On March 2, 2020 ITM Isotopen Technologien München AG (ITM), a biotechnology and radiopharmaceutical group of companies reported, that it has expanded a long-term agreement for the supply of highly pure no-carrier-added (n.c.a.) Lutetium-177 (177Lu), which was originally concluded in 2018 between ITM AG and Endocyte, Inc., a Novartis company (Press release, ITM Isotopen Technologien Munchen, MAR 2, 2020, View Source [SID1234555073]). Under the agreement, ITM will provide Endocyte with supply of n.c.a 177Lu for its investigational 177Lu-PSMA-617 radioligand therapy for patients with metastatic castration-resistant prostate cancer. Pending approval of investigational drug candidate 177Lu-PSMA-617, ITM would continue to provide its proprietary n.c.a. 177Lu during the commercial phase, supporting the scalability and security-of-supply for patients world-wide.

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In addition, the parties have agreed that n.c.a. 177Lu supplied by ITM will be used in future pipeline investigations.

Sidonie Golombowski-Daffner, President of Advanced Accelerator Applications, the radioligand business of Novartis, stated: "We believe in the potential of targeted radioligand therapy to transform the lives of patients and aspire to make it broadly available to those in need. This supply agreement supports our future growth and ability to investigate radioligand therapies in a variety of cancers."

Steffen Schuster, CEO of ITM, commented: "We are very pleased to expand this long-term alliance, which allows us to further expand and support the development and increasing global patient access to targeted radioligand therapies. We are proud to contribute to these treatments by providing our highly pure n.c.a. 177Lu. By 2030, therapeutic radiopharmaceuticals are expected to account for 50% of the nuclear medicine market. Thanks to the extension of the Endocyte supply agreement, we are confident that we can make a significant impact to this growing demand."

On March 1, 2020 MPM Capital (MPM) and Dana-Farber Cancer Institute have jointly reported on February 26, 2020 the successful closing of the MPM Oncology Innovations Fund (INV) with $100M in capital, and the Dana-Farber Innovations Research Fund (IRF) with more than $26M in pledged donations (Press release, MPM Capital, MAR 1, 2020, View Source [SID1234555005]). 3SBIO, a leading biopharmaceutical company in China, is a limited partner in the INV and has agreed to make donation to the IRF, to support early-stage oncology research at Dana-Farber. The collaboration of INV and IRF is a unique, first of its kind, impact investing collaboration.

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Through INV, MPM intends to create and invest in early-stage companies developing innovative therapeutic technologies in oncology. MPM expects 50% of the capital from INV to be invested in new companies generated from Dana-Farber research. As part of the collaboration, MPM, through INV, has the right of first offer to license certain Dana-Farber technologies that have been identified for commercialization.

"With innovative new funding structures, such as the collaboration between MPM and Dana-Farber, we believe we can expand and accelerate important research efforts while putting in place a path for developing this research into novel therapeutics. We are honored to partner with one of the world’s leading cancer research centers to execute on this strategy and, having partnered before to bring groundbreaking science to patients, we are excited to continue working with our esteemed colleagues at Dana-Farber to advance the search for cancer cures," said Ansbert Gadicke, co-founder and Managing Director at MPM Capital.

"We are thrilled to have partnered with MPM on this innovative venture philanthropy model. We have generated substantial philanthropic support for exciting areas of cancer research at Dana-Farber, and MPM will support the creation of biotech companies that seek to bring new and advanced treatments to our patients," said Laurie H. Glimcher, MD, President and Chief Executive Officer of Dana-Farber.

"3SBIO is pleased to invest in MPM’s INV as a limited partner and to make donation to IRF to support Dana-Farber, a top cancer research and treatment center in the world. We are looking forward to this collaboration to develop many promising oncology therapies that address the unmet medical needs of cancer patients," said Dr. Jing Lou, Chairman and CEO of 3SBIO.

MPM’s partnership with Dana-Farber comes as biotechnology venture investors seek access to the most-promising academic discoveries and as medical institutes pursue new ways to fund research and propel their innovations into the market

On February 28, 2020 Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to developing and commercializing new medicines for patients battling cancer, reported a new strategic direction to accelerate the advancement of certain of its clinical development programs (Press release, Verastem, FEB 28, 2020, View Source [SID1234554989]). The Company’s primary focus will be on the development of CH5126766 (VS-6766), its RAF/MEK inhibitor, in combination with defactinib, its focal adhesion kinase (FAK) inhibitor, for the treatment of KRAS mutant solid tumors. Verastem Oncology will also continue to advance the development of duvelisib (COPIKTRA) for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL).

