More than 20 years of passionate drug development
Oncopeptides AB (publ) was formed in year 2000 by some of Sweden’s leading cancer researchers and cancer research institutions. More than 20 years later the company is dedicated to providing patient access to its innovative alkylating drug.
Development of melflufen
The development of melflufen, stems from work originally undertaken by Dr Joachim Gullbo at Uppsala University, Sweden, on a molecule initially known as ‘J1’. Backed by funding from a leading venture capital firm, Industrifonden, and the Karolinska Institute, Oncopeptides embarked on a pre-clinical development journey.
Research collaboration in the US
Following an early study of the effectiveness of ‘J1’ in solid tumors, further work resulted in the development of melflufen. During this period, Oncopeptides established a research collaboration with the Harvard Medical School, Dana Farber Cancer Institute in the USA, a collaboration that has been instrumental to the development of melflufen.
Oncopeptides embarked on the phase 1/2 studies of melflufen in patients with late-stage multiple myeloma. These studies were financed by life sciences venture capital fund HealthCap, and Industrifonden together with members of the Board and Management.
Following the completion of the phase 1 of the phase 1/2 study in 2015, melflufen was granted Orphan Drug Designation in the US and EU, including extended marketing exclusivity.
Advancement of the Clinical Development Program
In 2016 the clinical research and regulatory engagement advanced. The phase 2 part of the phase 1/2 study was presented at the European Hematology Association Congress and several meetings were held with the US Food and Drug Administration (FDA) and European authorities. The design of the phase 3 study was approved by FDA through a Special Protocol Assessment process.
2022 – Making a difference for the patients
- The phase 3 OCEAN study is published in the Lancet Haematology
- Oncopeptides rescinds the voluntary withdrawal of Pepaxto in the US, based on additional analyses of survival data from OCEAN and other relevant studies
- CHMP issues a positive opinion recommending a full European approval of Pepaxti in combination with dexamethasone in triple class refractory multiple myeloma
- Oncopeptides carries out a directed share issue of approximately 435.6 million SEK
- The EU Commission approves Pepaxti for treatment of patients with relapsed refractory multiple myeloma
- FDA arranges a public hearing with the Oncologic Drugs Advisory Committee, ODAC. Most panel members state that OCEAN does not confirm a favorable benefit-risk profile in the current US indication
- Commercialization of Pepaxti in Europe is initiated, with Germany being the first market
- Clinical value of melflufen further confirmed through phase 3 LIGHTHOUSE study
- Market potential of Pepaxti in Europe I estimated to 1.5-2.0 Billion SEK
- Pepaxti is granted marketing authorization by MHRA in the UK
- Company enters into a renewed loan facility agreement of up to 30 million euro with the European Investment Bank
- FDA requests a withdrawal of the marketing authorization of Pepaxto in the US
2021 – Science is leading the way
- On February 26, FDA granted Pepaxto plus dexamethasone accelerated approval for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.
- A European business unit was established to prepare the commercialization of melflufen in EU, and in addition an Early Access Program was initiated to provide patients with no or limited treatment options early access to melflufen.
- Oncopeptides applied to the European Medicines Agency, EMA, for conditional marketing authorization of melflufen in the EU, based on the pivotal phase 2 HORIZON study in relapsed refractory multiple myeloma.
- Results from the phase 3 OCEAN study was published. Melflufen met the primary endpoint of superior progression free survival (PFS) in a head-to-head comparison with pomalidomide. However, the overall survival (OS) HR was 1.10 in favour of pomalidomide in the Intention to Treat population.
- Based on the worse OS data, the FDA requested a partial clinical hold of all clinical studies, which means pausing the enrollment of new patients. The FDA also issued a safety alert for Pepaxto and announced a public advisory expert meeting (ODAC) to discuss the future of Pepaxto.
- On October 22, the company decided to withdraw Pepaxto from the US market and return to being a Sweden based R&D company, dedicated to further develop the proprietary PDC platform.
- Consequently, a significant part of the clinical development program for melflufen was discontinued, the commercial business units in the US and Europe were closed and the Stockholm based organization was significantly reduced.
- The application to the European Medicines Agency, EMA, for a potential Marketing Authorization of melflufen in the EU will proceed.
2020 – Building a commercial stage biotech company
- Organizational development to prepare for commercial launch of melflufen in the US
- Transformation into a fully integrated global biotech company
- Investigational New Drug filing of OPD5, the second drug candidate based on the proprietary Peptide Drug Conjugate platform, to enable clinical development in the U.S.
