You are now leaving

Please click 'Continue to external site' if you want to continue.



New data on Helsinn’s oncology pipeline presented at American Society of Clinical Oncology (ASCO) Annual Meeting 2022

Lugano, Switzerland, June 02, 2022 Helsinn Group (“Helsinn”), a fully integrated, global biopharma company with a diversified pipeline of innovative oncology assets, announces its participation in the upcoming 2022 American Society of Clinical Oncology (ASCO) Annual Meeting which will be held from 3-7 June.

In alignment with Helsinn’s new Fully Integrated Target Therapy (FITT) strategy in oncology, two abstracts have been accepted for poster presentation.

The first abstract shares results of a preclinical study exploring the brain penetrability of vepafestinib (TAS0953/HM06), Helsinn’s investigational oral treatment targeting rearranged during transfection (RET) abnormalities in solid tumors.

Title: Comparison of TAS0953/HM06 and selpercatinib in RET Fusion-driven Preclinical Disease Models of Intracranial Metastases

  • Author: Igor Odintsov, MD, Memorial Sloan Kettering Cancer Center
  • Session: Central Nervous System Tumors
  • Abstract ID: 2024

The second abstract provides an overview of the ongoing PROOF 301 trial of infigratinib, a kinase inhibitor targeting cancers driven by fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Details are as follows:

Title: PROOF 301: a multicenter, open-label, randomized, phase 3 trial of infigratinib vs gemcitabine + cisplatin in patients with advanced cholangiocarcinoma with an FGFR2 gene fusion/rearrangement

  • Author: Ghassan K. Abou-Alfa, MD, MBA, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College
  • Session: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary  
  • Abstract ID: TPS4171

Sergio Cantoreggi, Helsinn Group Chief Scientific Officer and Head of R&D, commented: “We are excited that the abstracts on two of our investigational products, vepafestinib and infigratinib, have been accepted for presentation at ASCO. In line with our Fully Integrated Targeted Therapy strategy, we are dedicated to progressing our pipeline of products to treat rare oncological diseases. More broadly, we are increasingly convinced of the importance of adequate screening for genetic alterations, through the use of comprehensive biomarker testing, to expand disease treatment options, thus ensuring that patients can receive the most appropriate and targeted treatment.”

Helsinn is a fully integrated, global biopharma company headquartered in Lugano, Switzerland. It is focused on improving the lives of cancer patients all over the world with a leading position in cancer supportive care and an innovative pipeline of cancer therapeutics.

Helsinn is a third-generation family-owned company, that since 1976 has been focused on improving the lives of patients, guided by core values of respect, integrity and quality. It operates a unique licensing business model with integrated drug development and manufacturing capabilities. Helsinn has a commercial presence in 190 countries either directly, with operating subsidiaries in the U.S. and China, or via its network of long-standing trusted partners. Helsinn also has a fully integrated supply chain and product development through its subsidiary in Ireland, Helsinn Birex Pharmaceuticals Ltd.

Helsinn plays an active and central role in promoting social transformation in favor of people and the environment. Corporate social responsibility is at the heart of everything we do, which is reinforced in the company’s strategic plan by a commitment to sustainable growth.

To learn more about Helsinn please visit

Vepafestinib (also known as TAS0953/HM06 in partnership with Taiho Pharmaceutical) is an investigational, potent, orally administered, highly selective RET inhibitor1,2. Relative to first generation RET inhibitors, vepafestinib is pharmacologically distinct, exhibits a distinct binding mode to RET, and has shown evidence of enhanced brain penetrability characteristics in preclinical models3. Vepafestinib is currently being evaluated in a phase 1 /2 study (the margaRET study, NCT04683250) in individuals with advanced solid cancers with RET abnormalities, including those resistant to first-generation selective RET inhibitors. Taiho and Helsinn signed a co-development and commercialization agreement for TAS0953/HM06 in 2017 and will continue to pursue together all preclinical, clinical and CMC developments.

