PharmaEssentia Provides U.S. Regulatory Update on Ropeginterferon alfa-2b-njft for the Treatment of Polycythemia Vera (PV)

On March 14, 2021 PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the company’s Biologics License Application (BLA) for ropeginterferon alfa-2b-njft for the treatment of polycythemia vera (PV), a rare blood cancer (Press release, PharmaEssentia, MAR 14, 2021, View Source [SID1234576612]).

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The rationale for the CRL was COVID-related travel restrictions, which delayed a required pre-approval inspection of the company’s manufacturing facility in Taiwan. The FDA also indicated a need for additional data about the administration format with the product. Importantly, no concerns were raised about the clinical profile of the product.

"We are confident that we can work with the Agency to address the requests highlighted in the response letter and resubmit in an expeditious manner," said Meredith Manning, U.S. General Manager. "We remain fully committed to introducing ropeginterferon alfa-2b-njft to the U.S. PV community."

Ropeginterferon alfa-2b-njft has Orphan Drug designation for the treatment of PV in the United States. Marketed as Besremi in Europe, the product was approved by the European Medicines Agency (EMA) in 2019. The molecule was invented and is manufactured by PharmaEssentia.

Long-term results from MINDACT trial confirm importance of gene expression signature in choosing breast cancer patients who can be spared chemotherapy

On March 13, 2021 EORTC reported that The MINDACT (10041/BIG3-04) study, a multicentre, randomised phase 3 clinical trial, aimed to provide an answer to this question, specifically in women with early-stage breast cancer where the cancer had either not spread to the lymph nodes under the arm, or to no more than three nodes (Press release, EORTC, MAR 13, 2021, View Source [SID1234576624]). Between 2007 and 2011, 6693 patients aged 18 to 70 were enrolled to the trial, which was carried out in 112 institutions in nine European countries.

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Women at low clinical and genomic risk received no chemotherapy, whereas those at high risk for both did. Patients with discordant risk results were randomly assigned to receive chemotherapy or not based either on the genomic risk (determined by the 70-gene signature MammaPrint) or the clinical risk (determined by the risk prediction model Adjuvant! Online). After a median follow-up of five years, the primary analysis showed that patients who were classified as high clinical risk and low genomic risk who did not receive chemotherapy had a 5-year distant metastasis-free survival of 94.7%, a result in line with the primary hypothesis of the trial.

A further analysis at a median 8.7 years of follow-up is published on 12th of March in The Lancet Oncology*. The updated results of the primary analysis confirm the previous results: with more than 90% patients with at least 5 years of follow-up, the 5-year distant metastasis-free survival rate is now estimated to be 95.1% in the primary test population. For the 1497 women who were at high clinical but low genomic risk, distant metastasis-free survival at 8 years was slightly higher (2.6%) in those who received chemotherapy (749 women) as compared to those who did not receive chemotherapy (748).

«There were important differences in results between age groups. For women younger than 50 years the benefit of chemotherapy is present (5%), perhaps because it suppresses ovarian function as an indirect mechanism of action. But in women over 50 years old our results further confirm our earlier conclusion that women with early breast cancer at clinical high but genomic low risk can continue to be spared adjuvant chemotherapy (benefit 0.2%). » said Principal Investigator Professor Martine Piccart, from the Institut Jules Bordet and cofounder of the Breast International Group (BIG), Brussels, Belgium. "More research is definitely needed for the younger women."

«Our findings will enable us to give individual patients better information about the risks and benefits of adjuvant chemotherapy, and allow them to play their part in making informed decisions on their treatment.»

«We are extremely proud to have partnered with the EORTC and BIG on the landmark MINDACT trial, » said William Audeh, MD, MS, Chief Medical Officer at Agendia. «The results seen in the long term follow-up data reinforce the immense effect the trial will have in advancing more effective treatment planning for patients with breast cancer. The study continues to produce meaningful clinical information on the de-escalation of treatment while encouraging us to conduct further research in additional patient populations to better understand the role of genomic information. »

Professor Michail Ignatiadis, the new chair of the EORTC Breast Cancer Group explained that the EORTC Breast Cancer Group has a long tradition on conducting practice changing trials. «The updated analyses of the MINDACT trial will help many postmenopausal women with breast cancer to avoid unnecessary adjuvant chemotherapy. The MINDACT trial exemplifies the new focus of the Group on embracing innovation in order to deliver precision medicine», he said.

