Italian biotech MolMed has been given the backing of European regulators for its new drug that can help blood cancer patients live longer after receiving stem cell transplants to help with their disease.
This makes Zalmoxis the first immunogene therapy as a patient-specific, adjunctive treatment in haplo-identical haematopoietic stem-cell transplantation (HSCT) for adult patients with high-risk blood cancers.
But the nod comes with a condition that the biotech provide results from an ongoing, comparative Phase III trial looking at disease-free survival rates.
Until these full data are available, the European regulator said it would “review the benefits and risks of Zalmoxis annually to determine whether the conditional marketing authorization can be maintained.”
The company had sought FDA approval but back in 2014 its application for the same license had been refused, with the regulator suggesting that the application be resubmitted when new clinical data come in.
Zalmoxis is a new type of therapy based on genetically engineering donor immune system T cells to carry an inducible “suicide gene."
When given to patients following HSCT from partially compatible donors (i.e., haploidentical HSCT), these cells create an anti-leukaemia effect by killing post-transplant immunosuppression prophylaxis and inducing a rapid immune reconstitution.
The suicide gene also helps to stop Graft versus Host Disease (GvHD)--the most significant and serious adverse event in haplo-identical transplantation, which is caused by the genetic disparity between patient and donor.
Trials have shown that Zalmoxis can significantly increase long-term survival--regardless of disease status at transplant--according to MoIMed.
In a recent trial, 45 patients treated with the drug saw a survival rate of 49% after one year with Zalmoxis, compared with the data from databases of 140 patients who had undergone haploidentical HSCT without the drug, which saw a survival rate of 37%.
An allogeneic haemopoietic stem cell (HPC) transplant involves matching a patient’s tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related or unrelated donor. HLA proteins are found on all cells of our body and are the main way the immune system tells the difference between our own cells and foreign cells.
The closer the HLA match between a donor and recipient, the greater the chance a transplant will be successful.
But if the HLA match is not close enough, the donor’s immune system, which accompanies the donated stem cells, recognizes the HLA mismatch, and will attack the recipient’s tissues. This process is known as GVHD.
Around 70% of people with a haematological malignancy or bone marrow failure syndrome who need an allogeneic transplant have a HLA-identical sibling or unrelated donor available.
For patients who need a stem cell transplant but do not have a HLA-matched related or unrelated donor, recent medical advances have made possible the use of a partially matched or haploidentical related donor. A haploidentical related donor is usually a 50% match to the recipient and may be the recipient’s parent, sibling or child.
In the EU, there is around 1,300 high-risk haematological malignancies patients per year who undergo haplo-identical HSCT, growing 30% every 12 months, according to the biotech.
On top of this, almost 11,000 patients with high-risk haematological malignancies are candidates for allogeneic transplant but lack a fully compatible donor--for whom Zalmoxis might also become a new option.
Riccardo Palmisano, CEO of MolMed, said: “This tremendous achievement is a real turnaround for MolMed, moving this cutting-edge innovative biotech company to the next step of its journey: the commercialization of its proprietary products, thus solving patients’ unmet clinical need and generating revenues.
“Furthermore, having Zalmoxis manufactured at MolMed’s facility, this positive opinion reinforces our well-known and unrivalled GMP manufacturing capabilities on a commercial scale, thus promoting new potential industrial partnership opportunities. Zalmoxis technology platform could be leveraged to find new applications in the future, though the path for potentially additional revenues from sales and commercial partnerships already opens today.”
This tops off a good six months for the company, which saw its partner GlaxoSmithKline ($GSK) gain European approval for the two companies’ gene therapy Strimvelis for the incredibly rare “bubble boy syndrome” SCID. MolMed’s facility is currently the only place where the drug, which is tailored to each patient it treats, can be made.
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