibitors (PIs). The HORIZON study
population includes subgroups of patients who were triple-class
refractory and/or had extramedullary disease and/or had cytogenetic
high-risk features.
Summary of results
End
Points
|
Intention to Treat
(n=157)
|
Triple Class
Refractory (n=119)
|
Extra Medullary
Disease (n=55)
|
Overall Response Rate
(ORR)
|
29%
|
26%
|
24%
|
Median Progression
Free Survival (PFS))
|
4.2 months
|
3.9 months
|
2.9 months
|
Median Overall
Survival (OS)
|
11.6
months
|
11.2
months
|
6.5 months
|
|
|
|
|
Responding
patients
|
n=45
|
n=31
|
n=13
|
Median Duration of Response
(DOR)
|
5.5 months
|
4.4 months
|
5.5 months
|
Median Progression Free
Survival (PFS)
|
8.5 months
|
8.5 months
|
17.3
months
|
All data were confirmed by the Independent Review Committee
(IRC), with only minimal discordance.
About melflufen
Melflufen (INN melphalan flufenamide) is a first-in-class
aminopeptidase-targeting peptide-drug conjugate that rapidly
delivers an alkylating payload into tumor cells. Melflufen is
rapidly taken up by myeloma cells due to its high lipophilicity and
is immediately hydrolyzed by peptidases to deliver an entrapped
hydrophilic alkylator payload. Peptidases play a key role in
protein homeostasis and feature in cellular processes such as
cell-cycle progression and programmed cell death. In vitro,
melflufen is 50-fold more potent in myeloma cells than the
alkylator payload itself due to the increased intracellular
alkylator concentration. Melflufen displays cytotoxic activity
against myeloma cell lines resistant to other treatments, including
alkylators, and has also demonstrated inhibition of DNA repair
induction and angiogenesis in preclinical studies.
Expanded access policy
The preparations for an Expanded Access Program (EAP) in the US
are well underway and the program will open in Q3. Oncopeptides
encourages awareness of and participation in its clinical trials
and believes that participating in clinical trials is a good way
for patients to access investigational drugs prior to regulatory
approval. Individuals interested in participating in clinical
trials for melflufen may visit
https://www.oncopeptides.se/en/our-clinical-trials-summary/ for
information about ongoing clinical trials. Patients are encouraged
to consult their physician regarding the possibility of
participating in one of the ongoing clinical trials.
About Oncopeptides
Oncopeptides is a pharmaceutical company focused on the
development of targeted therapies for difficult-to-treat
hematological diseases. The company is focusing on the development
of the lead product candidate melflufen, a first-in-class
aminopeptidase-targeting peptide-drug conjugate that rapidly
delivers an alkylating payload into tumor cells. Melflufen is
in development as a new treatment for the hematological malignancy
multiple myeloma and is currently being tested in multiple clinical
studies including the pivotal phase 2 HORIZON study and the ongoing
phase 3 OCEAN study. Oncopeptides' headquarters is in Stockholm, Sweden with its U.S. headquarters
in Boston, Mass. The company is
listed in the Mid Cap segment on Nasdaq Stockholm with the ticker
ONCO.
More information is available on www.oncopeptides.com.
For more information, please contact:
Jakob Lindberg
CEO, Oncopeptides AB
E-mail: jakob.lindberg@oncopeptides.com
Telephone: +46-8-615-20-40
Rein Piir
Head of Investor Relations at Oncopeptides
E-mail: rein.piir@oncopeptides.com
Cell phone: +46-70-853-72-92
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PR - Oncopeptides
submits a New Drug Application to the FDA
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