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PharmaMar Group reports year to date results up to September 30th, 2019

On October 23, 2019 PharmaMar Group (MSE:PHM) reported total revenues of €62.5 million for 2019 up to September 30th, compared to €88.7 million in the same period last year (Press release, PharmaMar, OCT 23, 2019, View Source [SID1234542450]). The variation in revenues between the first nine months of 2019 and 2018 is due to the difference in revenues from license agreements. In 2018, more than €24 million in revenues from different license agreements were recorded. Some of these agreements may lead to new future revenues, but they have not yet reached the milestones that will lead to them.

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Yondelis’ cumulative sales up to September 30th were €54 million, compared to €57 million in the same period last year.

In relation to the Group’s operating expenses, during the first nine months of the year these decreased in practically all items. In R&D, total expenditure up to September 30th was €41 million, which represents a 27% reduction in expenditure compared to the same period last year. This cost reduction is mainly due to the oncology area. Last year, in addition to the ATLANTIS Phase III trial with lurbinectedin for the treatment of Small Cell Lung Cancer (SCLC), there were other open and active clinical trials that have not been active during the first nine months of 2019, although they remain open until they can be definitively concluded.

In relation to the clinical trials with lurbinectedin, it is important to point out that on August 19th it was announced that the Company will file lurbinectedin’s NDA for the treatment of relapsed Small Cell Lung Cancer (SCLC) in the United States under the "accelerated approval" program during the last quarter of this year. This would open up the possibility that the FDA could approve lurbinectedin in the U.S. for the treatment of SCLC within the next year.

Up to September 30th 2019, the Group has recorded a net attributable result of €- 26.9 million.

Finally, at the end of the first nine months of this year, PharmaMar Group recorded total cash and cash equivalents of €27 million and reduced its net debt to €57.8 million, compared to €65.6 million at the beginning of the year.

NEW RESEARCH STUDY PROVIDES TARGET VALIDATION FOR MATEON’S FIRST-IN-CLASS RNA THERAPEUTIC OT101 FOR THE TREATMENT OF DIFFICULT TO TREAT FORMS OF CHILDHOOD BRAIN TUMOR

On October 23, 2019 Oncotelic Inc. ("Oncotelic"), a wholly owned subsidiary of Mateon Therapeutics Inc. (OTCQB:MATN) dedicated to the development of innovative treatments for cancer, reported the publication of a peer-reviewed research article authored by Fatih Uckun MD PhD and his colleagues at Oncotelic in the Journal of Clinical Research in Pediatrics (View Source) (Press release, Oncotelic, OCT 23, 2019, View Source [SID1234542449]). The article titled "In silico Molecular Target Validation Demonstrates Transforming Growth Factor Beta 2 is Strongly Expressed in Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme" demonstrates the therapeutic potential of Mateon’s first-in-class RNA therapeutic OT101 for the treatment of diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma multiforme (GBM).

Citation Reference: Uckun FM, Trieu V, Hwang L, Qazi S. In silico Molecular Target Validation Demonstrates Transforming Growth Factor Beta 2 is Strongly Expressed in Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme. Clin Res Pediatr 2019;2(1):1-10.

"Our research has revealed that the TGF beta2 gene product, which is the molecular target for OT101/Trabedersen, may serve as a target for immunotherapy in pediatric high-grade gliomas, especially DIPG. These in silico target validation data extend the promising clinical data on the therapeutic activity of OT101 in adults and young adults and further demonstrate the potential of OT101 as a promising drug candidate in the treatment of pediatric DIPG, an orphan disease with a low survival rate and no established or effective standard of care," explained Fatih Uckun, MD, PhD, the Chief Medical Officer of Mateon. "The durable objective responses achieved in adult patients with recurrent/refractory high-grade gliomas after treatment with our lead anti-TGF beta2 compound OT-101 contribute to our optimism that new treatment strategies leveraging OT101 may favorably change the therapeutic landscape for difficult-to-treat brain tumors with a very poor prognosis, including pediatric GBM and DIPG," Dr. Uckun explained. Last month, US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for OT101/Trabedersen for the treatment of diffuse intrinsic pontine glioma (DIPG) as a drug for a "rare pediatric disease," as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act.

