On April 20, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it has received innovative licensing and access pathway (ILAP) designation from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for AUTO4 being studied in a Phase 1 study in TRBC1 positive Peripheral T Cell Lymphoma (PTCL) (Press release, Autolus, APR 20, 2021, View Source [SID1234578261]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The granting of the Innovation Passport and entry into ILAP comes soon after we received PRIority MEdicines (PRIME) designation from the European Medicines Agency for AUTO1, with both designations accelerating the review of promising therapies targeting unmet medical needs," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "We look forward to working with the MHRA as we progress AUTO4 through the clinic and to providing an interim update on the Phase 1 program later this year."

About ILAP
ILAP was announced in December 2020 and launched at the start of 2021 in order to accelerate the development and access to promising medicines and is geared toward medicines that are in the early stages of development. The pathway, part of the UK’s plan to attract life sciences development in the post-Brexit era, features enhanced input and interactions with MHRA and other stakeholders including the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). (RELATED: MHRA sheds light on pathway to accelerate R&D, Regulatory Focus 24 December 2020).

The innovation passport designation is the first step in the ILAP process and triggers the MHRA and its partner agencies to create a target development profile (TDP) document to chart out a roadmap for regulatory and development milestones with the goal of early patient access in the UK. Other benefits of ILAP include access to range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit risk assessment.

On April 20, 2021 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that the Company has invested into OxVax, a new immuno-oncology company based on research from Oxford University, which enables the development of the next generation of cancer vaccines with the potential to overcome the limitations of the current approaches (Press release, Evotec, APR 20, 2021, View Source;announcements/press-releases/p/evotec-invests-into-oxford-cell-therapy-company-oxvax-6053 [SID1234578281]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

OxVax is developing an innovative cancer vaccine platform based on a unique and proprietary population of dendritic cells capable of inducing a potent anti-tumour immune response. The company’s technology allows the bulk manufacture of these cells from donor blood derived stem cells. By ex vivo loading of these cells with tumour antigens, OxVax will be able to create a vaccine which can train the body to target and eliminate tumours.

The research is based on the work of Professor Paul J Fairchild and Tim Davies from the Sir William Dunn School of Pathology at Oxford University. The founders will be joined by Marcelo Bravo as Chief Executive Officer, a serial entrepreneur who has taken two companies public.

Evotec’s investment is co-led by the South Korean-based venture capital company Lead Compass Investment. Financial details were not disclosed.

Dr. Thomas Hanke, Head of Academic Partnerships at Evotec, said: ‘We are excited to work with the Oxford researchers and Lead Compass to support the development of next-generation dendritic cell therapies to treat tumours with a high unmet medical need in the future. This investment fits well with Evotec’s ambition to become a biotech powerhouse in off-the-shelf cell therapy offerings.’

Professor Paul J Fairchild, Associate Professor of the Immunobiology of Stem Cells at Oxford University, said: "Our research has shown how stem cells can be used to create potentially unlimited numbers of a rare cell type of the immune system responsible for orchestrating the immune response to solid tumours. We believe that access to these cells can open the field of cancer vaccination and transform the treatment of some of the most intractable cancers."

Marcelo Bravo, Chief Executive Officer at OxVax, added: "Our platform enables the manufacture at scale of an off-the-shelf highly potent vaccine which addresses the major limitations that have frustrated cancer vaccine development in the past. Our immediate focus will be the definition of the quality profile of the product and the industrialisation of the manufacturing protocol which will put us in a strong position to proceed towards the clinic."

Tae-erk Kim, Chief Executive officer at Lead Compass Investment, added: "We are excited to invest in OxVax’s technology since it addresses the low migration, cross-presentation, and T-cell activation problems of past dendritic cell cancer treatments. Promising results in an oncology setting would further pave the way for OxVax to expand its technology into other therapeutic areas and be the first company to have dendritic cells act as the true control tower of the immune system."