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"With our newly expanded development pipeline and strengthened balance sheet, we believe this new strategic direction will be transformative for Verastem Oncology as we will have the opportunity to rapidly advance the development of the clinical programs that we believe will yield the greatest results for patients, physicians and shareholders," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We are honored to have leading life science investors participate in our recently announced private placement. Verastem Oncology’s mission is centered on improving the lives of cancer patients and we believe our work in collaboration with the scientific community has presented significant opportunity to make further meaningful strides in areas of critical need."

Accelerating Development of CH5126766 (VS-6766) and Defactinib Combination

In early 2020, Verastem Oncology licensed exclusive global development and commercialization rights to CH5126766 (VS-6766), a unique and promising inhibitor of the RAF/MEK signaling pathway. The combination of CH5126766 (VS-6766) and defactinib is currently being investigated in a Phase 1 clinical study and expansion cohorts in patients with KRAS mutant advanced solid tumors, including low grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). Data from this Phase 1 study have been submitted for presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2020 Annual Meeting. Verastem Oncology plans to initiate discussions with regulatory authorities during the first half of 2020, with the goal of commencing a registration-directed trial as soon as possible.

Advancing Duvelisib in Relapsed/Refractory PTCL

At the American Society of Hematology (ASH) (Free ASH Whitepaper) 2019 Annual Meeting, Verastem Oncology presented positive data from the dose optimization portion of the Phase 2 PRIMO study evaluating duvelisib in patients with relapsed or refractory PTCL, an aggressive disease with a lack of effective therapeutic options. This initial phase of the trial demonstrated promising clinical activity including complete and durable responses, as assessed by independent central review, with a manageable safety profile. The expansion phase of this registration-directed study continues to accrue patients and Verastem Oncology expects to complete enrollment in 2020 and report top-line results from the expansion cohorts in early 2021. Verastem Oncology intends to build on the existing Fast Track and Orphan Drug Designations and submit a regulatory package to the U.S. Food and Drug Administration to expand the approved indications for COPIKTRA to include relapsed or refractory PTCL.

Focusing COPIKTRA Commercial Activities

Verastem Oncology generated preliminary unaudited COPIKTRA net product revenue of $3.6 million for the fourth quarter of 2019, compared to $4.0 million for the third quarter of 2019, and $12.3 million for the full year 2019. Net sales were impacted by timing of purchases and gross to net adjustments associated with Medicare Part D ("donut hole"). Demand units increased 20% from third quarter to fourth quarter 2019. Going forward, Verastem Oncology will be reducing the resources directed to the promotion and sale of COPIKTRA in its current indications, including reducing the size of its salesforce and non-core clinical research. Verastem Oncology plans to shift its COPIKTRA promotional resources toward large, community-based practices and academic institutions, which represent the majority of the appropriate third-line patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and follicular lymphoma (FL).

Financial Benefits of the Strategic Realignment and 2020 Financial Outlook

As a result of this strategic realignment, Verastem Oncology expects to reduce its operating expenses by approximately 40% for 2020 compared to 2019. Based on its current operating plans, Verastem Oncology expects its research and development and selling, general and administrative expenses for the full year 2020 to be in the range of $70 million to $85 million. As of December 31, 2019, Verastem Oncology had preliminary unaudited cash and short-term investments of $111.3 million. As announced today, Verastem Oncology anticipates completing a private placement of approximately 46.5 million shares of its common stock at an offering price of $2.15 per share on March 3, 2020, resulting in gross proceeds of approximately $100 million to Verastem Oncology before deducting expenses to the placement agents and other estimated offering expenses. Verastem Oncology expects that its existing cash and cash equivalents, along with the revenue it expects to generate from COPIKTRA, will be sufficient to fund its planned operations into the fourth quarter of 2021.

Conference Call and Webcast Information

The Verastem Oncology management team will host a conference call and webcast today, Friday, February 28, 2020, at 8:00 AM ET. The call can be accessed by dialing (877) 341-5660 (U.S. and Canada) or (315) 625-3226 (international), five minutes prior to the start of the call and providing the passcode 4164157.

The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company’s website at www.verastem.com. A replay of the webcast will be archived on the Company’s website for 90 days following the call.

About CH5126766

CH5126766 (VS-6766) (previously referred to as CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, CH5126766 (VS-6766) blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows CH5126766 (VS-6766) to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The combination of CH5126766 (VS-6766) and the focal adhesion kinase (FAK) inhibitor defactinib is currently being investigated in a clinical study (Phase 1 followed by expansion cohorts) with the expansion cohorts now ongoing in patients with KRAS mutant advanced solid tumors, including low grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). The ongoing clinical study of the CH5126766 (VS-6766)/defactinib combination is supported by single-agent Phase 2 studies which investigated defactinib in KRAS mutant NSCLC and CH5126766 (VS-6766) in KRAS mutant NSCLC and LGSOC.