OT101, a first-in-class RNA therapeutic designed to abrogate the immunosuppressive actions of TGF-beta 2, is Oncotelic’s lead anti-brain tumor drug candidate. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM. DIPG, an orphan disease with a low survival rate and no established or effective standard of care. Despite numerous clinical trials of chemotherapeutic agents, immuno-oncology drugs and specific targeted therapies, no significant progress has been made in the treatment of DIPG and the prognosis remains dismal, with a mean OS of 9–12 months from the time of diagnosis, a median survival time of approximately 10 months, and a two-year OS rate of less than 10 percent. Five-year survival is less than 3 percent, and many long-term survivors have evidence of moderate or severe cognitive impairment, likely as a consequence of radiation therapy. Chemotherapy does not have an established role in the management of patients with DIPG. Furthermore, there is no standard treatment for progressive DIPG after the failure of radiation therapy and no salvage regimen has been shown to extend survival. Therefore, there is an urgent need for therapeutic innovations for treatment of DIPG, as reflected by multiple treatment modalities being evaluated in early neuro-oncology clinical trials. Further development of OT-101 may offer renewed hope for salvage therapy of pediatric DIPG patients who have this rare and fatal disease.

"As we move Trabedersen through its clinical development for Pediatric Diffuse Intrinsic Pontine Glioma and Glioblastoma Multiforme, we are moving CA4P forward along the same path as potential treatment for Pediatric Unresectable or Metastatic Melanoma- a rare and difficult to treat pediatric skin cancer, " said Dr. Vuong Trieu, Chief Executive Officer of Mateon "From the corporate side we are moving forward with activities to uplist Mateon. Looking forward to updating our shareholders on the coming quarter."

FibroGen Announces First Patient Dosed in LAPIS, a Phase 3 Clinical Trial of Pamrevlumab for the Treatment of Patients With Locally Advanced Pancreatic Cancer

On October 23, 2019 FibroGen, Inc. (NASDAQ: FGEN), reported the dosing of the first patient in the LAPIS Phase 3 clinical study of pamrevlumab in patients with unresectable locally advanced pancreatic cancer (LAPC) (Press release, FibroGen, OCT 23, 2019, View Source [SID1234542448]).

"Patients with unresectable locally advanced pancreatic cancer face a dire prognosis. The use of pamrevlumab, a novel anti-fibrotic used as neoadjuvant therapy, may change tumor status from unresectable to resectable thus potentially improving the prognosis of the patient", said Pascal Hammel, MD, PhD a gastroenterologist, oncologist, Hôpital Beaujon, Clichy France. "If this pivotal phase 3 trial is successful, I believe this type of treatment will be highly valued by the physician community treating LAPC."

"With this announcement of dosing of the first patient in our LAPIS study, we are excited to further advance late-stage development of pamrevlumab as a treatment for locally-advanced pancreatic cancer," said Elias Kouchakji, M.D., Senior Vice President, Clinical Development, and Drug Safety and Pharmacovigilance, with FibroGen. "We are grateful for the opportunity to build on supportive data from our previous Phase 2 studies, as we continue to explore the potential of pamrevlumab as an important treatment option for patients with LAPC."

In LAPC patients who undergo resection, median survival and five-year survival rates are higher than for unresectable LAPC patients who did not undergo resection. Therefore, achieving surgical resection in this patient population is a meaningful treatment goal.

LAPIS is a multinational randomized, double-blind, placebo-controlled Phase 3 study that will evaluate neoadjuvant pamrevlumab therapy in combination with gemcitabine and nab-paclitaxel. The design of this study is similar to FibroGen’s prior Phase 2 trials. In these studies, a higher percentage of patients treated with the pamrevlumab combination achieved surgical resection and had a statistically significant median survival rate. LAPIS is expected to enroll approximately 260 patients. The primary endpoint of the study is overall survival. The resection rate is a surrogate endpoint, and, should the resection rates favor the pamrevlumab combination, FibroGen plans to request a meeting with the FDA to discuss a marketing application under the provisions of accelerated approval.

