Fostrox + Lenvima® holds promise of greatly improved outcomes for advanced liver cancer patients, reveals new data from Medivir at ESMO GI

Rhea-AI Summary

Medivir announced promising results from its Phase 1b/2a trial of fostrox combined with Lenvima® for treating advanced hepatocellular carcinoma (HCC), a challenging condition with effective treatments. The trial showed a 24% overall response rate (ORR) and a median time to progression (TTP) of 10.8 months, significantly better than the current standard treatments. One patient has continued treatment for 22 months. The data supports fostrox’s selective action on tumor cells without harming normal liver function, indicating a favorable safety profile. These findings were presented at the ESMO GI Cancers Congress in Munich.

Medivir is now proceeding with study feasibility and finalizing protocols to open an Investigational New Drug (IND) application in the US by H2 2024. HCC accounts for over 80% of liver cancer cases globally, with approximately 660,000 new cases annually and a five-year survival rate under 20%.

Positive

• 24% overall response rate (ORR) in the trial for fostrox + Lenvima®.
• Median time to progression (TTP) of 10.8 months.
• Sustained partial response in one patient for 22 months.
• 81% disease control rate (DCR).
• Selective DNA damage to tumor cells without affecting normal liver function.
• Only 5% of patients discontinued due to adverse events.

Negative

• None.

Insights

Medical Research Analyst

The combination therapy of fostrox and Lenvima® targeting advanced hepatocellular carcinoma (HCC) is noteworthy because it addresses a significant treatment gap. Current second-line therapies for HCC are limited, with low response rates and a short median time to progression. The reported 24% overall response rate (ORR) and a median time to progression (TTP) of 10.8 months represent a substantial improvement over existing treatments. This is particularly significant as second-line HCC patients generally have poor prognoses, with response rates typically between 5-10% using current therapies.

Importantly, the selective targeting mechanism of fostrox, delivering its cell-killing compound directly to tumor cells while sparing healthy cells, is a notable advancement. This is evidenced by the stable liver function markers (ALT/AST levels and ALBI score), which suggest that this therapy is not only effective but also well-tolerated.

For retail investors, it is important to understand that these results, although promising, are from early-phase trials. While the data are encouraging, the efficacy and safety profile still need validation through larger, randomized controlled trials. The long-term impact on survival rates and quality of life for patients remains to be seen.

Financial Analyst

Medivir's release of positive results from the Phase 1b/2a trial of fostrox in combination with Lenvima® could position the company favorably within the pharmaceutical market. The therapy's potential for addressing a high unmet need in HCC treatment can significantly drive investor interest and bolster the company's valuation. Given that HCC accounts for over 80% of primary liver cancer cases worldwide, the market potential for a successful second-line therapy is substantial.

From a financial perspective, the data presented may increase market confidence and attract investment, paving the way for future funding rounds to support later-stage trials. However, investors should remain cautious, as the transition from promising early-phase results to market approval often involves extensive and costly phases of research, regulatory scrutiny and potential setbacks.

Retail investors should also note the potential timeline for further development. Medivir’s plan to initiate an Investigational New Drug (IND) application in the US in the second half of 2024 indicates that any market approval and subsequent revenue generation are still several years away. Hence, while the data is promising, the financial benefits will take time to materialize.

Oncology Doctor

From a clinical standpoint, the combination of fostrox and Lenvima® provides a glimmer of hope for patients with advanced hepatocellular carcinoma, who have limited treatment options after first-line therapies fail. The selective targeting of tumor cells while preserving normal liver function is a significant advantage, potentially enhancing the quality of life for patients.

The reported disease control rate (DCR) of 81% is particularly impressive, suggesting that a significant majority of patients experienced some form of disease stabilization or response. This is a marked improvement over the current therapeutic landscape, where options are sparse and not very effective. The ongoing presence of one patient on treatment after 22 months underlines the potential durability of this therapy.

However, it is important for investors to recognize that while these early results are promising, further studies are necessary to determine the full efficacy and safety profile. The next phases of clinical trials will be important in confirming these findings and in understanding the long-term impacts on overall survival and quality of life for patients.

• New results from Medivir's Phase 1b / 2a open label trial of fostrox + Lenvima® show great promise treating second-line hepatocellular carcinoma (HCC) patients, an extremely hard to treat population
• Fostrox + Lenvima® achieved a 24% overall response rate (ORR) and estimated median time to progression (TTP) of 10.8 months¹ (4.1 – NE) – with one patient still on treatment after 22 months
• Results come despite very poor prognosis for most second-line HCC patients today, with just 5 – 10% responding to current standard of care treatment, and a typical TTP of only 3 - 4 months
• Medivir's fostrox (fostroxacitabine bralpamide) is an orally-administered, liver-targeted inhibitor of DNA replication. The mechanism is different to existing first-line HCC treatments, delivering its cell-killing compound selectively to tumor cells while minimizing harm to healthy cells

STOCKHOLM, June 27, 2024 /PRNewswire/ -- Medivir AB (NASDAQ: MVIR) (STOCKHOLM: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, today presented positive new data from its ongoing phase Ib / IIa study of fostroxacitabine bralpamide (fostrox) + Lenvima® in advanced hepatocellular carcinoma (HCC) at the ESMO GI (European Society of Medical Oncology, Gastrointestinal Cancers) Cancers Congress in Munich, Germany.

