Oncopeptides is a science and data driven company. We are innovative and curious, and committed to bringing innovation to patients with rare hematological diseases. We are passionate to make a difference for patients who have an urgent need for better treatment options.
Oncopeptides is developing innovative drug candidates for difficult to treat haematological diseases. The development is built on our two unique technology platforms for Peptide Drug Conjugates (PDC), and Small Polypeptide based Innate Killer Engagers (SPiKE), and allows us to build a robust pipeline, with potential to expand into new indications.
PDC – A true innovation
The Peptide Drug Conjugates (PDC) compounds are composed to enable efficient distribution in the body, enabling a wide therapeutic window (i.e. a wide margin between effective doses and doses that give unacceptable side effects), and an optimized benefit risk profile. The PDCs are designed around two components: a peptide carrier and a cytotoxic payload. The PDCs are lipophilic which allows a rapid diffusion into cells. The peptide carrier utilizes the altered metabolism of cancer cells to hydrolyze PDC into active hydrophilic metabolites, which lead to an enrichment in cancer cells.
get more information on the PDC technology platform
Melflufen
Multiple Myeloma (RRMM)
The first PDC with an alkylating payload
Melflufen is the first PDC with an alkylating payload. The drug utilizes peptidases and esterases that are overexpressed in multiple myeloma cells, to release a toxic payload inside cells, leading to DNA damage ang killing of cancer cells. Melflufen has demonstrated effect on cancer cells that have lost the important tumor suppressor TP53. It has been developed for relapsed refractory multiple myeloma, through a comprehensive preclinical and clinical program, that lead to accelerated approval by the FDA in US in February 2021, and full marketing approval by EMA in August 2022 and MHRA in UK in November 2022 (see regulatory status of melflufen here).
OPD5
Hematologic or solid tumors
OPD5 – follow-up PDC
OPD5 is a new chemical entity adapted from melflufen. It was initially aimed at high dose treatment for myeloablation ahead of stem cell transplant but can be formulated for conventional dose in either multiple myeloma or other hematologic or solid tumors. OPD5 is a Phase 1 ready asset. The first Phase 1/2 study was approved by regulatory authorities and ethics committees but was terminated before including the first patient.
OPDC3
Hematologic or solid tumors
OPDC3 – Next generation PDC
OPDC3 builds on the clinical experience of melflufen. Like melflufen, OPDC3 generates an enrichment of alkylating payloads in cancer cells. In addition, it is designed to prevent the cytotoxic payload to be transported out of cancer cells, a common mechanism of cancer cells resistance to cytotoxic agents. These unique properties may translate into an even more effective and well tolerated therapeutic option. This warrants further evaluation in clinical studies in either hematologic or solid tumors. OPDC3 is currently in late preclinical development.
Gliopep
Glioblastoma
Glioblastoma is the most aggressive form of brain cancer, characterized by rapid growth, invariable relapse, and lack of curative treatment options. With a median survival of only 12–15 months, there is a profound unmet medical need for therapies capable of crossing the blood-brain barrier (BBB) – the primary obstacle that causes most drugs to fail in this indication. Agents from the Oncopeptides’ PDC platform can bypass this barrier due to their lipophilicity and enter cells freely. This novel Mode of Action, results in cytotoxic activity directly within tumor cells.
Oncopeptides has achieved key regulatory agreement with both the Swedish and Norwegian Medical Products Agencies (Läkemedelsverket, MPA and Direktoratet for medisinske produkter, DMP) regarding the design of its planned “Window-of-Opportunity” (WoO) study evaluating a Peptide Drug Conjugate (PDC) in glioblastoma. The study will be conducted in Norway, where DMP has granted fast-track designation.
The SPiKE Technology Platform
This research utilizes the immune system to fight cancer. It has evolved from targeting the T-cells, such as CAR-T therapy or bi-specific antibodies, to targeting the natural killer cells, or NK-cells. This may reduce the often dose-limiting side-effects, such as cytokine release syndrome (CRS) and PNS/CNS symptoms, whilst achieving similar levels of clinical efficacy, even after previous exposure to and relapse after T-cell directed therapies. Oncopeptides has developed a proprietary technology platform for Small Polypeptide based innate Killer Engagers (SPiKE).