On April 20, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) Application to initiate a Phase 1b dose escalation study evaluating VIP152, a highly selective PTEFb/CDK9 inhibitor, in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and Richter syndrome (RS) (Press release, Vincerx Pharma, APR 20, 2021, View Source [SID1234578246]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The IND clearance for VIP152 in CLL is an important milestone for Vincerx, marking our first IND clearance and now second clinical program for what we believe is the most selective CDK9 inhibitor in clinical development," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "Preclinical data for VIP152 show highly selective, ATP-independent, inhibition of CDK9 which translates to robust on-target activity across key gene targets. Most importantly, we believe this differentiated profile leads to encouraging early clinical activity, with demonstrated durable single-agent activity in hematologic malignancies and heavily pretreated solid tumors. This new dose-escalation study in CLL and Richter syndrome, expected to initiate before year end, builds upon our planned Phase 1b expansion cohort study in MYC-driven hematologic malignancies and solid tumors, which is on track to begin patient dosing in Q2 2021. We are proud of our rapid progress and look forward to continued execution as we advance VIP152 through our targeted oncology clinical programs to address a broad range of aggressive, resistant cancers."

The Phase 1b dose-escalation study will evaluate VIP152 in patients with relapsed/refractory CLL who have failed a Bruton tyrosine kinase inhibitor (BTKi) and venetoclax. Part 1 of the study will enroll CLL patients treated with ³2 prior regimens including either a BTKi or venetoclax. Part 2 of the study will consist of a CLL Phase 1b expansion which will enroll 20 patients with CLL relapsed/refractory to venetoclax and BTKi, and a RS Phase 1b expansion which will enroll 20 patients with CLL transformed to diffuse large B cell lymphoma (DLBCL) who have relapsed after, or been refractory to, at least 1 prior line of therapy for DLBCL and having MYC overexpression/ amplification/translocation. The Company expects to initiate the Phase 1b dose-escalation study in 2H 2021.

The Phase 1b dose-escalation in CLL and RS builds upon Vincerx’s ongoing first-in-human (FIH) study in patients with advanced cancer. Part 2 of the FIH study is on-track to begin patient dosing in 2Q 2021 and will consist of two expansion arms. Arm 1 will enroll up to 30 patients with relapsed/refractory aggressive lymphoma including DLBCL, transformed follicular lymphoma, or blastoid mantle cell lymphoma. Arm 2 will enroll up to 40 patients with advanced solid tumors, including patients with ovarian cancer, triple negative breast cancer, castration-resistant neuroendocrine prostate cancer, and any other solid tumor with MYC aberration. All patients must have confirmed MYC overexpression or translocation.

On April 20, 2021 Daiichi Sankyo UK, Limited (hereafter, Daiichi Sankyo) and AstraZeneca UK reported the news that the National Institute for Health and Care Excellence (NICE) has recommended Enhertu (trastuzumab deruxtecan) for use within the Cancer Drugs Fund (CDF) as an option for treating HER2 positive unresectable or metastatic breast cancer in adults who have received two or more prior anti-HER2 based therapies (Press release, Daiichi Sankyo, APR 20, 2021, View Source [SID1234578262]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the UK, almost 54,000 cases of breast cancer in women are diagnosed annually, with an estimated one in five cases being HER2 positive.1,2,3 The impact of the disease is significant, with breast cancer responsible for approximately 12,000 deaths per year.1 There are an estimated 35,000 people living with metastatic breast cancer in the UK, and in around 5% of women the breast cancer has already spread by the time it is diagnosed.4

"HER2 positive disease impacts one in five women with breast cancer, yet there is still no clear standard of care for patients with HER2 positive disease who have progressed following first-and second-line therapy," said Professor Peter Schmid, Barts Cancer Institute. "The availability of trastuzumab deruxtecan through NHS England’s Cancer Drugs Fund is good news for patients and brings an important new treatment option to those whose disease has continued to progress despite previous treatment."