- FDA grants priority review for the submission of a New Drug Application, seeking approval of melflufen in combination with dexamethasone for treatment of multiple myeloma
- Phase 3 OCEAN study is fully recruited. Top line results expected H1, 2021
- First patient enrolled in phase 1/2 Immunoglobulin Light Chain (AL) amyloidosis study, the first study to explore the effect of melflufen outside multiple myeloma
- A New Drug Application submitted to the FDA, for accelerated approval of melflufen in combination with dexamethasone for the treatment of multiple myeloma
- Commercial and R&D leadership strengthened, Marty J Duval becomes new CEO and Jakob Lindberg CSO
- Final data from the pivotal phase2 HORIZON study presented at virtual EHA-meeting
- A directed share issue raising approximately SEK 1,414 million (USD 144 M) in gross proceeds was completed
2019 – Preparing regulatory submission in the US
- Patient recruitment in the pivotal phase 2 HORIZON study completed
- New Interim Data in RRMM Patients with Extramedullary Disease from the pivotal phase 2 HORIZON study presented at International Myeloma Workshop
- Directed share issue of approximately SEK 727 million (USD 78 M) was completed.
- Decision to submit for Accelerated Approval in the US
- Additional patent protection was granted for melflufen in the US until 2033
- Updated data from the clinical studies O-12-M1, HORIZON and ANCHOR presented at multiple conferences including ASCO, EHA, SOHO
- A directed share issue of approximately SEK 546 million (USD 62 M) was completed
2018 – Expanding organization and raising ambitions
- Oncopeptides held a Capital Markets Day in New York
- First interim data from ongoing phase 2 combination ANCHOR study and updated Interim data from ongoing HORIZON study presented at the American Society of Hematology, ASH
- First patient included in the phase 2 BRIDGE study in RRMM patients with renal impairment
- Updated interim data from ongoing phase 2 HORIZON study with melflufen presented at the 23rd Congress of The European Hematology Association (EHA)
- The phase 1/2 combination ANCHOR study with melflufen started
- A directed share issue of SEK 314 million was completed
- Additional patent protection for melflufen was granted in Japan until October 2033
2017 – Public company with growing footprint
- Survival data from the phase 2 O-12-M1 study of melflufen and interim data from the ongoing phase 2 HORIZON study was presented at the 59th Annual Meeting of ASH
- Phase 3 OCEAN study with melflufen in RRMM patients started
- Additional patent protection for melflufen was granted in Europe until 2032
- Oncopeptides AB (publ) was listed on the Nasdaq Stockholm Stock Exchange
- Phase 2 HORIZON study, in late stage RRMM started
2016 – Regulatory dialogue evolving
- Approval of phase 3 protocol with MHRA and the FDA through Special Protocol Assessment
- Scientific meetings with European authorities and FDA regarding design of phase 3 study
- End of phase 2 meeting with FDA based on data in late-stage multiple myeloma patients
- Phase 2 part of the phase 1/2 study presented at the European Hematology Association
2014–2015 – Orphan status and promising phase 1 data
- Melflufen received Orphan Drug Designation in the EU and US
- Phase 1 part of the phase 1/2 study in late-stage multiple myeloma patients presented at the American Society of Hematology annual meeting
2012–2013 – Attracting key investors
- Phase 1/2 study initiated in late-stage multiple myeloma patients together with Harvard Medical School, Dana-Farber Cancer Institute, lead hospital
- HealthCap and Industrifonden, together with participation by the Chairman of the Board and the CEO, provided financing for the clinical phase 1/2 program in late-stage multiple myeloma patients
- Industrifonden acquired Karolinska Development’s stake in Oncopeptides
- HealthCap became a shareholder
2010–2011 – Strengthening leadership and research collaboration in US
- Jakob Lindberg was appointed CEO
- Research collaboration with Harvard Medical School – Dana-Farber Cancer Institute
- New melflufen freeze-dried pharmaceutical formulation developed and patented
- Development of new pharmaceutical formulation of melflufen
2009–2011 – First clinical studies
- Phase 1 study in solid tumors conducted at Uppsala Academic Hospital, Sweden
2003–2009 – Exploratory phase
- Pre-clinical development stage, funded by Industrifonden and Karolinska Development AB
2000 – Foundation of Oncopeptides
- Melflufen was patented
- Oncopeptides AB (publ) was founded to further develop drug candidates, including melflufen