About Defactinib

Defactinib is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2 A Phase 1/2 clinical trial of defactinib in combination with CH5126766 (VS-6766) in patients with KRAS mutant advanced solid tumors, including low grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC) is underway.3 The CH5126766 (VS-6766)/defactinib combination is supported by single-agent Phase 2 studies which investigated defactinib in KRAS mutant NSCLC4 and CH5126766 (VS-6766) in KRAS mutant NSCLC and LGSOC.5 Defactinib is also in clinical testing in combination with pembrolizumab for treatment of patients with pancreatic cancer, NSCLC and mesothelioma.6

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.7,8,9

COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status and Orphan Drug Designation, and is being investigated in combination with other agents through investigator-sponsored studies.10 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On February 28, 2020 Cancer Research UK and its European partners, Fondazione AIRC and Asociación Española Contra el Cáncer (AECC), reported that it will invest £27.4 million into seven new international projects to accelerate lifesaving cancer research, announced today (Friday) (Press release, Cancer Research UK, FEB 28, 2020, View Source [SID1234554923]).

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"If current trends continue, the world will see a 60% increase in cancer cases over the next two decades. Cancer is a global problem and no one country can tackle it alone." – Dr Iain Foulkes, Cancer Research UK
The Cancer Research UK Accelerator Awards programme, which provides five years of funding for international projects, reflects a longstanding link between the UK and Europe, working together to drive progress for people affected by cancer.

The successful teams will develop new research tools, resources and infrastructure to improve scientists’ understanding of cancer, including rare and hard-to-treat cancers, helping to find better ways to treat them. Projects include investigating how to best combine radiotherapy with the latest immunotherapies, and refocussing research efforts on mesothelioma, an asbestos related cancer, cases of which continue to rise globally.*

Dr Iain Foulkes, executive director of research and innovation at Cancer Research UK, said: "If current trends continue, the world will see a 60% increase in cancer cases over the next two decades. Cancer is a global problem and no one country can tackle it alone.

"Now the UK has left the European Union, the need to retain collaborative cancer research between the EU and the UK has never been greater. This partnership will also strengthen UK cancer research by the sharing of expertise, new technologies and research talent."

Professor Paul French, a physicist based at Imperial College London, will lead one of the projects to improve the way we develop and test anti-cancer drugs in the lab.

Traditionally, drug discovery research has used thin ‘2D’ layers of cells that can be easily controlled and imaged on conventional microscopes. However, such simplified disease models don’t adequately represent the complex 3D cancer environment within the body.

Professor French said: "The complexity of cancer is beyond our current understanding and existing therapies. By developing technology to investigate more sophisticated 3D cancer models, which allow us to explore how drugs work on tumour cells interacting with their local environment, we could find new ways to overcome challenges like drug resistance."

Professor Kevin Blyth, a clinician scientist based at the University of Glasgow, will lead another of the projects, focussed on finding better treatments for mesothelioma, a hard-to-treat asbestos related cancer that most commonly starts in the layers of tissue that cover each lung.

Professor Blyth said: "It’s been difficult to build a network of scientists, with enough cases of the disease to build a thorough understanding of how to best treat mesothelioma. And this has left people with the disease very few treatment options.

"In the future it’s likely that the global incidence of mesothelioma will continue to increase, as there are many countries that still don’t regulate the use of asbestos. So, it’s important to build a research community now and improve our understanding to help those affected by this cancer."

The Accelerator Awards bring together world-class researchers who would not previously have been able to work together, without the unique structure of this grant scheme. Each collaboration aims to deepen the research community’s understanding of cancer and bring people with cancer closer to new treatments, making use of the expertise and diverse technologies of different partner organisations.

Ignacio Muñoz Pidal, president of the AECC, said: "The AECC believes international collaboration is essential to accelerate cancer research. The Accelerator Awards allow European researchers to not only receive five years of funding, but also enables them access to an international collaborative platform where they can share knowledge and data."

Professor Federico Caligaris Cappio, scientific director of Fondazione AIRC, said: "Rare tumours are individually of little interest to drug developers and are difficult to study because patients are few. For this reason, the knowledge we have of many of these cancers is fairly basic.

"For research to be as effective as possible, it is necessary to bring together expertise from several countries. This is one of the strengths of the Accelerator Awards that sees Fondazione AIRC, Cancer Research UK and AECC come together. Supporting research projects that are aimed at accelerating skills and seeking new and more effective therapeutic solutions."

The next round of the Accelerator Awards will open on 2 March for international research groups.

