On August 6, 2020 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the Phase III IMpassion131 study, evaluating Tecentriq (atezolizumab) in combination with paclitaxel (chemotherapy), in comparison to placebo plus paclitaxel, did not meet statistical significance on its primary endpoint of progression-free survival (PFS) for the initial (first-line) treatment of people with metastatic triple-negative breast cancer (TNBC), in the PD-L1-positive population (Press release, Hoffmann-La Roche, AUG 6, 2020, View Source [SID1234563238]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The data for the secondary endpoint of overall survival (OS) showed a negative trend, however, the study was not powered for the secondary endpoint of OS and data were immature at time of analysis. OS follow-up is planned to continue until final analysis. Safety for the Tecentriq combination appeared to be consistent with the known safety profile of the individual medicines, and no new safety signals were identified.

In the previous IMpassion130 study, Tecentriq (atezolizumab) in combination with Abraxane (paclitaxel protein-bound particles for injectable suspension (albumin-bound); nab-paclitaxel) demonstrated a statistically significant PFS benefit and, while not formally tested, showed clinically meaningful improvements in OS for people with metastatic TNBC whose tumours express PD-L1 (≥1%). Tecentriq in combination with nab-paclitaxel is approved in over 70 countries for the treatment of adults with unresectable locally advanced or metastatic TNBC whose tumours express PD-L1 (≥1%).

"While we are disappointed by the results from the IMpassion131 study, we are grateful for all the patients, families and physicians who were involved in the study," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. "Today’s results underscore the need to better understand the cancer and immune system interactions, including the chemotherapy backbone and associated regimens. We remain committed to finding optimal treatments for all people living with this aggressive disease."

Full results from IMpassion131 are being discussed with global health authorities and will be presented at a future medical meeting. The insights will also be used to inform existing and future studies in triple-negative breast cancer with Tecentriq in combination with paclitaxel.

Roche has an extensive development programme for Tecentriq, including multiple ongoing and planned Phase III studies across several types of lung, genitourinary, skin, breast, gastrointestinal, gynaecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpassion131 study
The IMpassion131 study is a Phase III, multicentre, randomised, double-blind study evaluating the efficacy and safety of Tecentriq in combination with paclitaxel, in comparison to placebo plus paclitaxel, in people with previously untreated, inoperable, locally advanced or metastatic TNBC. The study enrolled 651 people who were randomised in a 2:1 ratio to receive Tecentriq or placebo plus paclitaxel. The primary endpoint is PFS per investigator assessment (RECIST 1.1) in the PD-L1-positive population followed by intention-to-treat (ITT) populations. Secondary endpoints include OS, objective response rate and duration of response in the PD-L1-positive and ITT populations.

About triple-negative breast cancer
Breast cancer is the most common cancer among women with more than 2 million diagnosed worldwide each year.1 TNBC represents ~15% of all breast cancers and is more common in women under the age of 50, compared with other forms of breast cancer.2-4 It is defined by the lack of expression and/or amplification of the targetable receptors for oestrogen, progesterone and HER2 amplification.5 Patients with metastatic TNBC generally experience rapid progression and shorter OS compared to other subtypes of breast cancer.3

About Roche in breast cancer
Roche has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have contributed to bringing breakthrough innovations in HER2-positive and triple-negative breast cancers. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for all forms of early and advanced breast cancer, including triple-negative and hormone receptor-positive.

Our targeted medicines Herceptin, Perjeta, Kadcyla and Tecentriq are continuing to transform the treatment of early and advanced HER2-positive and triple-negative breast cancers and, through our Tecentriq and ipatasertib clinical programmes, we hope to bring new treatment combinations to people with breast cancer, ultimately improving outcomes.

About Tecentriq
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1, which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T-cells. Tecentriq is a cancer immunotherapy that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. The development of Tecentriq and its clinical programme is based on our greater understanding of how the immune system interacts with tumours and how harnessing a person’s immune system combats cancer more effectively.

