Amplia Therapeutics (ATX: $0.059) is conducting a Phase I/IIa study with its drug candidate narmafotinib in the very difficult-to-treat advanced pancreatic cancer. Not only has the company completed the study ahead of time, which is always a good sign of treatment demand, but it has also reached its endpoint early. The target for the company was to obtain at least 15 partial responses from the 55 patients (27%), which has now been met (50 evaluable). This exceeds historical data from standard-of-care. Amplia's therapy was used in combination with the standard-of-care (gemcitabine with Abraxane).

Early Study and Response Rates

Of the 55 patients, 21 remain on the study, and it is expected that additional responses will be achieved. A partial response is recorded when, for two consecutive assessments spaced two months apart, the main tumours regress by 30% and no new lesions are formed. Another useful measure is the median number of days patients remain on therapy. Historical data with gemcitabine and Abraxane in a study involving 431 patients showed that patients stayed on treatment for 117 days. Data from the first 29 patients (reported 28 April 2025) showed a median duration on treatment of 208 days, suggesting some additive benefit from narmafotinib. Patients generally only come off treatment if they begin to regress. From the first 29 patients there were 11 partial responses which equates to a response rate of 38%. This compares to 23% from historical data with gemcitabine with Abraxane. Amplia completed full recruitment into the study at the end of January.

Upcoming Readout: Progression-Free Survival (PFS)

The next measure for the company to report (in July) will be Progression-Free Survival (PFS) at six months. Historical data shows gemcitabine with Abraxane alone achieves PFS of between 5.5–5.6 months. From data reported last month, 13 of the 26 evaluable patients had already surpassed this mark. Amplia CEO Chris Burns is optimistic that the PFS will be significantly better with the addition of narmafotinib. Anything over a two-month improvement from current treatment will be viewed as significant, believes Burns.

Future Positioning and Combinations

Whilst not being positioned as a cure for patients with advanced pancreatic cancer, Burns believes that given the drug's excellent safety profile, there is the opportunity to position it as a baseline therapy with existing chemotherapy treatment. This also includes in combination with other new therapies in development, including KRAS inhibitors and antibody drug conjugates, which are still some time away from approval. Burns said that the company did not know that the addition of narmafotinib would be as well tolerated as it is.

Of the patients in the current study, the longest sustained treatment period to date is 430 days, with five patients living for more than one year. Once the final results are in, the company will begin plans for a registration-enabling study for the US, which is expected to involve less than 200 patients. It is likely to use the gemcitabine and Abraxane combination as the control, which is dominant in Australia and Japan for pancreatic cancer, and in similar use as FOLFIRINOX in the US. The registration study is expected to cost US$50 million to complete. The company is likely to proceed with regional partnering deals, similar to Dimerix, to help fund the study.

US Study to Commence with FOLFIRINOX

Aside from receiving full data from the current study, the next step for the company is to advance its US/Australian study which will use a different chemotherapy regime, called FOLFIRINOX. This is a more aggressive therapy, achieving a life expectancy of 11 months, compared to 7–8 months with gemcitabine and Abraxane, or 3–4 months with no therapy.

That study will explore two doses (after a dose-ranging phase) and is expected to involve around 60 patients. The first patient in this study has yet to be dosed, with that expected to occur this half. The Australian sites, which have been involved in the current study, are expected to recruit first.

Approval of FAK First Inhibitor from Competitor

Earlier this month, one of Amplia's competitors in the FAK inhibition field, Verastem, gained FDA approval for a combination product—a MEK/RAS inhibitor (avutometinib) with a FAK inhibitor (defactinib)—for the treatment of low-grade ovarian cancer. Amplia's drug candidate is a FAK inhibitor which has shown in preclinical studies to be a more potent inhibitor of the FAK pathway, being more selective and with a better pharmacokinetic profile.

Verastem received accelerated approval based on a study involving just 57 patients. The overall response rate was 44%. The approval was based on tumour response rate and duration of response. It has numerous side effects including increased liver enzyme levels, nausea, fatigue, and rash. Verastem is capitalized at US$456 million.

Summary

Amplia Therapeutics is capitalized at $20 million. It held cash of $10.8 million at the end of March.

Bioshares recommendation: Speculative Buy Class B

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