VolitionRx Limited Announces Proof of Concept Data in Lymphoma and Leukemia

On December 3, 2019 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported results from its latest proof of concept study (Press release, VolitionRX, DEC 3, 2019, View Source [SID1234551897]). A single pre-clinical Nu.Q assay utilizing a basic microtiter plate platform detected 80% of newly diagnosed Non-Hodgkins Lymphoma ("NHL") and Leukemia cases (mixture of Acute Lymphocytic Leukemia ("ALL") and Acute Myeloid Leukemia ("AML") at 95% specificity among healthy subjects. The Area Under the Curve ("AUC") for this single assay was 91%. A number of other assays in development also demonstrated promising individual assay results with AUCs ranging from 79% to 91%.

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Commenting on these results Dr. Jake Micallef, Chief Scientific Officer at Volition said, "This is a very exciting outcome as we continue the development of our assays beyond colorectal, lung and prostate cancer. We have previously announced results from both single assay and panels of Nu.Q biomarkers but the results of this discovery study are the best single Nu.Q assay result in any cancer we have demonstrated to date. We are delighted to report such promising results for Nu.Q’s ability to detect a range of blood cancers in this small study.

The objective of this proof of concept study was to investigate a range of cancer conditions not previously studied for detection using Nu.Q assays and the results showed remarkable detection for all 3 blood cancers tested. The next steps are to evaluate these findings in independent larger clinical trials of symptomatic populations using Volition’s automated ELISA product platform whilst we also continue pre-analytics studies".

Dr. Jason Terrell, Chief Medical Officer at Volition said, "This is an important finding for us. These types of hematopoietic malignancies are difficult to diagnose. Symptoms are often vague and non-specific such as fatigue and recurrent infections and yet the definitive diagnostic tools of bone marrow or lymph node biopsy are invasive and can be expensive. There are over 700,000 new cases of NHL, ALL and AML diagnosed globally each year and approximately 415,000 deaths. Whilst clearly more studies need to be conducted, we hope that a routine blood test using our Nu.Q technology could lead to earlier diagnosis."

About the Study

Commercially obtained patient blood plasma samples were taken from 62 healthy volunteers (mean age 45 years) as well as approximately 300 patients previously diagnosed with bladder, bone, brain, esophageal, head and neck, melanoma, skin, uterine, or cervical cancers (mean age 56 years) and 54 subjects diagnosed with hematopoietic cancers (mean age 57 years).

Piramal Pharma Solutions Announces Collaboration With BerGenBio on the Development of FDA Fast Track Designated Leukemia Treatment

On December 3, 2019 Piramal Enterprises Limited’s Pharma Solutions business, a leading Contract Development and Manufacturing Organization (CDMO), reported that the company will be partnering with BerGenBio ASA (OSE: BGBIO) on the development of bemcentinib for the treatment of elderly patients with relapsed Acute Myeloid Leukemia (AML) (Press release, Piramal, DEC 3, 2019, View Source [SID1234551896]). Bemcentinib was recently designated as a Fast Track drug by the U.S. Food and Drug Administration (FDA), as there are currently no marketed drugs specifically approved for relapsed AML patients, representing a significant unmet medical need.

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Peter DeYoung, CEO, Piramal Pharma Solutions, stated, "BerGenBio’s decision to partner with us is a validation of our integrated business model. The breadth and scale of our assets and our expertise as a leading CDMO allow us to customize services according to their needs and deliver solutions that benefit the patients."

Richard Godfrey, Chief Executive Officer of BerGenBio, stated that "Outsourcing of development and manufacturing services is a requirement for us, and we were rigorous in our due diligence of potential partners. Piramal Pharma Solutions’ capabilities – especially the Xcelerate Integrated Solutions platform – represent an ideal solution for BerGenBio."

To accelerate the timelines required for Fast Track development, PPS will be developing the drug leveraging its Xcelerate Integrated Solutions platform that delivers speed, flexibility and versatility to the drug development process. The Xcelerate platform, which addresses the industry’s growing need for preferred partner relationships, has been successfully applied to more than eighty programs.