RET is a transmembrane receptor tyrosine kinase. Abnormalities in the RET gene, such as fusions and point mutations, are oncogenic drivers of multiple human cancers. RET fusions are present in 1-2 % of patients with non-small cell lung cancer (NSCLC) and are associated with a high incidence of brain metastasis at diagnosis. Patients are typically young and non-smokers. Although treatment for these patients has significantly improved in recent years, acquired resistance to first generation RET inhibitors has emerged. Overcoming this resistance and addressing the CNS progression in these patients are important areas of unmet need4,5.

Infigratinib (BGJ398) is an oral, selective, small molecule kinase inhibitor of FGFR 1, 2, and 3. Infigratinib has received regulatory approval in the USA, Canada, and Australia for the treatment of adults with previously treated locally advanced or metastatic cholangiocarcinoma (bile duct cancer) with a FGFR2 fusion or rearrangement. All three approvals were conditional and will require further evidence of efficacy. (See below for full US FDA-approved indication). The therapy is currently under investigation as a potential first-line treatment for adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion/rearrangement (PROOF 301 Trial, NCT03773302), in the adjuvant setting for adult patients with invasive urothelial carcinoma (bladder cancer) with susceptible FGFR3 genetic alterations (PROOF 302 trial, NCT04197986), and in pediatric patients with advanced solid and central nervous system tumors with selected FGFR1-3 alterations (NEWEL Trial, NCT05222165).

CCA represents an aggressive group of malignancies that form in the bile ducts. Although rare in most countries (with a worldwide estimated incidence of <6 per 100,000 people), the incidence of this malignancy is increasing worldwide. Because the disease is usually asymptomatic at early-stages, diagnosis may be delayed until advanced stages, when CCA typically presents as locally advanced or metastatic disease. Despite continuing advances in treatments, the prognosis for this disease remains poor, with a 5-year survival rate of 7-20%9. Cholangiocarcinoma is currently classified into intrahepatic and extrahepatic, on the basis of the anatomical site of origin. FGFR2 alterations are present in approximately 10-16%6,7 of CCA patients with intrahepatic disease and represent potential targets for treatments.8,9

1 Miyazaki I., Ishida K., Kato M.,  et al. Discovery of TAS0953/HM06, a novel next generation RET-specific inhibitor capable of inhibiting RET solvent front mutations. Mol Cancer Ther (2021) 20 (12_Supplement): P06-02.

2 Odintsov I., Kurth R.I., Ishizawa K., et al. TAS0953/HM06 is effective in preclinical models of diverse tumor types driven by RET alterations. Mol Cancer Ther (2021) 20 (12_Supplement): P233.

3 Odintsov I., Lui A. J. W., Ishizawa K., et al, Comparison of TAS0953/HM06 and selpercatinib in RET Fusion-driven Preclinical Disease Models of Intracranial Metastases. Abstract 2024, ASCO 2022.

4 Subbiah V & Cote GJ, Advances in Targeting RET-Dependent Cancers. Cancer Discov. 2020 Apr;10(4):498-505. doi: 10.1158/2159-8290.CD-19-1116.

5 Drusbosky L.M., Rodriguez E.Dawar R., Ikpeazu C.V. Therapeutic strategies in RET gene rearranged non-small cell lung cancer. J Hematol Oncol. 2021; Mar 26;14(1):50. doi: 10.1186/s13045-021-01063-9.

6 Farshidfar, F. , Zheng, S. , Gingras, M  et al. Integrative Genomic Analysis of Cholangiocarcinoma Identifies Distinct IDH-Mutant Molecular Profiles. Cell Rep 2017 Mar 4;18(11):2780-2794. doi: 10.1016/j.celrep.2017.02.033

7 Kongpetch, S.,  Jusakul, A.,  Quan Lim, J. et al. Lack of Targetable FGFR2 Fusions in Endemic Fluke-Associated Cholangiocarcinoma. CO Glob Oncol 2020 Apr;6:628-638.doi: 10.1200/GO.20.00030

8 Banales, J., Cardinale, V., Carpino, G. et al. Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol 13, 261–280 (2016).

9 Banales, J.M., Marin, J.J.G., Lamarca, A. et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol 17, 557–588 (2020).

Helsinn Media Contact:

Paola Bonvicini
Group Head of Communication
Lugano, Switzerland
Tel: +41 (0) 91 985 21 21