On September 10, 2020 Midatech Pharma PLC (AIM: MTPH.L; Nasdaq: MTP), a drug delivery technology company focused on improving the bio-delivery and bio-distribution of medicines, reported its unaudited interim results for the six months ended 30 June 2020 (Press release, Midatech Pharma, SEP 10, 2020, View Source [SID1234564945]).

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OPERATIONAL HIGHLIGHTS (including post period end)

·In March, an exploratory study was initiated with MTX110 by Columbia University in five patients with DIPG using an alternative convection enhanced delivery system.

·In March, the Company announced a wide-ranging Strategic Review, updated in April to include a Formal Sale Process under the Takeover Code. The Formal Sale Process was subsequently terminated in July.

·In March, the decision was taken to terminate further in-house development of the MTD201 programme with immediate effect although the asset remains available for licensing. All activities connected with MTD201 have been wound down expeditiously and the manufacturing facilities in Bilbao have been closed. Following the termination of in-house development of MTD201, the Company realigned its strategy towards exploiting its Q-Sphera technology more broadly.

·In April, an exploratory study was initiated with MTX110 by the University of Texas, Houston in five patients with recurrent medulloblastoma.

·In June, the Company signed a research collaboration with Dr Reddy’s Laboratories Ltd under which Midatech is deploying its in-house expertise and Q-Sphera drug delivery platform to medicines nominated by Dr Reddy’s.

·In July, the Company signed a collaboration with an unnamed European affiliate of a global pharmaceutical company, to establish the application of the Q-Sphera platform to new modalities in drug delivery.

FINANCIAL HIGHLIGHTS (including post period end)

·Total revenue in H1 2020 was £0.17m (H1 2019: £0.45m). Total revenue represents income from R&D collaborations plus grant revenue.

·Research and development costs increased by 15% to £3.99m (H1 2019: £3.46m) as a result of lower MTX110 development costs, redundancy costs of £0.88m and write-down of Spain assets of £0.55m, offset by a negative share-based payment charge of £0.35m.

·Administrative expenses increased to £2.93m (H1 2019: £2.05m) and included £0.35m one-time costs associated with Spanish Government loans, £0.07m UK redundancy costs and a £0.51m increase in legal and professional fees.

·Impairment of intangible assets of £11.59m (H1 2019: Nil) related to the termination of further in-house development of MTD201 and associated IPRD and goodwill.

·Net cash used in operating activities (after changes in working capital) in H1 2020 was £7.09m, compared with £4.56m in H1 2019.

·In May, in a concurrent Registered Direct Offering in the US and a Placing in the UK, the Company raised £4.26m before expenses through the sale of 15.76m ordinary shares at £0.27 per share and warrants exercisable for 16.55m ordinary shares at £0.34 per share.

·In July, the Company raised an additional £5.75m before expenses in an oversubscribed UK Placing, including a Broker Option, through the sale of 21.3m ordinary shares at £0.27 per share with no warrants.

·The cash balance at 30 June 2020 was £4.33m.

On September 10, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Manuel Litchman, M.D., President and Chief Executive Officer, will participate in three virtual investor conferences in September 2020 (Press release, Mustang Bio, SEP 10, 2020, View Source [SID1234564961]). The company will also host virtual one-on-one meetings during the conferences.

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Details of the events are as follows:

H.C. Wainwright 22nd Annual Global Investment Conference: Presentation on Monday, September 14, 2020, at 2:30 p.m. EDT
Cantor Virtual Global Healthcare Conference: Fireside Chat on Wednesday, September 16, 2020, at 2:00 p.m. EDT
Oppenheimer Fall Healthcare Life Sciences & MedTech Summit: Fireside Chat on Monday, September 21, 2020, at 3:20 p.m. EDT
Live webcasts of the presentation and fireside chats will be available on the Events page of the Investors Relations section of Mustang’s website: www.mustangbio.com. Archived replays of the webcasts will be available for approximately 30 days following each presentation and fireside chat.

On September 10, 2020 Genelux Corporation, a privately-held biopharmaceutical company, reported that the abstract covering data from the VIRO-15 Phase 2 trial (NCT02759588) has been accepted for an Oral Plenary Session at the 2020 xDigital Global Annual Meeting of the International Gynecologic Cancer Society (IGCS) (Press release, Genelux, SEP 10, 2020, View Source [SID1234564978]). VIRO-15 assessed Olvi-Vec in combination with a platinum-based regimen in platinum-resistant/refractory ovarian cancer (PRROC) patients. These data are being presented on Friday, September 11, 2020.

