On June 17, 2020 Ipsen (Euronext: IPN; ADR: IPSEY) reported the United States Food and Drug Administration (FDA) has granted the company Fast Track designation for the investigational use of liposomal irinotecan (ONIVYDE) in combination with 5- fluorouracil/leucovorin (5-FU/LV) and oxaliplatin (OX) together, known as NALIRIFOX for patients with previously untreated, unresectable, locally advanced and metastatic pancreatic ductal adenocarcinoma (PDAC) (Press release, Ipsen, JUN 17, 2020, View Source [SID1234561162]). The FDA’s Fast Track program facilitates the development and expedites the review of drugs that treat serious conditions and have the potential to address an unmet medical need.

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The final analysis from the multicenter, open-label Phase 1/2 study will be presented as a late-breaking oral presentation at the first time ever virtual ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer on July 1, 2020 and will include data on primary and secondary endpoints. Ipsen has also initiated patient enrollment in the international Phase 3 NAPOLI-3 clinical study investigating the safety and efficacy of NALIRIFOX versus gemcitabine + nab-paclitaxel in the first-line setting (NCT04083235).

ONIVYDE is approved in the United States and in Europe in combination with fluorouracil (5-FU) and leucovorin (LV) for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy. ONIVYDE is not indicated as a single agent for the treatment of patients with metastatic adenocarcinoma of the pancreas.

"Since the initial approval of ONIVYDE in metastatic pancreatic cancer, we have continued to dedicate our research efforts to better understand the needs of pancreatic cancer patients. Through ongoing clinical investigations and exploratory real-world analyses, we have sought to determine whether patients who receive active treatment early have an improvement in survival," said Howard Mayer, M.D., Executive Vice President, Head of Research and Development at Ipsen. "As we continue to enroll additional patients in the ongoing Phase 3 NAPOLI-3 clinical study, we look forward to working closely with the FDA to potentially bring ONIVYDE to more pancreatic cancer patients earlier in the disease."

Pancreatic cancer is a rare and deadly disease that accounts for about 3% of all cancer and 7% of all cancer deaths.1 Of the 57,600 people with pancreatic cancer in the United States, more than half are diagnosed with metastatic disease, which has an overall 5-year survival rate of three percent (3%).1

Programs with Fast Track designation may benefit from early and frequent interactions with the FDA over the course of drug development. In addition, the Fast Track designation program allows for the eligibility for accelerated approval and priority review if relevant study criteria are met and enables a company to submit individual sections of a New Drug Application (NDA) for review on a rolling-submission basis.

On June 17, 2020 STORM Therapeutics, the biotechnology company focused on the discovery of small molecule therapies modulating RNA epigenetics, reported that it has published a scientific paper in the peer reviewed journal Nature Reviews Cancer (Press release, STORM Therapeutics, APR 17, 2020, View Source [SID1234561037]).

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The paper entitled ‘Role of RNA modifications in cancer’ is an authoritative and comprehensive review of the emerging and highly promising field of RNA epigenetics, highlighting pathways implicated in cancer to-date, describing their biological functions and their connections to the disease. The paper, written by Tony Kouzarides and Isaia Barbieri from The Gurdon Institute, The University of Cambridge describes insights into seven different internal RNA modifications, their mechanisms of actions (where known) and the evidence linking them to cancers. See link to paper: View Source

Professor Tony Kouzarides, Founder of STORM Therapeutics and Deputy Director of Gurdon Institute, University of Cambridge, said: "This paper describes the importance of understanding key RNA epitranscriptomic pathways and their implications for a wide range of cancers. It highlights that an increasing number of RNA modifications and the enzymes responsible for their deposition, removal and detection (or writers, erasers and readers) play context-specific roles in a rapidly expanding catalogue of cancers. They are expected to be tractable for development of targeted small molecule therapies for a wide range of those cancers and could additionally enhance the efficacies of current cancer treatments by affecting primary and acquired drug resistance."

Keith Blundy, CEO of STORM Therapeutics, said: "I am delighted to see the publication by our Company Founder of this paper in a world leading journal. The diversity of RNA modifications and the molecular pathways in which they are involved demonstrates this is only the beginning of the era of RNA epigenetics in cancer therapy and treatment of a wide range of other diseases. STORM Therapeutics is the leading company currently tackling disease through modulating RNA modifying enzymes and is developing a unique platform to address these enzyme classes, including RNA methyltransferases. We expect our lead drug candidate, METTL3, to be the first RNA epigenetic drug to enter human clinical trials in 2021."

On June 16, 2020 The Lead Discovery Center GmbH (LDC) and the South Korean drug development specialist Qurient reported they have founded QLi5 Therapeutics GmbH, together with the LDC’s partners, the Max Planck Society (MPG) and Nobel laureate Robert Huber, emeritus director of the Max Planck Institute for Biochemistry (Press release, QLi5 Therapeutics, JUN 16, 2020, View Source [SID1234654542]). Dortmund based QLi5 Therapeutics licensed novel proteasome inhibitors from LDC and its partners. QLi5 Therapeutics will advance these towards preclinical and clinical development for the treatment of cancers and inflammatory and autoimmune disorders.