Tecentriq is approved in the US, EU and countries around the world, either alone or in combination with targeted therapies and/or chemotherapies in various forms of non-small cell and small cell lung cancer, certain types of metastatic urothelial cancer and in PD-L1-positive metastatic triple-negative breast cancer. In the US, Tecentriq in combination with Avastin is approved for people with unresectable or metastatic hepatocellular carcinoma.

About Roche in cancer immunotherapy
Roche’s rigorous pursuit of groundbreaking science has contributed to major therapeutic and diagnostic advances in oncology over the last 50 years, and today, realising the full potential of cancer immunotherapy is a major area of focus. With over 20 molecules in development, Roche is investigating the potential benefits of immunotherapy alone, and in combination with chemotherapy, targeted therapies or other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system to attack their cancer. Our scientific expertise, coupled with innovative pipeline and extensive partnerships, gives us the confidence to continue pursuing the vision of finding a cure for cancer by ensuring the right treatment for the right patient at the right time.

In addition to Roche’s approved PD-L1 checkpoint inhibitor, Tecentriq (atezolizumab), Roche’s broad cancer immunotherapy pipeline includes other checkpoint inhibitors, such as tiragolumab, a novel cancer immunotherapy designed to bind to TIGIT, individualised neoantigen therapies and T-cell bispecific antibodies. To learn more about Roche’s scientific-led approach to cancer immunotherapy, please follow this link:
View Source

On August 6, 2020 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that Iovance Biotherapeutics (NASDAQ: IOVA) has selected Avid to provide process development, pilot-batch manufacturing and CGMP manufacturing services to support development of IOV-3001, a novel antibody cytokine engrafted protein (Press release, Avid Bioservices, AUG 6, 2020, View Source [SID1234563237]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cell line development activities for IOV-3001 are currently being conducted by Aragen Bioscience under a subcontracting agreement with Avid. In parallel with cell line development, Avid will commence analytical activities, upstream and downstream process development, and pilot-scale non-GMP manufacturing for IOV-3001. Following completion of these activities, Avid will advance to CGMP manufacturing of IOV-3001 within Avid’s state-of-the-art Myford facility in Tustin, CA.

"We are pleased to be selected by Iovance to provide CDMO services to support the company’s continued advancement in developing novel anti-cancer drug candidates. As an Interleukin-2/antibody engrafted protein, IOV-3001 is the type of complex therapeutic candidate for which Avid possesses unique process development and manufacturing expertise, illustrated by our 27 years of producing related compounds," said Timothy Compton, chief commercial officer of Avid. "This new contract award also illustrates the value of Avid’s efforts to establish alliances with key providers of complementary CDMO services such as Aragen. We are pleased to have them contribute their cell line development expertise to this project."

On August 6, 2020 Lepu Biotech of Shanghai reported that it closed a $186 million Series B round to support its portfolio of affordable cancer immunotherapies (Press release, Lepu Biotech, AUG 6, 2020, View Source [SID1234563236]). Lepu Bio, a subsidiary of China medical device company Lepu Medical, was founded in 2018. It has built a pipeline of 12 candidates including seven that have started clinical trials. Lepu’s clinical-stage products include PD-1, PD-L1, oncolytic virus and multiple antibody drug conjugate candidates. The B Round was led by the investment arms of Ping An Insurance and Sunshine Insurance.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On August 6, 2020 T-knife GmbH, a next-generation adoptive T-cell company using its proprietary humanized T-cell receptor (HuTCR) mouse platform to treat solid tumors, reported the closing of a €66 million Series A round of financing (Press release, T-Knife, AUG 6, 2020, View Source [SID1234563234]). The round was led by Versant Ventures and RA Capital Management, with significant participation from existing investors Andera Partners and Boehringer Ingelheim Venture Fund (BIVF).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company was spun out of Max-Delbruck Center for Molecular Medicine with support of Charité University Hospital in Berlin in 2018, where its proprietary HuTCR transgenic mouse platform carrying the entire human TCRαβ gene loci was established by the pioneering work of Prof. Thomas Blankenstein, T-knife’s co-founder. Due to its natural in vivo selection of high-affinity TCRs, T-knife’s TCR-T-cell platform has the potential to be a marked improvement over existing TCR technologies in treating solid tumors.