Xcelerate Integrated Solutions takes advantage of the extensive PPS network of drug discovery and development capabilities located across its sites in North America, Europe and India. Customers benefit from streamlined and fully integrated services, all managed by a single point of contact who expedites the exchange of information and accelerates timelines.

The bemcentinib development program encompasses a fully integrated program of PPS resources that includes intermediates sourced from India, pilot process development and API validation in North America and formulation development in Europe. As part of the partnership agreement, PPS is also expected to provide commercial manufacturing of the final drug product.

Titan Pharmaceuticals To Present At The First Annual BioTuesdays Pre-JPM Virtual Conference

On December 3, 2019 Titan Pharmaceuticals, Inc. (NASDAQ: TTNP) reported that management will present an update on the Company’s business at the first annual BioTuesdays Pre-JPM Virtual Conference on Tuesday, December 10, 2019 at 3:00 p.m. EST / 12:00 p.m. PST (Press release, Titan Pharmaceuticals, DEC 3, 2019, View Source [SID1234551895]).

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The presentation will be broadcast live and archived on Titan’s website at www.titanpharm.com/news/events.

CASI Pharmaceuticals Announces China NMPA Approval Of CNCT19 (CD19 CAR-T) Clinical Trial Applications

On December 3, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported that the China National Medical Product Administration (NMPA), has approved the clinical trial applications for CNCT19 (CD19 CAR-T) in relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) and relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL) submitted by its partner Juventas Cell Therapy Ltd., a biopharmaceutical company focused on cell therapy (Juventas) (Press release, CASI Pharmaceuticals, DEC 3, 2019, View Source [SID1234551894]). CASI previously reported its acquisition of exclusive worldwide commercial rights to CNCT19 from Juventas. Juventas has responsibility for the clinical development of CNCT19.

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Larry Zhang, CASI’s President, commented, "This is a very exciting milestone for CASI. CAR-T therapies were first approved in the United States in 2017; there are currently no CAR-T therapies marketed in China. CNCT19 will be manufactured in China at a cost significantly less than the cost of imported therapies, which ultimately enables us to make it more widely available to the Chinese patient population. We expect to start enrolling patients in early 2020."

About CNCT19
CNCT19 targets CD19, a B-cell surface protein widely expressed during all phases of B-cell development and a validated target for B-cell driven hematological malignancies. CD19-targeted CAR constructs from several different institutions have demonstrated consistently high antitumor efficacy in children and adults with relapsed B-cell acute lymphoblastic leukemia (B-ALL), chronic lymphocytic leukemia (CLL), and B-cell non-Hodgkin lymphoma (B-NHL). CD19 antigen is the most frequently used target in the CAR-T cell therapy clinical trials for hematological malignancies such as leukemia and lymphoma. CASI holds the worldwide commercial rights to CNCT19. Juventas is responsible for the development of CNCT19 with CASI’s participation on the program’s steering committee.

About Juventas
Juventas Cell Therapy Ltd. is a China-based domestic biopharmaceutical company established in 2018 focused on R&D, clinical translation and commercialization of innovative immune cell therapy. Juventas’ R&D operations are based in Beijing and its GMP facility is located in Tianjin City. More information on Juventas and its pipeline is available at www.juventas.cn.

FDA Approves Genentech’s Tecentriq Plus Chemotherapy (Abraxane and Carboplatin) for the Initial Treatment of Metastatic Non-Squamous Non-Small Cell Lung Cancer

On December 3, 2019 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the U.S. Food and Drug Administration (FDA) approved Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [paclitaxel protein-bound; nab-paclitaxel] and carboplatin) for the initial (first-line) treatment of adults with metastatic non-squamous non-small cell lung cancer (NSCLC) with no EGFR or ALK genomic tumor aberrations (Press release, Genentech, DEC 3, 2019, View Source [SID1234551893]).