"This authorisation is a significant step forward for the many thousands of people in England living with HER2 positive metastatic breast cancer," said Jo Taylor, founder of METUP UK, an advocacy group for people living with metastatic breast cancer. "Disease progression in metastatic breast cancer patients is an unmet need beyond second line treatment and new medicines are essential in the challenge to suppress this incurable disease."

This recommendation from NICE is based on the results of the single arm, multicentre, open label, phase 2 DESTINY-Breast01 trial of trastuzumab deruxtecan (5.4 mg/kg) in 184 patients with HER2 positive metastatic breast cancer who had received two or more prior anti-HER2-based therapies. Results from the data cut-off in June 2020 demonstrated a confirmed objective response rate (ORR) of 61.4% (95% CI: 54.0-68.5), including a 6.5% complete response rate and a 54.9% partial response rate. After a median follow-up of 20.5 months, the median duration of response (DoR) was 20.8 months (95% CI: 15.0-NR).5 Trastuzumab deruxtecan showed a generally tolerable safety profile with 34 (18.5%) treatment discontinuations due to treatment-emergent adverse events.6

"We are very proud to have worked with NICE, NHS England and the breast cancer community to make trastuzumab deruxtecan available, through the Cancer Drugs Fund, to eligible patients in England with HER2 positive metastatic breast cancer whose disease has progressed following treatment with two anti-HER2 directed therapies," said Haran Maheson, Commercial Director for Oncology, Daiichi Sankyo U.K.

"Though many treatment advances have been made in HER2 positive metastatic breast cancer, there has been no clear standard of care for patients following progression after second line treatment and many patients do not have a durable response to other available later-line options. To know that patients in England now have access to a new treatment option is welcome news indeed," said Arun Krishna, Head of Oncology, AstraZeneca U.K.

Daiichi Sankyo and AstraZeneca UK will continue working in close partnership with NICE as additional data are collected throughout the managed access period. During this time, eligible patients will be able to access trastuzumab deruxtecan in advance of a decision from NICE on routine funding on the NHS.

The CDF recommendation is applicable to patients in England. Discussions with Welsh and Northern Irish health authorities are ongoing and the submission for the appraisal of trastuzumab deruxtecan to the Scottish Medicines Consortium is currently in development, with a decision expected later in 2021.

The safety of trastuzumab deruxtecan has been evaluated in a pooled analysis of 234 patients with unresectable or metastatic HER2 positive breast cancer who received at least one dose of trastuzumab deruxtecan 5.4 mg/kg in clinical studies. The median duration of exposure to trastuzumab deruxtecan was 9.8 months (range: 0.7 to 37.1 months). The most common adverse reactions were nausea (79.9%), fatigue (60.3%), vomiting (48.7%), alopecia (46.2%), constipation (35.9%), decreased appetite (34.6%), anaemia (33.8%), neutropenia (32.5%), diarrhoea (30.8%), thrombocytopenia (23.1%), cough (21.4%), leukopenia (20.5%), and headache (20.1%).5

Cases of interstitial lung disease (ILD) or pneumonitis were reported in 15% of the 234 patients. Fatal outcomes were observed in 3% of patients. Patients should be advised to immediately report cough, dyspnoea, fever, and/or any new or worsening respiratory symptoms. Patients should be monitored for signs and symptoms of ILD or pneumonitis and those with suspected ILD or pneumonitis should be evaluated by radiographic imaging, preferably a computed tomography (CT) scan. Patients with a history of ILD or pneumonitis may be at increased risk.5

For further information about trastuzumab deruxtecan, such as the licensed indication and safety profile, please refer to the summary of product characteristics.