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The novel proteasome inhibitors were generated within a long standing and successful collaboration between the LDC and Robert Huber. Combining Huber’s vast expertise in the field of proteasome with the LDC’s drug discovery capabilities, the partners have created a portfolio of proteasome inhibitors with unique binding characteristics and favourable pharmacodynamic properties. The further development in the joint venture with Qurient is the outcome of a successful strategic partnership between Qurient, LDC and Max Planck.

"The proteasome is a real treasure chest," Robert Huber comments, "and the launch of Qli5 Therapeutics enables us to harness its potential for the treatment of many diseases. Our new generation of proteasome inhibitors is set to overcome key challenges that have so far hampered a broader application of the first generation of covalently acting proteasome inhibitors." The proteasome plays an important role in cell regulation by degrading proteins and represents a well established clinical target for the treatment of liquid tumours, in particular multiple myeloma.

"We believe the LDC’s innovative proteasome inhibitors hold exceptional potential, and we are excited to be part of QLi5 Therapeutics to jointly advance them towards preclinical and clinical testing," Kiyean Nam, CEO and CSO of Qurient adds. "We very much appreciate the LDC as long-term partner and prime source of external innovation." Over the last years, Qurient has licensed two other inhibitor projects from the LDC, targeting the kinases, Axl and CDK7 respectively. Both leads have made considerable progress since, e.g. nomination for clinical development.

"The foundation of Qli5 Therapeutics is the current peak of our trustful and long-term collaborations with Robert Huber and Qurient, our exceptionally strong and committed South Korean partner," Bert Klebl, CEO and CSO of the LDC comments. "This joint venture is wonderful step forward in our mutual relationship and it is a great opportunity to translate the potential of the proteasome complex into more tangible benefits for patients."

"Having previously licensed assets to Qurient, we are delighted to now jointly start-up a venture with Qurient. QLi5 provides excellent prospects to enable a much needed next generation of proteasome inhibitors. Bringing the company on track together with Qurient has been a swift and smooth endeavour, adds Dieter Link, Licensing manager at Max Planck Innovation GmbH.

On June 16, 2020 Monopar Therapeutics Inc. (Nasdaq: MNPR) and NorthStar Medical Radioisotopes, LLC reported a 50/50 collaboration to develop potential Radio-Immuno-Therapeutics (RITs) to treat severe COVID-19 (patients with SARS-CoV-2 infection) (Press release, Monopar Therapeutics, JUN 16, 2020, View Source [SID1234611968]). Monopar is a clinical-stage biopharmaceutical company and NorthStar is a commercial producer and supplier of medical radioisotopes. This collaboration combines NorthStar’s expertise in the innovative production, supply, and distribution of important medical radioisotopes with Monopar’s expertise in therapeutic drug development and its pre-IND stage humanized urokinase plasminogen activator receptor (uPAR) targeted monoclonal antibody known as MNPR-101, along with a proprietary portfolio of related monoclonal antibodies that target uPAR or its ligand uPA.

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The uPA system (comprised of the serine protease enzyme uPA and its receptor uPAR) has been demonstrated to be selectively expressed on aberrantly activated immune cells. In response to coronavirus infection, these rogue immune cells produce pro-inflammatory cytokines that can cause runaway inflammation throughout the body, commonly referred to as a cytokine storm. It is this systemic hyper-inflammatory state that is thought to be largely responsible for the severe lung injury and further multiple organ damage that contributes to poor outcomes and death in patients with severe COVID-19.

In this collaboration, Monopar and NorthStar plan to couple MNPR-101 to a therapeutic radioisotope supplied by NorthStar in order to create a highly selective agent that has the potential to kill aberrantly activated cytokine-producing immune cells. By eradicating these cells with a targeted RIT, the goal is to spare healthy cells while quickly reducing the cytokine storm and its harmful systemic effects.

"This collaboration is a powerful combination of unique scientific and technical expertise to help combat severe COVID-19," said Chandler Robinson, MD, CEO of Monopar Therapeutics. "The companies will be conducting a development program to determine if Monopar’s uPA/uPAR monoclonal antibodies can be transformed into RITs that are effective as treatments against severe COVID-19, as well as other corona viruses."

"We are pleased to work together with Monopar in the battle against COVID-19," said Stephen Merrick, CEO of NorthStar Medical Radioisotopes." Our hope is that, by joining forces, we can develop a targeted radiopharmaceutical treatment that has the ability to prevent patients with severe COVID-19 from being placed on ventilators and from dying."

"To help mitigate the cytokine storm and reduce deaths associated with COVID-19, our goal is to develop an RIT that selectively eliminates the rogue aberrantly activated immune cells that produce these cytokines," said Andrew Mazar, PhD, Chief Scientific Officer of Monopar. "uPAR is selectively expressed on these rogue immune cells but not on healthy cells. An antibody carrying a therapeutic radioisotope could gain entry into these cells through uPAR and deliver its cytotoxic payload to kill these cells while sparing normal tissue."