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"We are pleased to offer this Tecentriq-based combination as a new treatment option that can provide a clinically meaningful survival benefit for people with non-squamous non-small cell lung cancer," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "Today’s approval offers another opportunity to help prolong the lives of people with this type of the disease."

This approval is based on results from the Phase III IMpower130 study, which showed Tecentriq in combination with chemotherapy helped people live significantly longer compared to chemotherapy alone (median overall survival [OS] = 18.6 versus 13.9 months; hazard ratio [HR] = 0.80; 95% CI: 0.64–0.99; p=0.0384) in the intention-to-treat wild-type (ITT-WT) population. The Tecentriq-based combination also significantly reduced the risk of disease worsening or death (progression-free survival; PFS) compared with chemotherapy alone (median PFS=7.2 versus 6.5 months; HR=0.75; 95% CI: 0.63–0.91; p=0.0024) in the ITT-WT population.

Safety for the Tecentriq plus chemotherapy combination appeared consistent with the known safety profiles of the individual medicines, and no new safety signals were identified with the combination. Grade 3-4 treatment-related adverse events were reported in 73.2% of people receiving Tecentriq plus chemotherapy compared with 60.3% of people receiving chemotherapy alone.

In lung cancer, Tecentriq is also approved in combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of adults with metastatic non-squamous NSCLC with no EGFR or ALK genomic tumor aberrations. Additionally, Tecentriq is approved by the FDA to treat adults with metastatic NSCLC who have disease progression during or following platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for NSCLC harboring these aberrations prior to receiving Tecentriq. Tecentriq is also approved in combination with carboplatin and etoposide (chemotherapy) for the initial (first-line) treatment of adults with extensive-stage small cell lung cancer (ES-SCLC).

Genentech has an extensive development program for Tecentriq, including nine Phase III studies underway across different types of lung cancer, and multiple ongoing and planned Phase III studies across genitourinary, skin, breast, gastrointestinal, gynecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

For those who qualify, Genentech offers patient assistance programs for people taking Tecentriq through Genentech Access Solutions. Doctors can contact Genentech Access Solutions at (866) 422-2377. More information is also available at View Source

About the IMpower130 study

IMpower130 is a Phase III, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq in combination nab-paclitaxel and carboplatin versus chemotherapy (nab-paclitaxel and carboplatin) alone for chemotherapy-naïve patients with stage IV non-squamous NSCLC. The study enrolled 724 people, of whom 681 were in the ITT-WT population and were randomized (2:1) to receive:

Tecentriq plus nab-paclitaxel and carboplatin (Arm A), or
Nab-paclitaxel and carboplatin (Arm B, control arm)
During the treatment-induction phase, people in Arm A received Tecentriq and carboplatin on day 1 of each 21-day cycle, and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until loss of clinical benefit, whichever occurred first. People in Arm A received Tecentriq during the maintenance treatment phase until loss of clinical benefit was observed.

During the treatment-induction phase, people in Arm B received carboplatin on day 1 and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until disease progression, whichever occurred first. People in Arm B received best supportive care during the maintenance treatment phase. Switch maintenance to pemetrexed was also permitted. People who were consented prior to a protocol revision were given the option to crossover to receive Tecentriq as monotherapy until further disease progression.

The co-primary endpoints were:

PFS as determined by the investigator using RECIST v1.1 in people without EGFR or ALK mutations (the ITT-WT population)
OS in the ITT-WT population
About lung cancer

According to the American Cancer Society, it is estimated that more than 228,000 Americans will be diagnosed with lung cancer in 2019, and NSCLC accounts for 80-85% of all lung cancers. It is estimated that approximately 60% of lung cancer diagnoses in the United States are made when the disease is in the advanced stages.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Abraxane is a registered trademark of Abraxis Bioscience, LLC, a wholly owned subsidiary of Celgene Corporation.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used with chemotherapy and other anti-cancer medicines as your first treatment when your lung cancer:
has spread or grown, and
is a type called "non-squamous NSCLC", and
your tumor does not have an abnormal "EGFR" or "ALK" gene.
Tecentriq may be used alone when your lung cancer:
has spread or grown, and
you have tried chemotherapy that contains platinum, and it did not work or is no longer working.
If your tumor has an abnormal "EGFR" or "ALK" gene, you should have also tried an FDA-approved therapy for tumors with these abnormal genes, and it did not work or is no longer working.
A type of lung cancer called small cell lung cancer (SCLC).

