Orbsen in fundraising efforts to drive Phase II and potentially Phase IIb mesenchymal stromal cell therapy plans; Phase IIb start likely in mid to late 2021, exec says
Published Date:20 Apr 2020
- Phase IIb could include COVID-19 focus depending on results
- Orbsen in talks with and open to CMOs to manage downstream manufacturing
Orbsen Therapeutics is in the midst of a fundraising round to support its Phase
II mesenchymal stromal cells (MSC) studies in multiple indications, and could potentially
expand the round to cover a planned Phase IIb trial, said CEO Larry Couture.
Couture did not comment on the exact amount being raised, but said while the ongoing
Phase II funding goal is modest, the next round will be larger and likely be in the tens of
millions. In the past, Orbsen has looked to retail investors, and received nondilutive
funding for its research and development program through several grants, but is only now
transitioning into seeking institutional investors and strategic partners. Hence, Couture
declined to classify the fundraising efforts as part of a particular series.
Orbsen is working with a third-party financial services firm to put together the expanded
round, he said. The economic collapse following the current pandemic has had a negative
effect on its fundraising ability, but Orbsen could be back at discussions in a few months
and branch out into Europe and Asia, said Couture. The fundraising plans are not etched
in granite and while the company plans to take only one study through to Phase IIb, if
that is successful, it could move to a second or third indication, said Couture. The
company is not reinventing its fundraising strategy specifically because of COVID-19, he
added.
ORBCEL clinical strategy
The Galway, Ireland-based company has five programs, including a recently initiated
one in patients with acute respiratory distress syndrome (ARDS) due to COVID-19. All
are based on the platform MSC therapy ORBCEL, derived from umbilical cord tissue. In
addition to the Phase I/II ORBCEL ARDS study, dubbed as REALIST (NCT03042143),
Orbsen has ongoing clinical trials in diabetic foot ulcers, diabetic kidney disease (DKD),
and primary sclerosing cholangitis, and a fifth study in autoimmune conditions in the
works. Orbsen is not positioning itself as a COVID-focused company but rather a stromal
cell-company for which COVID-19 is one of the indications, said Couture.
Even though the Phase II REALIST trial plans to enroll 60 patients, Couture talked about
the possibility of building in an interim analysis point in the study, such that if the results
are heavily skewed in favor of the product after 35 patients have enrolled, the data could
be unblinded. However, running the trial to its full course would objectively make the
study stronger, he said. Orbsen plans to wait for all trials to read out by mid-2021 or
YE21 and pick the indication that may be best to pursue for a Phase IIb study, said
Couture.
Even though one of Orbsen’s DKD trial sites in Bergamo, Italy — one of the epicenters
of the COVID-19 epidemic — was interested in conducting a study to evaluate ORBCEL
in COVID-19 patients, the region was imploding with cases and unable to do so, said
Couture. Northern Ireland, where one of the REALIST sites is recruiting, has not had a
crush of COVID cases yet, but the trial enrollment will be manageable and should
complete in the next 6–9 months, said Couture.
Scaling up ORBCEL manufacturing protocols
In terms of manufacturing, the company plans to manage the upstream portion of
ORBCEL production since it involves trade secrets, but is in talks with CMOs to do a
scale-up of their downstream manufacturing capabilities, said Couture. Through the
Phase IIb, Orbsen will manage the entire manufacturing process but it plans to bring a
CMO online during scale up-operations during the Phase IIb plans, said Couture. The
company would be open to CMOs reaching out to Orbsen for this, but the company has
already reached out to relevant ones in Europe and the UK, and two that have operations
in the US, he added.
Orbsen does plan to conduct part of the Phase IIb study in the US, and may even consider
opening a parallel study in the US due to the ongoing REALIST program, said Couture.
However, in terms of manufacturing for a potential study in the US, the FDA does not
allow the import of cells from the UK due to a rule established in the 1990s due to mad
cow disease. This issue will have to be resolved before initiating a study, said Couture.
Orbsen’s platform has identified markers on the stromal cells subpopulation among cells
that have immunomodulatory activity, which does not include fibroblasts, preadipocytes
and other cells that are usually found in MSC preparations, said Couture. This allows the
platform to get a near-pure, well-characterized population of cells. Furthermore, using
umbilical cords as a source allows the donors to all be age-matched as opposed to bone
marrow, he added. Also, the fact that the cell therapy is not genetically engineered makes
it favorable to regulators, who want to fast track such cell therapies, said Couture.
by Manasi Vaidya in New York
Region: Europe; North America
Country: Ireland; United States
Topic: CMO; Financing; Product Development Exec Updates
Company Name: Orbsen Therapeutics Ltd
Indication: Coronavirus Disease 2019 (COVID-19)
Drug(s)/Molecule(s): Stem Cell Therapy for Gastrointestinal, Infectious and Respiratory Disease
Trial Identifier Trial Phase Trial Status
GDCT0270827 Phase I/II Ongoing, recruiting
GDCT0385597 Phase II Planned
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