"NorthStar will apply its expertise to identify and supply the optimal therapeutic radioisotope to couple with Monopar’s uPAR monoclonal antibodies," said James T. Harvey, PhD, Senior Vice President and Chief Science Officer of NorthStar. "We will deploy the most appropriate development approaches and production technology to ensure that both development and, if successful, commercial-scale volumes of this radioisotope can be supplied in order to meet potential demand."

This targeted therapeutic approach to treating severe COVID-19 is supported by a recently published study (Rovina et al. 2020) demonstrating that soluble urokinase plasminogen activator receptor (suPAR), which is shed by aberrantly activated immune cells that make uPAR, is an early predictor of severe respiratory failure in COVID-19 and its presence increases as patients develop severe COVID-19.

On June 16, 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 audited preliminary results for the year ended 31 December 2019 (Press release, Midatech Pharma, JUN 16, 2020, View Source [SID1234562738]).

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Financial highlights

·Total gross revenue(1) for the year of £0.7m (2018: £1.9m, 2017: £1.0m).

·Statutory revenue(2) for 2019 of £0.3m (2018: £0.1m, 2017: £0.1m).

·Subscription, Placing and Open Offer in February 2019 raised £12.3m (net) and Registered Direct Offering in the US in October 2019 raised $2.5m (£1.8m) (net).

·Receipt of €3.6m (£3.1m) (net) non-dilutive Reindus loan and award of Guazatu loan of €1.5m.

·Provisional award of a GlioKIDS grant of €2.7m (£2.3m), subject to confirmation of Midatech’s status as an SME, to support a Phase II trial of MTX110.

·Cash and deposits at 31 December 2019 of £10.9m (2018: £2.3m, 2017: £13.2m).

·Net loss from continuing operations of £9.1m (2018: £10.4m loss, 2017: £11.7m loss) with net cash inflow in the year of £8.4m (2018: £10.9m outflow, 2017: £4.1m outflow).

·Tax credit receivable of £1.8m (2018: £1.9m, 2017: £1.2m).

1)Total gross revenue represents collaboration income from continuing operations plus grant revenue.

2Statutory Revenue represents total gross revenue, excluding grant revenue.

Operational highlights

·First substantive licensing agreement with China Medical System Holdings Ltd ("CMS") for the Group’s pipeline products for Greater China accompanied by an £8.0m strategic investment in the Company, as part of a Subscription, Placing and Open Offer executed in February 2019.

·MTX110 received orphan drug designation for malignant glioma including DIPG from the FDA.

Post period end highlights

·In January 2020, a study of subcutaneous administration of MTD201 compared with traditional intramuscular administration in healthy volunteers showed similar pharmacokinetics and bioavailability, offering the potential for a differentiated, more patient-friendly product profile.

·In March 2020, an exploratory study was initiated by Columbia University in five patients with DIPG using an alternative convection enhanced delivery system.

·Also in March 2020, following a General Meeting, the Company’s ordinary shares of £0.00005 each were consolidated on a one-for-20 basis into ordinary shares of £0.001 each. At the same meeting a resolution was passed to change the ratio of the Company’s American Depositary Receipts ("ADRs"). This will change from one ADR representing 20 Existing Ordinary Shares to one ADR representing five new ordinary shares.

·On 31 March 2020, the Company announced a wide-ranging strategic review including termination of MTD201, closure of the Company’s Bilbao operations and a re-alignment of the Board.

·On 20 April 2020, the Company announced an update to the strategic review including the appointment of an adviser and start of a ‘formal sale process’ under the Takeover Code.

·On 18 May 2020, the Company announced that it had raised gross proceeds of £4.3m (£3.8m net of expenses) in a combined UK Placing and Registered Direct Offering in the US. The combined offerings resulted in the issuance of 15.8 million new Ordinary Shares and 16.5 million new Warrants.

·On 8 June 2020, the Company received a letter sent on behalf of Secura Bio, Inc. ("Secura Bio"), dated 1 June 2020, purporting to terminate a License Agreement, dated 5 June 2017 (the "Secura License Agreement"), by and between Midatech Limited and Novartis AG, which Novartis AG subsequently transferred to Secura Bio. Pursuant to the Secura License Agreement, Midatech Limited was granted a non-exclusive worldwide, sublicenseable license to certain patents of panobinostat, the active pharmaceutical ingredient of the Company’s development product MTX110. Midatech Limited’s rights are limited to the treatment of brain cancer in humans, administered by convection-enhanced delivery. The Company plans to continue to pursue development of MTX110 and the strategic review process previously disclosed. The Company is also reviewing with its outside counsel remedies it may have if Secura Bio does not withdraw the notice and otherwise cease to interfere with its ongoing business and strategic review process, which the Company has formally requested. The Company is evaluating available actions to protect its rights under the Secura License Agreement and its assets.

Stephen Stamp, CEO and CFO commented "This has been an extremely difficult period for Midatech with the termination of in-house development of our lead programme, closure of our Bilbao operations and the loss of 47 jobs, over two-thirds of our employees. I should like to recognise the professionalism of the team in making these difficult decisions and the grace with which they have been accepted. Our focus now is to evaluate all available options for extracting maximum value from Midatech’s platform technologies."