Tecentriq may be used with the chemotherapy medicines carboplatin and etoposide as your first treatment when your lung cancer:
is a type of lung cancer called "extensive-stage small cell lung cancer," which means that it has spread or grown
It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Lung problems (pneumonitis)–signs and symptoms of pneumonitis may include new or worsening cough, shortness of breath, and chest pain
Liver problems (hepatitis)–signs and symptoms of hepatitis may include yellowing of the skin or the whites of the eyes, severe nausea or vomiting, pain on the right side of the stomach area (abdomen), drowsiness, dark urine (tea colored), bleeding or bruising more easily than normal, and feeling less hungry than usual
Intestinal problems (colitis)–signs and symptoms of colitis may include diarrhea (loose stools) or more bowel movements than usual, blood or mucus in stools or dark, tarry, sticky stools, and severe stomach area (abdomen) pain or tenderness
Hormone gland problems (especially the thyroid, adrenal glands, pancreas, and pituitary)–signs and symptoms that the hormone glands are not working properly may include headaches that will not go away or unusual headaches, extreme tiredness, weight gain or weight loss, dizziness or fainting, feeling more hungry or thirsty than usual, hair loss, changes in mood or behavior (such as decreased sex drive, irritability, or forgetfulness), feeling cold, constipation, the voice gets deeper, urinating more often than usual, nausea or vomiting, and stomach area (abdomen) pain
Problems in other organs–signs and symptoms may include severe muscle weakness, numbness or tingling in hands or feet, confusion, blurry vision, double vision, or other vision problems, changes in mood or behavior, extreme sensitivity to light, neck stiffness, eye pain or redness, skin blisters or peeling, chest pain, irregular heartbeat, shortness of breath, or swelling of the ankles
Severe infections–signs and symptoms of infection may include fever, cough, flu-like symptoms, pain when urinating, and frequent urination or back pain
Severe infusion reactions–signs and symptoms of infusion reactions may include chills or shaking, itching or rash, flushing, shortness of breath or wheezing, swelling of the face or lips, dizziness, fever, feeling like passing out, and back or neck pain
Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

have immune system problems (such as Crohn’s disease, ulcerative colitis, or lupus); have had an organ transplant; have lung or breathing problems; have liver problems; have a condition that affects the nervous system (such as myasthenia gravis or Guillain-Barre syndrome); or are being treated for an infection
are pregnant or plan to become pregnant. Tecentriq can harm an unborn baby. Patients should tell their healthcare provider right away if they become pregnant or think they may be pregnant during treatment with Tecentriq. Females who are able to become pregnant:
A healthcare provider should do a pregnancy test before they start treatment with Tecentriq
They should use an effective method of birth control during their treatment and for at least 5 months after the last dose of Tecentriq
are breastfeeding or plan to breastfeed. It is not known if Tecentriq passes into the breast milk. Patients should not breastfeed during treatment and for at least 5 months after the last dose of Tecentriq
Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

feeling tired or weak
nausea
cough
shortness of breath
decreased appetite
The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

feeling tired or weak
nausea
hair loss
constipation
diarrhea
decreased appetite
Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at 1-800-FDA-1088 or View Source

Report side effects to Genentech at 1-888-835-2555.

Please visit View Source for the Tecentriq full Prescribing Information for additional Important Safety Information.

About Genentech in personalized cancer immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, we’re investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is studying more than 10 cancer immunotherapy medicines across 70 clinical trials alone or in combination with other medicines. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit View Source

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.