The SPiKEs are designed to bind to both NK-cells and specific targets on various cancer cells. SPiKEs thereby induce a strong NK cell mediated killing of tumor cells that is strictly dependent on presence of the tumor associated antigen. The relatively small molecule facilitates tight interactions between NK cells and cancer cells, a beneficial distribution profile and the ability to tailor exposure to maintain immunological functionality by allowing transient periods of rest/non-activation.
The first compound selected from this technology platform is currently in late preclinical development for both solid tumors and hematologic malignancies.
In September 2022, the Company received a research grant from Sweden’s Innovation Agency, to develop pre-clinical proof of concept for a novel synthetic small polypeptide for the treatment of multiple myeloma.
The project has received a financial grant from the Eurostars 3-program, it is co-financed by the EU’s research and innovation program “Horizon Europe” and is driven by an international research consortium. This includes world-leading expertise from the department of Cancer Immunology at Oslo University Hospital, Norway, Pharmatest Services Ltd in Turku, Finland, and Oncopeptides, together with our collaborator the Royal Institute of Technology in Stockholm (KTH), where the technology originally stems from.
NK+BCMA
RRMM
The first PDC with an alkylating payload
Melflufen is the first PDC with an alkylating payload. The drug utilizes peptidases and esterases that are overexpressed in multiple myeloma cells, to release a toxic payload inside cells, leading to DNA damage ang killing of cancer cells. Melflufen has demonstrated effect on cancer cells that have lost the important tumor suppressor TP53. It has been developed for relapsed refractory multiple myeloma, through a comprehensive preclinical and clinical program, that lead to accelerated approval by the FDA in US in February 2021, and full marketing approval by EMA in August 2022 and MHRA in UK in November 2022 (see regulatory status of melflufen here).
NK+XX
Hematologic or solid tumors
The first PDC with an alkylating payload
Melflufen is the first PDC with an alkylating payload. The drug utilizes peptidases and esterases that are overexpressed in multiple myeloma cells, to release a toxic payload inside cells, leading to DNA damage ang killing of cancer cells. Melflufen has demonstrated effect on cancer cells that have lost the important tumor suppressor TP53. It has been developed for relapsed refractory multiple myeloma, through a comprehensive preclinical and clinical program, that lead to accelerated approval by the FDA in US in February 2021, and full marketing approval by EMA in August 2022 and MHRA in UK in November 2022 (see regulatory status of melflufen here).
What do the phases of clinical trials mean?
Intellectual property and orphan drug designation
Patents and intellectual property
Oncopeptides has an active patent strategy, spanning from protection of the early pre-clinical portfolio to the commercial product Pepaxti. Part of the strategy encompasses protection in all major geographic markets, including the U.S., Europe, Canada, Japan, and China.
The company’s intellectual property portfolio of registered intellectual property rights consists of granted patents, patent applications in different stages, as well as trademarks. In addition, the company has important rights to clinical data and significant unregistered rights including trade secrets.
Melflufen is protected by different families of granted patents, including formulations, manufacturing processes and methods for treatment.
The market authorizations in EU and UK during the second half of 2022, may enable us to extend the patent protection for up to five years via the Supplementary Protection Certificates (SPCs), which have now been filed. The possibility to extend the patent term in selected countries will be investigated if new markets are entered.
Since the establishment of the drug development lab in 2020, pre-clinical R&D efforts have been intensified, resulting in an increase in filed applications over the last couple of years. These applications, some yet unpublished, provide protection of new innovations in the pre-clinical pipeline, including both the Peptide Drug Conjugate (PDC) platform and the Small Polypeptide based Killer Engagers (SPiKE) platform.
To secure high quality IP protection, Oncopeptides has a long-lasting relationship with Abel & Imray patent and trademark attorneys and their international network of attorneys around the world.
Orphan designation
Orphan designation is granted to treatments that target rare diseases where it’s unlikely that the costs of developing such treatments would be recovered through sales. An orphan designation may provide benefits such as market exclusivity but is only given to drugs that are proven better than all available therapies in that indication.
Melflufen was initially granted orphan designation in both the US and Europe, but at the time of marketing authorization approval of Pepaxti in EU, in Aug 2022, the company decided to withdraw Pepaxti from the European Union register of orphan medicinal products as other products have become available.
When relevant, orphan designation may also be applied for new compounds in the portfolio.