Providing clinical benefit to patients
2022 was a landmark year for Oncopeptides. We received a full marketing authorization for Pepaxti in Europe. This enables us to bring hope to patients with multiple myeloma who despite the introduction of novel therapies need an accessible treatment option that offers robust efficacy, reduces treatment burden, and maintains quality of life.
Confirmatory OCEAN study
2022 kicked off with the publication of data from the confirmatory phase 3 OCEAN study in The Lancet Haematology. The study was a randomized head-to-head study which evaluated the efficacy and safety of melflufen and examethasone versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma (RRMM). Melflufen met the primary
endpoint of superior Progression Free Survival (PFS) in the Intention to Treat (ITT) population.
The OCEAN study showed numerically shorter overall survival (OS) for Melflufen. We analyzed additional studies to understand the complexity
of the results and shared the outcome with the regulatory agencies. The data demonstrated a true survival heterogeneity. Young transplanted patients
in the comparator arm had a much larger clinical benefit than in the melflufen arm. Older, nontransplanted patients had a larger clinical benefit in the melflufen arm. The European Medicines Agency (EMA), who had an ongoing assessment of the marketing authorization application for Pepaxti based on the HORIZON study, decided to include OCEAN as a confirmatory study.
Full marketing approval in Europe
By the end of the second quarter 2022 the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP),
unanimously recommended the European Commission to grant a full marketing approval of Pepaxti in the EU with no post approval commitments. EMA emphasized that Pepaxti has a positive benefit risk profile in triple
class refractory multiple myeloma patients and concluded that no safety signals were identified. This was excellent news for patients, shareholders, and employees.
On the heels of the positive opinion from CHMP, Oncopeptides successfully carried out a directed share issue, raising approximately SEK 435.6 M at market value. The full market approval in the EU for Pepaxti marked a turnaround and set the scene for a new and exciting phase as a commercial stage biotech company. We are now entering a European market with potential annual revenues of around SEK 1.5–2.0 bn for Pepaxti, based on a potential type 2 variation label in the EU, which may enable treatment in one earlier treatment line. During the autumn, we also received a full approval in the UK.
Commercialization of Pepaxti initiated
Pepaxti was granted marketing authorization on August 18, and in mid-October Germany became the first country in Europe to launch the drug. Pepaxti has been well received among those physicians that we have engaged with. We have managed to attract a patient focused team determined to bring new science to the benefit of patients. Our team has a strong network with key opinion leaders and specialists in multiple myeloma, excellent scientific knowledge and business acumen.
Within multiple myeloma, there is a clear unmet need that is not addressed by the current expensive treatment intensive products, that primarily are suitable for younger patients. Pepaxti represents a true innovation in drug design and is the first peptide drug conjugate PDC) with an alkylating payload. It offers an improved clinical experience and an option for patients who need additional treatment alternatives without detriment to their quality of life.
LIGHTHOUSE study supports clinical benefit
Later in October we presented encouraging data from the phase 3 LIGHTHOUSE study, a randomized combination study with daratumumab, that further supports the clinical benefit of melflufen in patients with relapsed refractory multiple myeloma, as well as the benefit in multiple myeloma patients with a treatment history with no or a successful stem cell transplant. This is in line with the recent marketing authorization in Europe. During the fall the US Food and Drug Administration (FDA) arranged a public hearing with the Oncologic Drugs Advisory Committee, ODAC, to discuss the benefit-risk profile of Pepaxto in the current US indication. We have a dialogue with the FDA regarding the regulatory challenges in the US, however no formal decision has been made.
Early pipeline advancing
We have continued to develop new drug candidates built on our proprietary technology platforms and have advanced our pre-clinical portfolio. One of the most exciting projects is SPiKE, a first in class Natural Killer (NK) cell engaging immunotherapy. It has superior immune cell activation. The project has been acknowledged by the Sweden’s Innovation Agency, as one of the most innovative projects, all categories. The NK Engage project is led by Oncopeptides and driven by a research consortium of world-leading expertise on NKcell engagers. The first pre-clinical data was presented at the Annual American Hematology Meeting in December.
Since starting as CEO, I have been impressed by the driven, experienced team that we have and excited about the opportunity we have with Pepaxti. As we move forward, we have clear corporate goals which are aligned to our value drivers. Completing our team in Germany to ensure that physicians will understand the benefits of the product and prescribe Pepaxti. Broaden our European footprint to deliver access across Europe. Progress our pipeline with the PDC and SPiKE platforms. Ensure that we have excellent financial discipline to target our investment and maximise our cash runway. I am excited about working toward the future as a sustainable business that delivers value both for patients and our shareholders.
Stockholm, April 18, 2023
Monica Shaw, CEO