On January 27, 2020 GT Medical Technologies, Inc., a company dedicated to improving the lives of patients with brain tumors, reported that the U.S. Food and Drug Administration (FDA) has cleared an expanded indication for GammaTile Therapy (Press release, GT Medical Technologies, JAN 27, 2020, View Source [SID1234553566]). Patients with newly diagnosed malignant brain tumors are now eligible to receive the FDA-cleared surgically targeted radiation therapy (STaRT).

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"We are pleased to offer GammaTile Therapy to patients who are newly diagnosed with malignant brain tumors, in addition to patients with recurrent brain tumors," said Matthew Likens, president and CEO of GT Medical Technologies. "This is a significant step forward that expands our ability to improve the lives of patients with brain tumors. Patients receiving GammaTile Therapy immediately after the removal of a brain tumor will have the peace of mind that they are accelerating their radiation treatment and targeting residual tumor cells where treatment is most needed to help prevent recurrence."

Approximately 700,000 Americans are living with some type of brain tumor each year.1 Despite the efforts of the most skilled brain tumor specialists throughout the world, outcomes for patients with brain tumors have improved very little over the past 30 years. During this time, there have been only four FDA-approved drugs and two FDA-cleared devices available to patients and physicians for the treatment of brain tumors. GammaTile Therapy, which became available to patients with recurrent brain tumors in January 2019, is the most recent treatment cleared or approved by the FDA for the treatment of brain tumors.

GammaTile Therapy is the only radiation therapy specifically designed for use in the brain and offers advantages for patients undergoing surgery for brain tumors. GammaTile begins targeting residual tumor cells immediately at the time of tumor removal surgery, rather than waiting several weeks for surgical wound healing before beginning treatment. GammaTile protects healthy brain tissue while delivering a targeted dose to any remaining tumor cells. The unique design also limits side effects typically associated with radiation therapy, including hair loss. Additionally, the burden of radiation treatment is reduced for patients treated with GammaTile Therapy. These patients receive their course of radiation while going about their daily lives, requiring no additional trips to the hospital or clinic for radiation therapy.

In a clinical study, GammaTile Therapy gave the average patient approximately ten extra months without a local recurrence with extended overall survival.2 Clark C. Chen, M.D., Ph.D., head of the Department of Neurosurgery at the University of Minnesota Medical School, presented data from his first patients treated with GammaTile at the 2019 Society of Neuro-Oncology (SNO) Annual Meeting. Consistent with data published in a peer-reviewed article, Chen reported that local control was achieved in approximately 90% of patients who underwent gross total resection. This impressive result was achieved without an increase in wound complications or length of hospital stay. "GammaTile Therapy is an important addition to the armamentarium of treatments available against brain cancer," said Dr. Chen. "It has the potential to improve the quality of life as well as clinical outcome. I am encouraged by the FDA’s decision to expand the indications for this therapy."

GammaTile Therapy became available to patients in January 2019 and is being used in top cancer treatment centers across the United States. Dr. Vincent DiNapoli, neurosurgeon and director of the Brain Tumor Center at The Jewish Hospital in Cincinnati, Ohio, has been treating patients with GammaTile Therapy since August 2019. "Having been encouraged by the outcomes for recurrent brain tumor patients treated with GammaTile Therapy in my practice, I am excited by the FDA’s decision to expand this technology to the many patients who can benefit from the treatment during their initial diagnosis," Dr. DiNapoli said. "This could potentially create a more meaningful impact on patient outcomes, treatment compliance, and quality of life."

About GammaTile Therapy

GammaTile Therapy is an FDA-cleared, Surgically Targeted Radiation Therapy (STaRT) that is designed to delay tumor regrowth for patients with brain tumors. Roughly the size of a postage stamp, GammaTile Therapy features a bioresorbable, conformable, 3D-collagen tile and uniform radiation source, Cesium-131, provided by Isoray, Inc. (NYSE: ISR). GammaTile Therapy is placed at the end of excision surgery to immediately target residual tumor cells while limiting the impact of radiation on healthy brain tissue.

On January 27, 2020 NeoImmuneTech, Inc., a clinical-stage T cell-focused biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for the combination of NeoImmuneTech’s Hyleukin-7 (NT-I7) and Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of patients with relapsed/refractory (R/R) advanced solid tumors (Press release, NeoImmuneTech, JAN 27, 2020, View Source [SID1234553590]). This IND clearance allows NeoImmuneTech to initiate a Phase 1b/2a basket study evaluating this combination in patients with both checkpoint inhibitor (CPI)-treated and CPI-naïve R/R advanced solid tumors.

