On April 8, 2020 AIVITA Biomedical, Inc., a biotechnology company specializing in innovative cell therapy applications, reported updated survival data from a year-end analysis of its ongoing Phase 2 clinical trial of AV-GBM-1 in patients with newly diagnosed glioblastoma (GBM) (Press release, AIVITA Biomedical, APR 8, 2020, View Source [SID1234556190]).

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In the analysis, the overall survival rate at both 12 and 15 months is 76% in AV-GBM-1-treated patients (n=50), compared to overall survival of 61% at 12 months and 48% at 15 months in control patients (n=287) receiving standard of care. Patient recruitment is complete in the study, with 55 patients enrolled across eight trial sites. Additional details are available through ClinicalTrials.gov.

"Although treatment and monitoring of patients is ongoing, we are very encouraged by these interim results," said Dr. Robert O. Dillman, AIVITA Chief Medical Officer. "We completed accrual to the trial ahead of schedule, thanks to the engagement of our clinical site principal investigators and a truly outstanding manufacturing success rate at AIVITA."

AV-GBM-1 is a novel immunotherapy consisting of autologous dendritic cells loaded with autologous tumor antigens derived from self-renewing tumor-initiating cells. The treatment is administered in a series of subcutaneous injections as an adjunctive therapy. The treatment is uniquely pan-antigenic, targeting multiple antigens on autologous tumor-initiating cells responsible for the rapid growth of the disease and resistance to standard therapy.

AIVITA is currently conducting three clinical studies in the United States investigating its platform immunotherapy in patients with ovarian cancer, glioblastoma and melanoma. In a past clinical trial in metastatic melanoma, AIVITA’s autologous dendritic cells has demonstrated 72% 2-year survival and 54% 5-year survival in patients (n=92). Based on data, AIVITA is currently seeking conditional commercial approval of its melanoma treatment in Japan. The clinical trial in advanced ovarian cancer is a Phase 2 randomized study currently enrolling patients at the time of initial diagnosis.

About AIVITA’S Clinical Trials

OVARIAN CANCER

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous tumor-initiating cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the tumor-initiating cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with AIVITA’s tumor-initiating cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

For additional information about AIVITA’s AV-MEL-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03743298

On April 7, 2020 Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to selectively and efficiently degrade and remove disease causing proteins, reported the closing of a USD $7.5m Series A round, led by Advent Life Sciences (Press release, Amphista Therapeutics, APR 7, 2020, View Source [SID1234557642]). Seed round funders the Scottish Investment Bank, with backing from the Scottish Growth Scheme, and the European Investment Fund joined the round, along with new investor, US-based life sciences BioMotiv.

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Amphista’s CEO Nicola Thompson said, "This international financing provides Amphista with a firm foundation to underpin a Series B round to progress our oncology pipeline to the clinic. Our vision is to create a leading protein degradation company on the global stage that delivers ground-breaking new medicines to patients in areas of high unmet need."

Raj Parekh, General Partner at lead investor Advent Life Sciences said, "We are excited to support Amphista in its next stage of development. The Company has a potentially unique approach to targeted protein degradation when compared with traditional proteolysis targeting chimera (PROTAC) platforms. We believe that Amphista has great potential with its differentiated proprietary technology to address traditionally undruggable targets."

Satish Jindal, CEO of BioMotiv, and newly appointed Amphista Chairman, commented, "We are a mission-driven accelerator, and we are excited by Amphista’s focus to rapidly produce potent bifunctional small molecules to augment the body’s own processes to remove disease-associated proteins. We see huge potential to accelerate Amphista’s breakthrough technology platform into medicines."

Amphista’s scientific founder, Professor Alessio Ciulli, based at the University of Dundee, is an internationally renowned expert in the field of targeted protein degradation (TPD). "Highly specific TPD is a transformative new modality for tackling previously undruggable targets with high therapeutic value." said Ciulli.

Amphista’s TPD small molecules instruct the cell to degrade the target directly rather than activating or inhibiting the target protein function. As protein-protein interactions are involved in disease progression, removing the target protein provides a clear therapeutic advantage over simple inhibition. Specifically, Amphista’s platform is independent of traditional E3 ubiquitin ligases used by the field, potentially expanding the available target scope of TPD approaches and should overcome recently identified PROTAC resistance mechanisms.

On April 7, 2020 Abbisko Therapeutics Co., Ltd. reported the completion of the cohort 1 study for ABSK-021 phase I clinical trial in the US and the enrollment of the first patient for ABSK-011 phase I trial in Taiwan, China (Press release, Abbisko Therapeutics, APR 7, 2020, View Source;article_id=142&brd=1 [SID1234556284]). ABSK-021 has demonstrated excellent PK properties in the first cohort of patients with strong target engagement. Both innovative drug candidates, independently discovered and developed by Abbisko Therapeutics, have officially entered into clinical stage development.

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ABSK-021 is a highly potent and selective small molecule inhibitor of CSF-1R with oral availability. Blocking CSF-1R signaling pathway can modulate tumor microenvironment, release tumor immunosuppression, and synergize with a variety of anticancer therapies such as tumor immune-checkpoint antibodies to achieve better tumor inhibition. Clinical studies have demonstrated that inhibition of CSF-1R activity can be used to treat tenosynovial giant cell tumor (TGCT) and many other human malignancies.