"Having worked in stealth mode to create a powerful humanized mouse platform bearing the human TCR loci, it is especially gratifying to now receive the validation from esteemed healthcare dedicated funds like Versant Ventures and RA Capital," commented Elisa Kieback, Chief Executive Officer and scientific co-founder of T-knife. "We are equally grateful for the continued support of our founding shareholders, Andera Partners and Boehringer Ingelheim Venture Fund, two top-tier healthcare investors who have been our true partners since inception. Going forward, our goal is to become a transatlantic company by establishing a U.S. presence and expanding our management team accordingly."

T-knife’s proprietary HuTCR mouse expresses only human TCRs that are restricted to human HLA. Due to their natural generation in mice without negative thymic selection, these TCRs are of high specificity and high affinity. The Company has generated a pipeline of patented, unique TCR candidates for clinical development. Proceeds from the Series A round will be allocated to advancing at least four programs into the clinic, ramping-up preclinical work for additional selected proprietary pipeline candidates and discovering TCRs against novel targets.

Moving forward, T-knife’s Board of Directors will be comprised of Josh Resnick (RA Capital), Alex Mayweg (Versant Ventures), Olivier Litzka (Andera Partners), Frank Kalkbrenner (BIVF), Thomas Blankenstein and Elisa Kieback. The Company was advised by Blueprint Life Science Group on the fundraising and by CMS on all legal aspects of the transaction. The new investors were advised by Goodwin Procter. The transaction will close upon governmental and anti-trust clearance.

Alex Mayweg of Versant Ventures commented, "While CAR-T-based therapies have already demonstrated their power in the treatment of hematological cancers, their foray into solid tumors has proven to be less successful. T-knife has developed an exciting technology as its TCR-T cell therapy targets tumor antigens in an MHC-restricted manner, allowing it to be one of the few platforms that is able to target solid tumors. We are consequently thrilled to co-lead this round with RA Capital, a preeminent healthcare dedicated fund, as their investment mandate mirrors our own mission to identify and support game-changing therapies with curative intent."

"We are delighted that T-knife is now an RA Capital portfolio company and are especially pleased to partner with Versant Ventures on leading this financing round," commented Josh Resnick of RA Capital Management. "With the Company’s financial and strategic support now in place, we look forward to working alongside management and fellow investors bring T-knife’s potentially transformative T-cell therapies to solid tumor patients."

Olivier Litzka of Andera Partners added, "Together with our seed round co-investor BIVF and their representative Detlev Mennerich, who also served as the Company’s Chairman over the past two years, we are extremely proud of T-knife’s progress, culminating in this transformational, top quality Series A round. We commend Elisa, Thomas and the team for their accomplishments, and welcome our new partners who share the vision of making T-knife the premier leader in the cell therapy field."

On August 6, 2020 Ardelyx, Inc. (Nasdaq: ARDX), a specialized biopharmaceutical company focused on developing innovative first-in-class medicines to improve treatment for people with kidney and cardiovascular diseases, reported business highlights and financial results for the second quarter ended June 30, 2020 (Press release, Ardelyx, AUG 6, 2020, View Source [SID1234563225]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Over the last quarter, we continued to make critical progress towards our goal of providing our first-in-class therapy tenapanor to adult CKD patients on dialysis with elevated serum phosphorus, a condition, despite traditional therapies, that has been associated with poor survival outcomes," said Mike Raab, president and chief executive officer of Ardelyx. "This past June, we submitted a New Drug Application to the FDA for this indication, and we expect to receive notification of its acceptance for substantive review and our PDUFA date by early September. As part of our filing, we included additional, robust data reconfirming tenapanor’s ability to lower and control serum phosphorous levels at a rate better than those reported with phosphate binders alone. In addition, during the quarter, we augmented our senior leadership team with the hiring of an experienced chief commercial officer and chief financial officer as we prepare for launch and evolving into a revenue-generating company."