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"As a company, we are striving to develop a safe and effective immunotherapy for patients with R/R advanced solid tumors, including those who have not been responsive to single-agent CPIs," said NgocDiep Le, MD, PhD, Chief Medical Officer and Executive Vice President of NeoImmuneTech. "This clearance marks the next step towards our goal of combining Hyleukin-7 with advanced cancer therapeutics such as pembrolizumab."

The goal of this study is to establish a recommended dosing regimen and explore the preliminary anti-tumor activity of the combination in patients with both CPI-treated and CPI-naïve R/R tumors. The results of this study will be used to further clinical development of this combination in select tumor types. The study will be led by Aung Naing, MD, FACP, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center.

"We are constantly looking for better and safer treatment options for our patients, as only a fraction of them can benefit from currently available immunotherapies," said Dr. Naing. "This agent has the potential to augment and broaden the reach of current treatments such as pembrolizumab, due to its T cell amplifying mechanism of action. We hope this study yields results helpful for our patients in dire need."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA.

About Hyleukin-7

Hyleukin-7 (NT-I7), the only clinical-stage long-acting human IL-7, is uniquely positioned to address unmet medical needs in immuno-oncology. IL-7 is a fundamental cytokine for T-cell development and for sustaining immune response to chronic antigens (as in cancer). Hyleukin-7’s favorable PK/PD and safety profiles make it an ideal combination partner for immunotherapy standard of care (SOC) such as Checkpoint Inhibitor and CAR-T therapies. Hyleukin-7 is being studied in multiple clinical trials in solid tumors, and being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.

On January 27, 2020 AVEO Oncology (NASDAQ: AVEO) and Biodesix, Inc. reported the presentation of results from an investigator-sponsored Phase 1b trial of ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody product candidate, in combination with nab-paclitaxel and gemcitabine in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (PDAC) (Press release, AVEO, JAN 27, 2020, View Source [SID1234553589]). The results were presented during a poster session at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal (GI) Cancers Symposium. A copy of the presentation, titled "Phase 1b Study of Gemcitabine, Nab-paclitaxel, and Ficlatuzumab in Patients with Advanced Pancreatic Cancer" (abstract 693), is available in the Publications & Presentations section of AVEO’s website.

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The study, which was based on preclinical findings demonstrating a synergistic effect of the combination in a preclinical model of PDAC, was designed to determine the maximum tolerated dose of ficlatuzumab when combined with gemcitabine and nab-paclitaxel. Secondary outcome measures included response rate and progression free survival. A total of 24 patients were enrolled. The average number of 28-day cycles received was 7.5 (range 1-15), with 3 patients remaining on active treatment. The combination was associated with a promising durable response rate relative to expectations for gemcitabine and nab-paclitaxel alone. This included a 29% partial response (PR) rate and 92% rate of disease control (PR + stable disease). Treatment with this regimen was associated with significant hypoalbuminemia and edema, and therefore a follow up safety study is under consideration of ficlatuzumab in combination with an alternate cytotoxic regimen.

"Pancreatic cancer remains among the most challenging diseases to treat, owing to late diagnoses, rapid progression and early mortality," said Kimberly Perez, MD, Dana-Farber Cancer Institute. "By targeting the c-MET pathway, ficlatuzumab inhibits the Prrx1b-HGF signaling associated with pancreatic development, pancreatitis, and carcinogenesis. These Phase 1b results show encouraging responses that support the further study of ficlatuzumab in pancreatic cancer."

"Ficlatuzumab continues to emerge as a promising clinical candidate, with these results adding to a growing body of clinical data in acute myeloid leukemia and head and neck cancer," said Michael Bailey, president and chief executive officer of AVEO. "As we continue to execute on our tivozanib Phase 3 clinical and U.S. registration strategy and move closer to its potential commercialization, we look forward to seeing ficlatuzumab progress in multiple clinical studies, with the goal of determining a pivotal strategy, assuming favorable study outcomes."

"Ficlatuzumab continues to demonstrate the potential for major clinical utility in areas of significant unmet need," said Scott Hutton, Chief Executive Officer of Biodesix. "We look forward to continuing our diagnostic development work alongside AVEO to help realize the full potential of this promising therapeutic candidate."

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in squamous cell carcinoma of the head and neck (SCCHN), metastatic pancreatic ductal cancer (PDAC), and acute myeloid leukemia (AML).