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"We are encouraged by the Phase 2 data in PRROC patients, which show Olvi-Vec is well tolerated and demonstrated remarkable anti-tumor activity with durable responses in combination with a platinum-based regimen, especially in patients with difficult-to-treat platinum-refractory disease," said Robert Holloway, MD, principal investigator for VIRO-15 and Chair of Genelux’s Clinical Advisory Board on gynecologic cancers. "Translational analyses results point to Olvi-Vec-mediated immune modulation of the tumor microenvironment and long-term therapeutic effect with cytotoxic platinum-based chemotherapy."

Key findings in ­­­27 heavily pre-treated PRROC patients (median 4 prior lines; 48% platinum-resistant, 52% platinum-refractory) who had documented disease progression from their last line of therapy prior to enrollment are as follows (data of patients eligible for evaluation after initiation of chemotherapy):

Median Progression-free Survival (PFS) is 11.0 months (95% CI: 6.7 – 13.0) and PFS-6-month is 77%.
Objective Response Rate (ORR) by RECIST1.1 criteria is 54% [95% CI: 33-74%; 2 (8%) complete response (CR), 11 (46%) partial response (PR)]; median Duration of Response is 7.6 months; and 86% of patients achieved tumor shrinkage.
ORR by CA-125 tumor biomarker is 85% [95% CI: 65-96%; 10 (38%) CR, 12 (46%) PR]; and 96% of patients achieved decrease of CA-125.
There are no differences in PFS & ORR between platinum-resistant & -refractory patients.
Most common adverse events: Grades 1&2 (≥ 20% patients) were pyrexia 59%, nausea 48%, abdominal distension 44%, abdominal pain 44%, chills 37%, fatigue 33% and vomiting 26%; Grade 3 (≥ 2 patients) were abdominal pain 7% and hypophosphatemia 7%; Grade 4 (none).
Performance status was preserved or improved in 93% of patients while on subsequent platinum-based regimen.
Translational analyses data indicate Olvi-Vec engages the immune system and induces favorable immune response (such as large intraepithelial infiltration of CD4+ & CD8+ T cells into tumors) and gene expression changes to the tumor microenvironment to aid clinical reversal of platinum resistance.
"Genelux is excited about the potential of Olvi-Vec-primed immunochemotherapy to generate meaningful clinical responses and improve the quality of life of PRROC patients who currently lack effective treatment options," said Thomas Zindrick, J.D., President and CEO of Genelux. "A registration trial of Olvi-Vec-primed immunochemotherapy is being planned."

Oral Presentation Session Details
Title: Oncolytic Vaccinia (Olvi-Vec) Primed Immunochemotherapy in Heavily Treated Platinum-Resistant/Refractory Ovarian Cancer
Session Information: Plenary III
Date/Time: ­­­­­­­­­­­­­­­­­­­­­­­­­­­­­­Friday, September 11, 2020/ 7:25 a.m. Eastern Time/11:25 a.m. Coordinated Universal Time
Presenter: Robert W. Holloway, MD, Medical Director, Gynecologic Oncology, AdventHealth Cancer Institute, Orlando, FL, USA
Additional information can be found at www.igcs.org

About Olvimulogene Nanivacirepvec
Olvi-Vec is a proprietary, non-pathogenic oncolytic vaccinia virus, modified to increase its safety, tumor selectivity and anti-tumor activity. Virus-mediated oncolysis results in immunogenic cell death and triggers immune activation and memory for long-term immunotherapy against cancer. Clinical results in over 150 subjects treated in Genelux studies have shown Olvi-Vec is well tolerated with documented clinical benefits.

On September 10, 2020 The University of Texas MD Anderson Cancer reported that Results from a Phase II trial led by researchers at Center suggest that a combination of ipilimumab (anti-CTLA-4) plus nivolumab (anti-PD-1) can generate durable responses in a subset of patients with metastatic castration-resistant prostate cancer (mCRPC), an "immune-cold" cancer that does not typically respond well to immunotherapy (Press release, MD Anderson, SEP 10, 2020, View Source [SID1234564946]).

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In a cohort of patients without previous chemotherapy treatment, the overall response rate (ORR) was 25% and median overall survival (OS) was 19 months. In a post-chemotherapy cohort, the ORR was 10% and media OS was 15.2 months. Four patients (two in each cohort) achieved a complete response.