For more information regarding this study please visit www.clinicaltrials.gov (NCT03941093).

About Locally Advanced Pancreatic Cancer
In locally advanced pancreatic cancer (LAPC), the patient’s tumor typically involves structures, particularly blood vessels that are closely associated with the pancreas such as the superior mesenteric artery and superior mesenteric vein. Involvement of the cancer around these blood vessels precludes surgical removal of the tumor. Patients with unresectable LAPC have a median survival of 6 to 10 months, only slightly better than patients with metastatic pancreatic cancer, and only 20 percent of newly diagnosed patients are classified as having resectable disease. Patients who have their tumor surgically removed have a much better prognosis with median survival of approximately 23 months with some patients being cured.

About Pamrevlumab
Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and for the treatment of locally advanced unresectable pancreatic cancer (LAPC), and in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (DMD). The U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to pamrevlumab for the treatment of patients with IPF, LAPC, and DMD. Pamrevlumab has also received Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with IPF and LAPC. Across all clinical studies, pamrevlumab has consistently demonstrated a good safety and tolerability profile to date. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

Verastem Oncology Announces COPIKTRA™ (Duvelisib) Presentations at the Lymphoma & Myeloma 2019 International Congress

On October 23, 2019 Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported that five posters highlighting clinical data for COPIKTRA (duvelisib) will be presented at the Lymphoma & Myeloma 2019 International Congress taking place October 23-26, 2019, in New York City (Press release, Verastem, OCT 23, 2019, View Source [SID1234542447]). The presented abstracts focus on clinical data from the Phase 3 DUO study, including evaluation of COPIKTRA efficacy and safety in high-risk patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), dose modification data and results from a post-hoc analysis evaluating the effect of COPIKTRA on lymphocytosis in patients. Other presented data include the characterization of duvelisib in patients with refractory Marginal Zone Lymphoma from the Phase 2 DYNAMO study, and an update on the safety profile and management of adverse events in heavily pre-treated patients with advanced hematological malignancies.

"Findings from the DUO study demonstrated that patients taking duvelisib who have received two or more prior therapies experienced improved clinical outcomes and a manageable safety profile," states Matthew S. Davids, MD, Associate Director, Center for Chronic Lymphocytic Leukemia at Dana-Farber Cancer Institute. "These results with duvelisib are important for this patient population, which is in need of targeted therapies to control their disease."

"The data presented at this year’s Lymphoma & Myeloma Congress reflect the utility of duvelisib in patients with relapsed or refractory CLL/SLL after at least two prior therapies, including in patients with advanced disease or at high-risk of recurrence," commented Hagop Youssoufian, MSc, M.D., Head of Medical Strategy at Verastem Oncology. "Further, the research supports the approach to management of adverse events through dose interruptions or dose reductions without an impact on the patient’s response, which could allow patients who are benefitting to stay on therapy longer."

COPIKTRA, a targeted oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, received approval as monotherapy from the U.S. Food and Drug Administration (FDA) in September 2018 for the treatment of patients with relapsed or refractory CLL/SLL after at least two prior therapies. COPIKTRA also received accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Continued approval in FL may be contingent upon verification and description of clinical benefit in confirmatory trials.

Details for the poster presentations are as follows:

Title: An improved benefit-risk profile of duvelisib in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who received ≥2 prior therapies
Lead author: Matt Davids, M.D., Dana-Farber Cancer Institute, Boston, MA
Presentation ID: P-012

Title: Effect of dose modifications on response to duvelisib in patients with relapsed or refractory CLL/SLL in the DUO trial
Lead author: Nicole Lamanna, Columbia University Medical Center
Presentation ID: P-030

Title: Patterns of duvelisib-induced lymphocytosis in patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma, including those with high-risk factors treated in the DUO trial
Lead author: Jacqueline Barrientos, Zucker School of Medicine at Hofstra/Northwell
Presentation ID: P-015

Title: Characterization of duvelisib in patients with refractory marginal zone lymphoma: data from the phase 2 DYNAMO trial
Lead author: Eric Jacobsen, Dana-Farber Cancer Institute, Boston, MA
Presentation ID: P-029

Title: Safety Profile and Management of Adverse Events Associated with Duvelisib in Patients with Advanced Hematologic Malignancies
Lead author: Karen Francoeur, Verastem Oncology
Presentation ID: P-031

PDF copies of these poster presentations will be available here after the meeting.

