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]).

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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.

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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).

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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]).

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"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.

On August 6, 2020 Tessa Therapeutics (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments, reported the publication of results in the Journal of Clinical Oncology from two investigator-sponsored Phase 1/2 trials led by Baylor College of Medicine and the University of North Carolina Lineberger Comprehensive Cancer Center (Press release, Tessa Therapeutics, AUG 6, 2020, View Source [SID1234563224]).

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Results of the trials, which evaluated the safety and efficacy of CD30 CAR-T cell therapy in patients with relapsed/refractory ("R/R") Hodgkin lymphoma, showed a high rate of durable complete responses and very favorable safety profile using autologous CD30 CAR-T cell therapy.

"These data are significant, as they demonstrate that CAR-T cell therapy may be a safe and effective treatment option for patients with Hodgkin lymphoma and potentially other lymphomas expressing the CD30 antigen," said Dr. Natalie Grover, study co-first author, assistant professor in the UNC Department of Medicine and a UNC Lineberger member. "The highest dose treatment led to the complete disappearance of tumors in the majority of patients, and almost all subjects had clinical benefit. As such, we believe further study of this treatment approach is warranted," said Dr. Carlos Ramos, study co-first author, professor at the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital and Texas Children’s Hospital.

The trials enrolled 41 adult patients with relapsed/refractory Hodgkin Lymphoma who received CD30 CAR-T cell therapy following lymphodepletion with chemotherapy. Overall, 94 percent of the treated patients were still alive a year after treatment. Of the patients who had a complete response, 61 percent still had no evidence of recurrence a year later. None of the patients experienced the serious, life-threatening complications that have been seen with several CD19 CAR-T cell trials. The overall response rate in the 32 patients with active disease who received fludarabine-based lymphodepletion was 72%, including 19 patients (59%) with complete response.

"We have been working with Baylor and the University of North Carolina to confirm these impressive results further in a Tessa-sponsored regulatory Phase 2 trial, which we aim to initiate this year," said Ivan D. Horak, M.D., President of Research and Development at Tessa Therapeutics. "Longer term, we seek to explore the potential of this therapy beyond Hodgkin’s lymphoma to CD30+ expressing Non-Hodgkin lymphomas, where there is a demonstrated unmet need."

University of North Carolina has granted Tessa an exclusive license to its patents, data and know-how, and Baylor College of Medicine has granted Tessa the rights to use its data and know-how, for the further development and commercialization of this therapy. "We are excited to collaborate with Tessa. Their ability to run multi-center cell therapy clinical trials will be invaluable for the further development of this therapy," said Helen Heslop, director of the Center for Cell and Gene Therapy and Dan L Duncan Chair at Baylor.