The results of the CheckMate 650 trial, published today in Cancer Cell, are the first report of combination immune checkpoint inhibitors in mCRPC. Early results from this study were presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. Based on the findings, alternate dosing regimens are now being evaluated in an expanded clinical trial to reduce treatment-related toxicities.

"Historically, prostate cancer has been very resistant to checkpoint inhibitors because it is immunologically cold with few tumor-infiltrating T cells," said principal investigator Padmanee Sharma, M.D., Ph.D., professor of Genitourinary Medical Oncology and Immunology. "These results suggest that a combination approach to increase T cell infiltration and then block inhibitory pathways may be a useful strategy for treating these patients. Going forward, we plan to optimize the schedule and dosing to improve the safety profile."

Designing a combination strategy

In previous research published in Nature Medicine, Sharma and colleagues discovered that prostate cancers deploy multiple mechanisms to dampen the anti-tumor immune response. Although anti-CTLA-4 therapy could recruit T cells, the tumor-infiltrating T cells elicited compensatory inhibitory pathways, including immune-suppressing proteins PD-L1 and VISTA.

This would explain why previous clinical trials evaluating single-agent checkpoint inhibitors have not been effective in treating patients with mCRPC, said Sharma, who co-directs MD Anderson’s immunotherapy platform, part of the institution’s Moon Shots Program.

The researchers hypothesized that combining anti-CTLA-4 (ipilimumab) with anti-PD-1 (nivolumab) may be effective in bringing T cells into the tumor and overcoming the resulting immunosuppressive response.

The multi-institution, open-label study enrolled 90 men with mCRPC, who received the combination therapy every three weeks. Patients were enrolled in two cohorts: one with and one without prior chemotherapy. Participants were 77.8% Caucasian, 10% Black/African-American and 12.2% other.

In addition to response rates, the combination therapy achieved disease control in 46.9% and 13.3% of patients, with a median progression-free survival of 5.5 and 3.8 months in the pre- and post-chemotherapy cohorts, respectively.

Despite the positive responses, grade 3 and 4 treatment-related adverse events occurred in 42.2% of pre-chemotherapy patients and 53.3% of post-chemotherapy patients. The most common of these events was diarrhea, pneumonitis, colitis and increased lipase. Treatment-related adverse events led to discontinuation of therapy in a total of 31 patients. There were four treatment-related deaths, two in each cohort.

"There were patients who had clear benefit as a result of treatment, but there also were patients who had serious adverse events, which led us to amend the protocol to evaluate alternate schedules and doses and improve the safety of this approach," said Sharma.

Based on these data, the trial has been expanded to include more than 400 patients, with different dosing and schedules to identify strategies that can improve efficacy and minimize toxicities.

Exploring biomarkers associated with response

The researcher team also conducted analyses to identify potential biomarkers associated with clinical outcomes in these patients.

While this study represents a small number of patients, their findings suggest that the combination may be more effective in patients with a relatively high tumor mutational burden (TMB). This is in agreement with previous work that suggests certain patients with mCRPC may respond to checkpoint blockade despite having low TMB relative to other cancers, such as melanoma and lung cancer.

"The current study represents the first step in trying to identify mCRPC patients who would benefit from combination therapy with ipilimumab plus nivolumab based on chemotherapy exposure as well as preliminary biomarker analyses," said co-author Sumit Subudhi, M.D., Ph.D., assistant professor of Genitourinary Medical Oncology. "The data generated to date are encouraging, but we clearly have more work to do in the expansion cohort as we try to administer effective combination strategies with fewer toxicities."

This study was supported by Bristol-Meyers Squibb and ONO Pharmaceutical Company, Limited. Sharma is a member of the Parker Institute for Cancer Immunotherapy (PICI) and co-director of PICI at MD Anderson. A full list of authors and their disclosures can be found with the paper here.

On September 10, 2020 TRACON Pharmaceuticals (Nasdaq: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that Charles Theuer, M.D., Ph.D., President and CEO, will present a corporate overview at the H.C. Wainwright 22nd Annual Global Investment Conference on Wednesday, September 16th, at 12:00 pm EDT (Press release, Tracon Pharmaceuticals, SEP 10, 2020, View Source [SID1234564963]).

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To access a live webcast of the presentation, please visit the "Events and Presentations" page within the "Investors" section of the TRACON Pharmaceuticals website at www.traconpharma.com. A replay of the webcast will also be available following the event.