Recent Business and Pipeline Updates

Submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the review of tenapanor as a first-in-class therapy to control serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. The filing is supported by three successful Phase 3 studies demonstrating tenapanor’s ability to reduce phosphate levels, with two trials evaluating tenapanor as a monotherapy and the third evaluating tenapanor as part of a dual mechanism approach with phosphate binders.
Released additional positive data from the ongoing NORMALIZE Phase 4 study, which showed that foundational use of tenapanor as monotherapy or with sevelamer enabled up to 47.4% of CKD patients on dialysis to achieve normal serum phosphorus levels (<4.6 mg/dL), a 58% improvement over current standard of care.
Announced that Ardelyx’s collaboration partner in Japan, Kyowa Kirin Co., Ltd. (KKC), presented data at the European Renal Association-European Dialysis and Transplant Association annual meeting (ERA-EDTA 2020) from a Phase 2 study designed to evaluate if, with tenapanor, patients with hyperphosphatemia undergoing hemodialysis could achieve at least a 30% decrease in mean pill burden while maintaining their serum phosphorus level. The results demonstrated that tenapanor enabled a significant reduction in overall pill burden (mean reduction in phosphate binder pill usage by 80%), while maintaining serum phosphorus control.
Strengthened leadership team with the appointment of two senior executives:
Justin Renz, a veteran biopharma executive with over 20 years of experience, as chief financial officer; and
Susan Rodriguez, a highly experienced global biopharma marketing and sales executive with a proven track record of building commercial organizations and leading successful new product launches, as chief commercial officer
Expected 2020 Milestones

Receive notification from the FDA regarding the acceptance for substantive review of the NDA submission and PDUFA date in September 2020
Initiate the OPTIMIZE clinical trial, a study designed to inform physicians on the integration of tenapanor as a foundational therapy into clinical practice, this year
Present AMPLIFY and PHREEDOM Phase 3 clinical trial results at upcoming medical congresses
Report completed NORMALIZE Phase 4 clinical trial results at an upcoming medical conference
Second Quarter 2020 Financial Results

Cash Position: As of June 30, 2020, Ardelyx had total cash, cash equivalents and short-term investments of $204.8 million, as compared to total cash, cash equivalents and short-term investments of $247.5 million as of December 31, 2019.
Revenue: The company generated $1.8 million in revenue, which primarily represents collaborative development revenue, for the three months ended June 30, 2020.
R&D Expenses: Research and development expenses were $18.9 million for the three months ended June 30, 2020, a decrease of $0.6 million, or approximately 3 percent, compared to $19.5 million for the three months ended June 30, 2019. The decrease was due primarily to a decrease in external R&D expenses, with a $1.1 million decrease in tenapanor-related expenses, as well as a $0.6 million decrease in RDX013 program-related expenses, partially offset by $0.7 million of higher expenses attributable to the research programs being conducted under the Research Collaboration and Option Agreement entered into between Ardelyx and KKC in 2019 and general R&D expenses. Of the overall tenapanor-related decrease, approximately $7.9 million was related to lower clinical study costs due to the winding down of expenses associated with the Phase 3 clinical program for tenapanor for the control of hyperphosphatemia, offset by an out-of-period adjustment that reduced clinical trial expenses by $4.1 million; and an approximately $2.1 million decrease in validation-related manufacturing expenses; offset by increase of $4.6 million related to regulatory expenses that included $2.9 million paid to the FDA for the filing of the NDA for tenapanor for control of serum phosphorus.
G&A Expenses: General and administrative expenses were $7.0 million for the three months ended June 30, 2020, an increase of $1.6 million, or approximately 31 percent, compared to $5.4 million for the three months ended June 30, 2019. The increase was primarily due to an increase in headcount and related personnel costs, including stock-based compensation costs, and an increase in professional services.
Net Loss: Net loss for the quarter ended June 30, 2020 was $25.0 million, as compared to $25.5 million for the quarter ended June 30, 2019.