Oncopeptides – 20 years of passionate drug development

Oncopeptides AB (publ) was formed in 2000 to develop anti-cancer drugs based on research by some of Sweden’s leading cancer researchers and cancer research institutions.

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.

Major milestones

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