FM November2020 Digital P

The Patient-Reported
Outcomes data from the
ATLAS-2M study showed
high levels of treatment
satisfaction and acceptance,
with 98% (n=300/306)
of participants who were
randomised to receive an oral
lead-in followed by once every
2-months dosing preferring
treatment once every
2-months compared to daily
oral treatment (oral lead-in).
Results indicate
that administration frequency
and convenience were the
most common reasons for
preferring treatment every
2-months.
RHB-204 is
orphan drug
to treat NT
mycobacteria
disease
RedHill Biopharma
announced that the US
FDA has granted Orphan
Drug Designation to RHB-
204 for the treatment of
nontuberculous mycobacteria
(NTM) disease.
A phase 3 study to
evaluate the safety and
efficacy of RHB-204 in
patients with pulmonary
NTM infections caused by
Orphan drug status to
sacituzumab for glioblastoma
Immunomedics said the US FDA has
granted sacituzumab govitecan-hziy
(Trodelvy) orphan status for the treatment
of adult and paediatric patients with
glioblastoma.
Sacituzumab is an antibody-drug
conjugate (ADC) directed against Trop-2,
a cell-surface protein expressed in many
solid cancers. It binds to Trop-2 and
delivers the anti-cancer drug, SN-38, to kill
cancer cells.
Immunomedics has multiple ongoing
studies in triple-negative breast cancer,
metastatic urothelial cancer, hormone
receptor-positive/human epidermal growth
factor receptor 2-negative metastatic
breast cancer, and metastatic non-small
cell lung cancer, either as a monotherapy
or in combination with other agents.
Mycobacterium avium Complex
(MAC) is planned to be
initiated soon in the US.
The multi-centre,
randomised, double-blind,
placebo-controlled, parallelgroup
phase 3 study is
planned to be conducted at up
to 40 sites across the US and
aims to enroll 125 patients,
randomised at a 3:2 ratio to
receive either RHB-204 or
placebo.
Currently, there is no FDAapproved
first-line standardof-care
therapy for NTM.
RHB-204 is a fixed-dose
oral capsule containing a
combination of clarithromycin,
rifabutin, and clofazimine.
Immunotherapy
for treating MM
gets Orphan
drug status
Glycostem Therapeutics has
received the FDA’s Orphan
Drug Designation (ODD) for
the treatment of multiple
myeloma (MM) patients with
its investigational product
oNKord.
oNKord is a first-generation
off-the-shelf natural killer
(NK) cellular immunotherapy
product. Over the coming
months, AML patients will
receive this form of treatment
as part of a phase I-IIa
(pivotal) trial in AML. A phase
II trial for MM patients is
expected to start in 2021.
“Since 2012 we have
been pioneers in the field of
developing and manufacturing
off-the-shelf natural killer cell
therapy products for cancer
treatment. In 2020 we’re
entering a new and exciting
phase,” says Troels Jordansen,
CEO of Glycostem, in a
statement.
MM is the second most
common blood cancer,
accounting for 15% of blood
cancers, and 2% of all cancers.
MM occurs in infection-fighting
plasma cells found in the bone
marrow
Orphan drug
status to APG-
115 & APG-1252
to treat AML and
SCLC
Ascentage Pharma
announced that the US
FDA has granted two Orphan
Drug Designations (ODDs)
to two of the company’s
apoptosis-targeting assets: the
MDM2-p53 inhibitor, APG-115,
for the treatment of acute
myeloid leukaemia (AML);
and the Bcl-2/Bcl-xL inhibitor,
APG-1252, for the treatment of
small-cell lung cancer (SCLC).
APG-115 is an orally
administered, selective, small-
November 2020 / FUTURE MEDICINE / 41