On January 27, 2020 PharmaCyte Biotech (OTCQB: PMCB) reported that its clinical trial product, which will be employed during the company’s upcoming Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC), has passed each of the U.S. Food and Drug Administration’s (FDA) required "release tests" for safety and functionality (Press release, PharmaCyte Biotech, JAN 27, 2020, View Source [SID1234553588]). With a clinical trial product that has proven to be safe and functional, PharmaCyte is now "on-the-clock" for its submission of an Investigational New Drug application or "IND" package to the U.S. FDA requesting a Phase 2b clinical trial in LAPC at trial sites throughout the United States.

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In a series of 10 "release tests" over two batches of the company’s clinical trial product for a total of 20 "release tests," PharmaCyte’s signature live-cell encapsulation technology, Cell-in-a-Box, performed admirably and proved to be both safe to place into humans in a clinical trial, and to function properly. During the tests, the more than 20,000 genetically modified live cells that fully fill each Cell-in-a-Box capsule were functioning as expected after being manufactured, placed into syringes, frozen, thawed and then tested for enzymatic activity, cell viability, and biologic activity among a host of other tests.

And now that "release testing" has ended successfully, Austrianova will issue 2 Certificates of Analysis to PharmaCyte—1 for each manufactured and tested batch of the company’s clinical trial product. Additionally, Austrianova will turn over all of the completed batch records to PharmaCyte from both production runs and the subsequent "release testing." The data generated from these tests are necessary information that must be entered into the company’s IND application.

PharmaCyte has methodically taken extra steps to position the company for a greater chance at success once the IND is submitted to the FDA. One such step is the manufacture and testing of two batches of its clinical trial product, which was requested by cGMP Validation, the company taking responsibility for releasing the clinical trial product into the U.S. for use in PharmaCyte’s upcoming clinical trial.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, commenting on manufacturing a second batch of the company’s clinical trial product, said, "Our GMP consultant, cGMP Validation, has informed us that while two successful manufacturing runs are not required by the FDA to request a Phase 2b clinical trial, it could go a long way in demonstrating to the FDA that our manufacturing process is robust and reproducible – manufacturing qualities that are highly embraced by the FDA."

Successfully completing the manufacturing process of PharmaCyte’s clinical trial product, conducting and successfully completing "release testing" on both batches of the company’s product, and the data being generated from those tests are all critical to the completion of PharmaCyte’s IND package. With the announced success of the "release tests," PharmaCyte has essentially conquered what was the greatest hinderance in the company’s ability to move forward with completing its IND application.

Because pancreatic cancer is the third leading cause of cancer-related deaths, this work for PharmaCyte is important and necessary, and the company has painstakingly endured a process of perfection to land it on the precipice of engaging with the FDA in an opportunity to enter into a clinical trial. In 2020 alone, an estimated 57,600 Americans will be diagnosed with pancreatic cancer, and approximately 47,050 Americans are expected to die from the disease this year.

PharmaCyte hopes to better those dismal numbers by using its targeted chemotherapy—Cell-in-a-Box plus low doses of the anticancer drug ifosfamide—in patients with LAPC who no longer see any benefit after being treated for 4-6 months with one of the two first-line therapies offered to this patient population. Its stated goal is to make inoperable tumors operable and give these patients a second chance at life. Needless to say, PharmaCyte is closer than ever to submitting its IND, so the countdown to this momentous milestone for the company’s shareholders should be "officially" underway and is certainly highly anticipated.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

On January 27, 2020 Santhera Pharmaceuticals (SIX: SANN) reported net revenues of CHF 27.9 million in 2019 from sales of Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON), which was out-licensed to Chiesi Group from August 2019 (Press release, Santhera Pharmaceuticals, JAN 27, 2020, View Source [SID1234553587]). The Company made strong progress in advancing its lead neuromuscular compounds Puldysa (idebenone) and vamorolone towards market entry and regulatory submission, respectively.

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"We are pleased about Santhera’s strong progress in 2019 and are excited about the prospects 2020 holds," said Dario Eklund, CEO of Santhera. "Our late-stage neuromuscular assets targeting the high value Duchenne muscular dystrophy (DMD) market are nearing key inflection points. With Puldysa (idebenone), we anticipate offering the first drug for the treatment of respiratory dysfunction for non-ambulant DMD patients who are not taking glucocorticoids. Vamorolone, a first-in-class anti-inflammatory drug candidate shown in studies to improve muscle and motor function with a favorable clinical safety profile, is being developed to replace glucocorticoids as standard of care in ambulant DMD patients. We will continue working with clinical experts, patient advocacy groups and regulators in order to bring these promising treatments to patients with currently few alternative treatment options as soon as possible."