* 70-gene signature as an aid to treatment decisions in early breast cancer: updated results of the Phase 3 randomized MINDACT trial

AACR 2021 presentation on preclinical data for TAC-001

On March 12, 2021 TallacTherapeutics reported AACR (Free AACR Whitepaper) 2021 presentation on preclinical data for TAC-001, the lead program from our novel Toll-like Receptor Agonist Antibody Conjugate (TRAAC) platform (Press release, Tallac Therapeutics, MAR 12, 2021, View Source [SID1234579806]). "TAC-001, a toll-like receptor 9 (TLR9) agonist antibody conjugate targeting B cells, promotes anti-tumor immunity and favorable safety profile following systemic administration in preclinical models"

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BERGENBIO COMPLETES ENROLMENT INTO LATEST COHORT OF PHASE II BEMCENTINIB COMBINATION STUDY IN REFRACTORY NSCLC

On March 12, 2021 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical need, reported that it has completed enrolment into Cohort C1 of its Phase II study (BGBC008) of bemcentinib in combination with anti-PD-1 therapy Keytruda (pembrolizumab) in refractory non-small cell lung cancer (NSCLC) patients (Press release, BerGenBio, MAR 12, 2021, https://www.bergenbio.com/bergenbio-completes-enrolment-into-latest-cohort-of-phase-ii-bemcentinib-combination-study-in-refractory-nsclc/ [SID1234578614]).

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The BGBC008 trial ClinicalTrials.gov Identifier: NCT03184571 is being conducted in three cohorts evaluating the safety and benefit of bemcentinib and pembrolizumab combination in refractory NSCLC patients. Cohort C is assessing second line patients refractory to first line treatment with checkpoint inhibitors in combination with chemotherapy. A total of 13 patients have been enrolled in Cohort C1 and the first efficacy data is expected within 24 weeks. If successful the trial will expand to Cohort C2, assessing a further 29 patients.

The study is being sponsored by BerGenBio in collaboration with MSD, a tradename of Merck & Co., Inc., Kenilworth, New Jersey, USA, who continue to supply Keytruda (pembrolizumab) for use in the study, under a collaboration agreement signed in March 2017.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We are pleased to have completed enrolment into the first stage of the third cohort planned for this study. We are excited by the potential patient benefit of
selective AXL inhibition with bemcentinib, to reverse acquired resistance to immune checkpoint inhibitors in cAXL-positive patients who have relapsed on immunotherapy and chemotherapy. There are currently limited treatment options for patients for whom first line therapies in NSCLC have been ineffective. If successful, this combination treatment could provide an important alternative to second line chemotherapy standard-of-care. I look forward to providing an update on the data from this cohort in due course."

About AXL

AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases.

In COVID-19, AXL has two synergistic mechanisms of action, it acts a co-receptor to ACE2, to which the spike protein of the Sars-Cov-2 virus attaches and enters the host cell, and AXL expression is upregulated that leads to suppression of the Type 1 Interferon immune response by host cells and in their environment. Research data confirms bemcentinib inhibits SARS-CoV-2 host cell entry and promotes the anti-viral Type I interferon response.

In cancer, increase in AXL expression has been linked to key mechanisms of drug resistance and immune escape by tumour cells, leading to aggressive metastatic cancers. AXL suppresses the body’s immune response to tumours and drives treatment failure across many cancers. High AXL expression defines a very poor prognosis subgroup in most cancers. AXL inhibitors, such as bemcentinib, therefore, have potential high value as monotherapy and as the cornerstone of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases including fibrosis.

About Bemcentinib

Bemcentinib (formerly known as BGB324), is a potential first-in-class, potent and highly selective AXL inhibitor, currently in a broad phase II clinical development programme. It is administered as an oral capsule and taken once per day. Ongoing clinical trials are investigating bemcentinib in COVID-19, and multiple solid and haematological tumours, in combination with current and emerging therapies (including immunotherapies, targeted therapies and chemotherapy), and as a single agent. Bemcentinib targets and binds to the intracellular catalytic kinase domain of AXL receptor tyrosine kinase and inhibits its activity.

Precision BioSciences to Report Fourth Quarter and Fiscal Year 2020 Financial Results on March 18, 2021

On March 12, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company, reported it will publish fourth quarter and fiscal year 2020 financial results and provide a business update on Thursday, March 18, 2021 (Press release, Precision Biosciences, MAR 12, 2021, View Source [SID1234576692]).

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