COPIKTRA includes a Boxed Warning for fatal and serious toxicities including infections, diarrhea or colitis, cutaneous reactions and pneumonitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

SELECT IMPORTANT SAFETY INFORMATION

This does not include all information needed to use COPIKTRA (duvelisib) safely and effectively. See full Prescribing Information.

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS
See full Prescribing Information for complete boxed warning
Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% (<1% fatal) of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
INDICATIONS AND USAGE

COPIKTRA is a kinase inhibitor indicated for the treatment of adult patients with:

Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies.
Relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Accelerated approval based on overall response rate and continued approval may be contingent upon confirmatory trials
WARNINGS AND PRECAUTIONS

Hepatotoxicity: Monitor hepatic function.
Neutropenia: Monitor blood counts.
Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.
ADVERSE REACTIONS

The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at 1-877-7RXVSTM (1-877-779-8786).

DRUG INTERACTIONS

CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors.
CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates.
USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed.

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.1,2,3 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.4 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

BioInvent Signs Manufacturing Agreement With Cancer Research UK Expected to Generate Approximately SEK 30 Million (~$3 Million)

On October 23, 2019 BioInvent International AB (publ) (OMXS: BINV) reported it has concluded a production agreement with Cancer Research UK, the world’s largest independent funder of cancer research, for the production of Hummingbird Bioscience’s anti-HER3 antibody drug HMBD-001 for use within a clinical partnership formed between Cancer Research UK and Hummingbird Bioscience to test the agent in a phase I trial (Press release, BioInvent, OCT 23, 2019, View Source [SID1234542445]).

Under the agreement, which is expected to generate revenue of approximately SEK 30 million (~$3 million), mostly in 2020, BioInvent will provide HMBD-001 to Cancer Research UK. It includes process, analytical and formulation development, process scale up and manufacturing of toxicology and clinical grade material in 200- and 1,000-liters scale.

"We are very pleased to have concluded an agreement to provide this service to Cancer Research UK and are looking forward to producing Hummingbird’s drug candidate for clinical trials. The fact that BioInvent has its own production facility gives us added flexibility, meaning we can develop our proprietary immuno-oncology programs more efficiently, and also generate revenue from our services to help fund our pipeline," says Martin Welschof, CEO of BioInvent.

Dr Nigel Blackburn, Cancer Research UK’s director of drug development, says: "We are delighted to be partnering with BioInvent who will produce Hummingbird Bioscience’s anti-HER3 antibody, ready to be tested in clinical trials. Our clinical development partnerships fast track promising new treatments like this through one of the most difficult stages of drug development, benefitting people with cancer much sooner."

Cancer Research UK’s Centre for Drug Development entered into a partnership with Hummingbird Bioscience to manufacture clinical grade anti-HER3 antibody and conduct a Phase I clinical trial to evaluate drug safety, toxicity and efficacy. HMBD-001 is the only anti-HER3 antibody in development that binds to the most critical region involved in HER3 activation, turning off this signal completely. If successful, this new antibody could be used in the treatment of multiple cancers and be effective against drug resistant cancers.

BioInvent’s manufacturing capabilities
The BioInvent manufacturing facility is compliant with Current Good Manufacturing Practice (cGMP) regulations and is fully based on disposable technology and can produce batches in sizes from 40 L to 1,000 L. The platform process ensures rapid and efficient process development and spans everything from cell line development to final release of drug substance for clinical trials. BioInvent offers a range of cell line development options that include a royalty free GS knocked CHO K1 cell line.