CHMP issues positive opinion on Type II variation to extend the therapeutic indication of Pepaxti based on OCEAN trial results

September 14, 2023

Stockholm — September 14, 2023 — Oncopeptides AB (publ), a biotech company focused on difficult-to-treat cancers, today announces that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), has, following their scientific assessment, adopted a positive opinion on Oncopeptides’ application for earlier lines of treatment for patients with relapsed, refractory multiple myeloma (RRMM). The opinion from the CHMP will now be sent to the European Commission for a final decision.

Based on findings from its OCEAN trial, Oncopeptides submitted, on 28 Nov 2022, an application to extend the therapeutic indication of Pepaxti to the treatment of adult patients with multiple myeloma who have received at least two prior lines of therapies, whose disease is refractory to lenalidomide and the last line of therapy.

With their opinion, CHMP recommends that the use of Pepaxti could be expanded to earlier lines of treatment and also peripheral administration, meaning delivery of treatment through peripheral rather than central veins, a less invasive way of administration.

Oncopeptides will as a next step assess the current market dynamics of the rapidly evolving multiple myeloma landscape, including the competition in different lines of treatment and the effect that extending Pepaxti into earlier lines of treatment would have on Oncopeptides´ ability to receive a reimbursed price that reflects its innovation.

“The positive opinion from the CHMP further validates the scientific data on the efficacy, safety and increased quality of life that Pepaxti is able to bring to patients,” says Sofia Heigis, CEO of Oncopeptides. “While we are convinced that our drug best serves patients in later lines of treatment where the unmet need and our chances to receive a price that reflects our innovation are high, we will closely evaluate the CHMP opinion and our potential next steps, always keeping value for patients and our shareholders as paramount priorities.”

Investor Q&A

What is this?

We are announcing that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), has issued a positive opinion of Oncopeptides’ application to allow access to earlier lines of treatment for patients with relapsed, refractory multiple myeloma (RRMM).

Is this recommendation final?

No, the opinion will be sent to the European Commission, who will make the final decision. They normally follow the recommendations of CHMP.

What does a positive CHMP recommendation mean for the market potential of Pepaxti?

It means the drug can be prescribed to a broader group of patients. It does, however, not say anything about at what price the drug would be sold in a new indication, which makes estimating market potential difficult, especially since the multiple myeloma landscape is complex and ever evolving.

What is the OCEAN study?

The phase 3 OCEAN study is a global, randomized, head-to-head, open-label study, evaluating the efficacy and safety of melflufen and dexamethasone, versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma who have received 2-4 prior therapies.

Find more information about OCEAN here.

How will this affect the company’s revenue projections?

It is too early to say, as a next step Oncopeptides will assess the current market dynamic, including the competitive landscape in different lines of treatment and the effect that extending Pepaxti into new lines of treatment would have on our chances to receive a price that reflects our innovation.

We see no reason to adjust our estimated market potential for Pepaxti based on this event.

Added September 15: Did you not start this analysis until now, didn´t you plan for a positive recommendation?

Yes we have planned for it. However, there are still unknown factors, including onging price negotiations in Germany for Pepaxti, that are relevant. We aim to have communicated a clear path forward within the coming month

How soon could Pepaxti become prescribed for a new indication?

After the European Commission (EC) gives its final approval. We would like to stress that no decisions on expanding the indication have been made at this time and that we believe that Pepaxti creates its optimal value in later lines of therapy where there is a high unmet need and a better chance on receiving a price that reflects our innovation. Any decision on a next step will be made in the best interest of our patients and shareholders in mind.

What is the indication that CHMP has issued a positive opinion on?

Pepaxti is indicated, in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least two prior lines of therapies, whose disease is refractory to lenalidomide and the last line of therapy.

For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

What do you mean when you speak of “lines of treatment”?

A line of therapy means a planned treatment regimen, that may consist of one therapy or a sequence of therapies. A new line of therapy starts when treatment is modified to include new agents as a result of progression or toxicity or when a planned period of observation is interrupted by the need for additional treatment of multiple myeloma.

Where can I find more information?

For additional information, reach out to David Augustsson, Director of Corporate Affairs, david.augustsson@oncopeptides.com.