On February 28, 2020 KemPharm, Inc. (Nasdaq: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, provided an update on the KP415 NDA and reported its financial results for the fourth quarter and full-year ended December 31, 2019 (Press release, KemPharm, FEB 28, 2020, View Source [SID1234554990]).

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"Our highest priority at KemPharm is filing the New Drug Application (NDA) for KP415, our prodrug product candidate of d-methylphenidate (d-MPH) being developed for the treatment of attention deficit hyperactivity disorder (ADHD)," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "During the last several months, we have been working hand-in-hand with the team of advisors assembled by our partner, Gurnet Point Capital (GPC), with the primary goal of submitting the best NDA for review by the FDA (U.S. Food and Drug Administration). We believe this investment in time and additional review will improve the probability of acceptance and subsequent approval of KP415 with the best possible label. The timing for the filing of the KP415 NDA is subject to the discretion of GPC and we anticipate receiving their approval to proceed shortly. We intend to file as soon as possible and remain on track to submit the NDA this quarter."

Dr. Mickle continued, "Once the KP415 NDA is submitted to the FDA, our full attention will be focused on ensuring a successful completion of the regulatory review process with the goal of achieving approval for KP415 with the broadest label. KP415 has the potential to be the first truly differentiated methylphenidate-based product introduced to the ADHD market in several years. This market accounted for approximately $3.9 billion in sales in 2018, and we believe KP415’s potential attributes, including onset of action at 30 minutes, duration of effect of 13 hours, and substantially lower abuse potential than relevant d-methylphenidate comparators, could provide a solution to these unmet needs that have been repeatedly cited by both patients and prescribers."

Q4 and FY 2019 Financial Results:

For Q4 2019, KemPharm reported revenue of $1.4 million from research and development services, as compared to Q3 2019 revenue of $11.5 million, which was comprised of a one-time upfront payment of $10 million under the KP415 License Agreement (as defined below), and research and development services revenue of $1.5 million. This is KemPharm’s second sequential quarter reporting research and development services revenue, and the Company expects this trend to continue as the Company provides services to GPC and its affiliates under the license agreement by and between KemPharm and Commave Therapeutics, S.A., an affiliate of GPC, dated September 3, 2019 (the KP415 License Agreement).

KemPharm’s net loss for Q4 2019 was $6.0 million, or $0.18 per basic share and diluted share, compared to a net loss of $5.2 million, or $0.20 per basic and diluted share for the same period in 2018. Net loss for Q4 2019 was driven primarily by an operating loss of $4.4 million and net interest expense and other items of $1.8 million. The net operating loss of $4.4 million for Q4 2019 was a decrease of $4.9 million compared to $9.3 million in the same period in 2018, which was primarily due to revenue of $1.4 million, a decrease in research and development expenses of $3.8 million and a decrease in general and administrative expenses of $1.6 million, offset by royalty and direct contract acquisition costs of $1.9 million.

For FY 2019, revenue totaled $12.8 million, which is comprised of the $10.0 million upfront payment under the KP415 License Agreement, and research and development services revenue of $2.8 million. KemPharm’s reported net loss was $24.5 million, or $0.83 per basic and diluted share, compared to a net loss of $56.5 million, or $3.15 per basic and diluted share for the year ended December 31, 2018. FY 2019 net loss was driven primarily by operating loss of $20.3 million and net interest expense and other items of $6.2 million, partially offset by non-cash fair value adjustment income of $2.0 million. Operating loss for FY 2019 of $20.3 million was a decrease of $35.6 million compared to the FY 2018 operating loss of $55.9 million, which was primarily due to FY 2019 revenue of $12.8 million, a decrease in research and development expenses of $22.3 million, a decrease in general and administrative expenses of $1.7 million, and a decrease in severance expense of $1.6 million, offset by royalty and direct contract acquisition costs of $2.9 million.

As of December 31, 2019, total cash and investments, which is comprised of cash, cash equivalents, and restricted cash, was $3.6 million, which was a decrease of $3.4 million compared to September 30, 2019. Based on the Company’s current operating forecast, existing resources are sufficient to continue operations into, but not through Q3 2020. Existing resources combined with the potential milestones and revenue under the KP415 License Agreement have the potential to extend the cash runway into, but not through, Q1 2021.

"The measures we have taken to reduce our operating spend and restructure our debt obligations, as well as the potential milestones and revenue under the KP415 License Agreement, potentially extend our cash runway into, but not through, Q1 2021," stated LaDuane Clifton, KemPharm’s Chief Financial Officer. "We expect research and development services revenue to continue through FY 2020 and beyond as we provide consultation services to support our partner in their commercial preparation activities for KP415 and with the potential initiation of the product development plan for KP484."