Today's ESMO GI update, poster number 176P, is being presented by Dr Hong Jae Chon on Thursday June 27, shows: 

• An overall response rate (ORR) of 24%, with a disease control rate (DCR) of 81%, while the median TTP is now 10.8 months, with 25% of patients still on treatment
• One patient remains on treatment after 22 months, benefiting from a sustained partial response
• Biopsies confirm selective DNA damage to tumor cells, while there is no impact on normal liver function as measured by ALT/ AST levels and stable ALBI score over time

The lack of impact on normal liver function supports the previously reported encouraging safety and tolerability profile, where only 5% of patients had to discontinue due to adverse events and the need for dose modification was lower than expected.

Jens Lindberg, CEO at Medivir, said: "With new data, including clear evidence of tumor selectivity, the clinical benefit of adding fostrox to Lenvima has greatly improved. We are particularly encouraged by the duration of benefit, with patients staying on treatment much longer than anticipated. Second-line hepatocellular carcinoma remains an indication with substantial need for improved outcomes for patients, with no approved treatment options after current standard of care. The data presented at ESMO GI makes us even more convinced of fostrox's future potential in the treatment of HCC. We are now initiating study feasibility and finalizing the study protocol and synopsis which will lead to the opening of IND in the US, which is expected in H2 2024."

Dr Hong Jae Chon, Professor at CHA Bundang Hospital in Korea, and investigator in the fostrox + Lenvima® study, commented: "Advanced hepatocellular cancer is a challenging disease to treat, especially in second-line after patients have stopped responding to an immunotherapy (IO) combination. There is a need for new treatments that do not worsen liver function and that have different mechanisms of action compared with first line. These data for fostrox + Lenvima show highly encouraging clinical benefits for patients. They indicate that when adding fostrox to Lenvima, efficacy is better than expected from Lenvima alone. I look forward to further explore the efficacy of fostrox plus Lenvima in a randomized, controlled trial."

The data are from Medivir's ongoing phase 1b/2a open-label, multi-center, dose-escalation and dose-expansion study, evaluating the safety and efficacy of fostrox in combination with Lenvima in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable.

HCC is the most common type of liver cancer, accounting for more than 80% of cases worldwide.² There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent³.

The poster will also be available on Medivir's website after it has been presented at ESMO GI.

For additional information, please contact:
Magnus Christensen, CFO, Medivir AB
Telephone: +46 8 5468 3100
E-mail: magnus.christensen@medivir.com

Optimum Strategic Communications
Nick Bastin, Stephen Adams, Eleanor Cooper
Telephone: +44 20 3922 0891
E-mail: medivir@optimumcomms.com

Cord Communications
Lars Wahlstrom, Adam Ewing
Telephone: +46 734 340 771

About fostrox

Fostrox is a liver-targeted inhibitor of DNA replication that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. This design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver. With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing where it has shown encouraging anti-cancer efficacy with a good safety and tolerability profile.

About primary liver cancer

Primary liver cancer is the third leading cause of cancer-related deaths worldwide. Hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver and it is the fastest growing cancer in the USA. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent^2,3. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need.

About Medivir

Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a smart, targeted chemotherapy designed to selectively treat liver cancer cells and to minimize side effects. Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. www.medivir.com.

1)    Data cut-off 30 May, 2024

2)    Rumgay et al.,European Journal of Cancer 2022 vol.161, 108-118.

3)    Yang, J.D., Hainaut, P., Gores, G.J. et al. A global view of hepatocellular carcinoma: trends, risk, prevention and management. Nat Rev Gastroenterol Hepatol 16, 589–604 (2019).

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Medivir ESMO GI PR_27 June ENG

 

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SOURCE Medivir

FAQ

What were the results of the Medivir Phase 1b/2a trial for fostrox + Lenvima® in advanced liver cancer?

The trial showed a 24% overall response rate (ORR) and a median time to progression (TTP) of 10.8 months for treating advanced hepatocellular carcinoma (HCC).

How did fostrox + Lenvima® perform in terms of disease control rate (DCR) in the Medivir trial?

The disease control rate (DCR) was 81% in Medivir's trial of fostrox + Lenvima® for advanced hepatocellular carcinoma (HCC).

What is the significance of the new data presented by Medivir at ESMO GI 2024 for MVIR stock?

The positive trial results for fostrox + Lenvima® in treating advanced hepatocellular carcinoma (HCC) could boost investor confidence in Medivir's future potential, possibly benefiting MVIR stock.

When is Medivir expected to open the IND application for fostrox in the US?

Medivir aims to open the Investigational New Drug (IND) application in the US for fostrox in the second half of 2024.

How does fostrox's mechanism of action differ from existing HCC treatments?

Fostrox is a liver-targeted inhibitor of DNA replication that selectively damages tumor cells while minimizing harm to healthy cells, unlike existing first-line HCC treatments.

What was the overall response rate (ORR) for second-line HCC patients treated with fostrox + Lenvima® in Medivir's trial?

The overall response rate (ORR) for second-line HCC patients treated with fostrox + Lenvima® was 24%.

What is the projected median time to progression (TTP) for patients treated with fostrox + Lenvima® according to Medivir's recent data?

The median time to progression (TTP) projected for patients treated with fostrox + Lenvima® is 10.8 months.