2019 Turnover

Net revenues CHF 27.9 million
Gross income of CHF 49.3 million from Raxone out-licensing agreement
Freely available liquid funds of CHF 31.4 million (December 31, 2019)
2019 full-year net revenues slightly above guidance
In 2019, Santhera reported net revenues of CHF 27.9 million (2018: CHF 31.7 million), slightly surpassing the Company’s full-year guidance. This includes sales of Raxone in the approved indication Leber’s hereditary optic neuropathy (LHON) in the first seven months of 2019. From August 2019, after the closing of the licensing transaction and the transfer of the Raxone-business to Chiesi Group, Santhera is commercializing Raxone for LHON in France in a transitional phase.

Upfront payment from Chiesi Group following closing of licensing agreement
In August 2019, Santhera recognized an initial gross income of CHF 49.3 million (EUR 44 million) from the licensing agreement with Chiesi Group. As previously announced per the agreement, Chiesi Group has in-licensed Raxone for LHON and all other ophthalmologic indications for all territories worldwide except the US and Canada for a total consideration of up to EUR 93 million.

As of December 31, 2019, freely available liquid funds (cash and cash equivalents) amounted to CHF 31.4 million (August 31, 2019: CHF 43.7 million). In addition, the Company held CHF 1.5 million of restricted cash designated for the interest payments related to the convertible bonds issued in 2017.

Pipeline and Regulatory Update

Anticipated near-term inflection points towards approval for both DMD pipeline candidates Puldysa (idebenone) and vamorolone are:

Q2-2020: CHMP opinion on marketing authorization application for Puldysa in DMD in Europe
Q4-2020: Launch of Puldysa in first European markets
Q4-2020: Read-out of topline data of pivotal trial for vamorolone in DMD
Q1-2021: Filing a New Drug Application for vamorolone in DMD in the US
Puldysa first launch in Europe in 2020 subject to positive opinion and approval from CHMP/EMA
The review of Santhera’s application for conditional marketing authorization (CMA) for Puldysa in the treatment of respiratory dysfunction in patients with DMD who are not using glucocorticoids is ongoing and the Company expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) around mid-2020. Subject to a positive opinion and subsequent EU approval, Santhera expects to launch Puldysa in the first European markets in late 2020.

SIDEROS DMD trial enrollment with idebenone nearing completion
Patient enrollment in Santhera’s randomized, double-blind, placebo-controlled Phase 3 SIDEROS trial is expected to complete in Q2-2020 (clinicaltrials.gov: NCT02814019). With a study duration of 18 months, the last patient’s last visit is scheduled for Q4-2021. If positive, the study data will allow for regulatory submissions supporting the use of Puldysa in all DMD patients experiencing respiratory decline, irrespective of their glucocorticoid use, in Europe and the US.

Vamorolone pivotal VISION-DMD study progressing as cornerstone for US/European regulatory filings
ReveraGen BioPharma is currently enrolling the Phase 2b VISION-DMD study with vamorolone, designed as a pivotal efficacy and safety trial (VBP15-004; clinicaltrials.gov: NCT03439670). Read-out of topline 6-month data from the randomized placebo-controlled treatment period is expected by Q4-2020 followed by an NDA submission in the US by Q1-2021. Under the agreements between the parties and upon exercising its option, Santhera would receive the sub-license to ReveraGen’s vamorolone from Idorsia Ltd (SIX: IDIA) for all indications and all countries worldwide except Japan and South Korea. The marketing authorization application in Europe will require inclusion of 12-month data expected for Q2-2021.

Neuromuscular franchise a top priority for 2020
Santhera’s strategic priorities for 2020 are its neuromuscular franchise: Puldysa and vamorolone in DMD. For Puldysa, the focus is on the preparation for European market entry in DMD later in the year and the completion of enrollment into the SIDEROS trial to support planned regulatory submissions, particularly in the US. For vamorolone, the key milestone will be the VISION-DMD topline data readout, which if positive would allow for preparation of the NDA filing and pave the way for Santhera’s option exercise.

In parallel, the Company is advancing its clinical stage candidate POL6014 for cystic fibrosis and is evaluating further diversification of its platform type pipeline products, including development of additional indications in collaboration with partners.

Corporate Calendar

March 24, 2020 – Publication of the Annual Report 2019 (07h00 CET)
April 22, 2020 – Annual Shareholders’ Meeting