About HER2 positive breast cancer

HER2 is an epidermal growth factor receptor expressed on the surface of many types of tumours, including breast cancer. HER2 overexpression may be associated with a specific HER2 gene alteration known as HER2 amplification and is often associated with aggressive disease and poor prognosis in breast cancer.7

There remain significant unmet clinical needs for patients with HER2 positive metastatic breast cancer. The disease remains incurable, with patients eventually progressing after currently available treatment options.8,9

About DESTINY-Breast01

DESTINY-Breast01 was a phase 2, single-arm, open-label, global, multicentre, two-part trial evaluating the safety and efficacy of trastuzumab deruxtecan in patients with HER2 positive unresectable and/or metastatic breast cancer who had received two or more prior anti-HER2-based regimens, including trastuzumab emtansine (100%), trastuzumab (100%), and pertuzumab (65.8%). The primary endpoint of the trial was confirmed ORR, as determined by independent central review. Key secondary objectives were disease control rate, clinical-benefit rate, duration of response, progression-free survival, and safety.

About trastuzumab deruxtecan

Trastuzumab deruxtecan is a HER2 directed antibody drug conjugate (ADC). Designed using Daiichi Sankyo’s proprietary DXd ADC technology, trastuzumab deruxtecan is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform.

ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy (‘payload’) to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Trastuzumab deruxtecan is comprised of a humanised anti-HER2 IgG1 monoclonal antibody with the same amino acid sequence as trastuzumab attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a tetrapeptide-based cleavable linker.

About the collaboration between Daiichi Sankyo and AstraZeneca

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialise trastuzumab deruxtecan in March 2019, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of trastuzumab deruxtecan.

On April 20, 2021 Race Oncology Limited ("Race") reported the appointment of Dr David Fuller as Chief Medical Officer (CMO) (Press release, Race Oncology, APR 20, 2021, View Source [SID1234578231]). This appointment follows the announcement by Race on 19 February 2021 that the Company would be recruiting a full-time, Australian- based CMO to drive its three-pillar strategy forward.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

An internationally experienced biopharmaceutical executive and physician, Dr Fuller brings a deep understanding of the successful development & commercialisation of novel pharmaceuticals. His 30 years of R&D experience spans large, mid and small cap companies including pre-clinical and clinical development, medical and regulatory affairs, and commercialisation.

Dr Fuller will join Race Oncology on 1 July 2021 from Syneos Health Clinical Solutions, where he has been the Senior Vice President of Clinical Development in the Oncology Business Unit. Dr Fuller also currently serves as Chairman for EpiAxis Therapeutics, a privately owned epigenetic therapeutic and diagnostic company, and is a Non-Executive Director of the ASX- listed biotech company, AdAlta (ASX: 1AD).

Dr Fuller was formerly a Director at Linear Clinical Research, Chairman of Dimerix Bioscience, CMO/COO at Trident Clinical Research, CMO at Arana Therapeutics, and Vice-President Clinical at Genzyme Europe.

During his career, he has directly led successful major market drug approvals including Moraxen (UK), Busulfex (US Paediatrics and EU Adult indications), Xyrem (US) and Renagel (EU). He has also designed and executed multiple Phase I, II and III studies (US, EU, Asia) for both orphan and non-orphan drug products.

This is an important appointment for Race as David brings significant experience and capability. David has been successful in developing a number of pharmaceutical products across various international markets and we look forward to his future guidance in optimising plans for the execution of our Three Pillar strategy and maximising the significant opportunities we have ahead for Bisantrene.

Race Oncology CEO and Managing Director, Phil Lynch
I am honoured and excited to be joining Race at such a critical time in the Company’s evolution given the huge potential of Bisantrene as both a targeted precision oncology drug and a differentiated chemotherapeutic. I look forward to applying my experience and expertise to help Race successfully realise the objectives of its three-pillar strategy. And I am pleased to have a new opportunity to again work with Professor Borje Anderson after our successful prior collaboration on Busulfex.

Dr David Fuller
Dr Fuller holds a Bachelor of Medicine / Bachelor of Surgery degree, and a Bachelor of Pharmacy (First Class Honours in Pharmacology), both from University of Sydney. He is also a Member of the American Society Clinical Oncology (ASCO) (Free ASCO Whitepaper).