ABSK-011 is an oral, potent, and highly selective small molecule inhibitor of FGFR4 that exhibited strong anti-tumor effects in preclinical models with excessive activation of FGFR4 signaling. In December of 2019, ABSK-011 was first approved by the FDA of Taiwan (China) to enter into Phase I clinical study. In February, ABSK-011 was also approved for entering clinical studies by the national medical products administration of China (NMPA).

On April 7, 2020 A new study published in Cancer Discovery shows a possible alternative to chemotherapy for infants with high-grade glioma tumors (Press release, The Institute of Cancer Research, APR 7, 2020, View Source [SID1234556253]). The study reveals that high-grade glioma brain tumors in babies are molecularly unique and could be candidates for targeted treatments.

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High-grade gliomas, tumors of the glial cells, are a rapidly growing type of brain tumor that 150-180 children in North America are diagnosed with each year. More than half of all brain tumors in children are gliomas. Children who are diagnosed as infants, less than 12 months, have a better chance at survival, with only 20% of older children living past five years.

"It’s especially cruel when cancer affects such small children, so it is encouraging that this study offers some hope for very young patients and their families," said Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London. "The research offers a better understanding of the biology of brain tumors in infants and finds that they might potentially be treated with new targeted drugs. That’s vitally important because infants are particularly vulnerable to the side effects from standard treatments such as chemotherapy, and we need much better, kinder options."

This new research has found that when cancer is looked at in infants, the tumors are biologically different than those in older children. Scientists discovered that infant tumors could be targeted with precision medicines because of the genetic make-up of the tumors, which can have devastating side effects and be harmful while their brain is still developing.

"Our study offers the biological evidence to pick out those infants who are likely to have a better outcome from their disease, so these very small children and their families can be spared the harmful effects of chemotherapy," said Professor Chris Jones, Professor of Pediatric Brain Tumour Biology at The Institute of Cancer Research, London.

Scientists at The Institute of Cancer Research in London, working with colleagues at the Hopp Children’s Cancer Center Heidelberg in Germany, the UCL Great Ormond Street Institute of Child Health, and St. Jude Children’s Research Hospital in the U.S., looked at 241 infants diagnosed with glioma brain tumors. This was the largest and most comprehensive study of infant gliomas to date. Research showed a genetic difference from the samples of infant and children’s tumor samples, though they look similar under a microscope. Of the 241 samples, 130 of them had entirely different genetic make-up from other forms of childhood brain tumors. Around 65 of the 130 unique types of tumors had specific molecular weaknesses like ALK and NTRK gene fusions that can be targeted with non-chemo medicines.

In research with mice, they found mice treated with Lorlatinib had a significant shrink in tumor size, compared to those treated with chemotherapy, who’s tumors kept growing, although at a slower rate.

In ‘mini tumors’ grown from patient samples, tumors with NTRK and other genes were sensitive to targeted treatments.

A small number of patients from the study were able to be successfully treated with ALK or NTRK targeted drugs.

These results could mean new hope for families affected and are set to change the World Health Organization’s diagnostic guidelines with brain tumors in infants to be classed separately from other childhood brain tumors.

"Chemotherapy is currently a good treatment option for babies with brain tumors – but our study has found that some children could be spared this treatment," said Dr. Matthew Clarke, Clinical Research Fellow in the Glioma Team at The Institute of Cancer Research, London. "With further testing, I’m hopeful that existing targeted drugs could expand our arsenal of options to treat these smallest of patients."

On April 7, 2020 Cancer Research UK has reported that it has taken the difficult decision to cut funding to its existing grants and institutes by up to 10% and its national network of Centres by around 20% (Press release, Cancer Research UK, APR 7, 2020, View Source [SID1234556252]). This works out as a £44 million cut to its research portfolio across the year, Iain Foulkes, Professor Karen Vousden, and Professor Charles Swanton wrote in an open letter to its researcher community.

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"Our mission is so important to people all over the UK and by helping the global effort of tackling COVID-19, we hope we can get back to beating cancer as soon as possible." – Iain Foulkes, executive director of research and innovation at Cancer Research UK
Iain Foulkes, executive director of research and innovation at Cancer Research UK, said: "COVID-19 has left the whole world in uncharted waters. And the unprecedented measures to control the global COVID-19 pandemic have had a huge impact on both our researchers’ ability to carry on in the lab, and on our ability to fundraise. Faced with a predicted loss of 20-25% of fundraising income, we are forced to look for savings across our current portfolio.

"Cancer Research UK funds nearly 50% of the cancer research in the UK and making cuts to research funding is the most difficult decision we have had to make. We don’t do so lightly. We have worked hard to ensure the cuts are limited and give our researchers flexibility in how to make them. Ultimately, it is our research that delivers benefit to people affected by cancer, and this remains our first priority. We are hopeful that limiting our spending now will enable us to continue funding life-saving research in the long run.

"Cancer doesn’t go away during or after COVID-19, but we’re incredibly proud of our community of researchers who have been very quick to respond to the crisis, using their kit, skills and talent to support the NHS and the COVID-19 response. Our mission is so important to people all over the UK and by helping the global effort of tackling COVID-19, we hope we can get back to beating cancer as soon as possible."