On December 11, 2019 Kite, a Gilead Company (Nasdaq: GILD), reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the investigational chimeric antigen receptor (CAR) T cell therapy, KTE-X19, for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) (Press release, Kite Pharma, DEC 11, 2019, View Source [SID1234552275]).

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 BLA submission is based on data from the Phase 2 ZUMA-2 trial, which demonstrated an overall response rate of 93 percent, including 67 percent with complete response, as assessed by an Independent Radiologic Review Committee (IRRC) following a single infusion of KTE-X19. In the safety analysis, Grade 3 or higher cytokine release syndrome (CRS) and neurologic events were seen in 15 percent and 31 percent of patients, respectively. No Grade 5 CRS or neurologic events occurred. Detailed findings from this trial were recently presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in Orlando.

"There remains a significant need for new treatments for patients with relapsed/refractory MCL despite recent advances, so this regulatory filing is an especially important milestone for the MCL community," said Ken Takeshita, MD, Kite’s Global Head of Clinical Development. "We look forward to working with the FDA to bring KTE-X19 to appropriate patients as quickly as possible and continuing to deliver on the promise of our industry-leading cell therapy development program with a second CAR T therapy."

Kite plans to submit a Marketing Authorization Application for KTE-X19 in the European Union in early 2020. KTE-X19 has been granted Breakthrough Therapy Designation (BTD) by the FDA and Priority Medicines (PRIME) by the European Medicines Agency (EMA) for relapsed or refractory MCL.

KTE-X19 is investigational and not approved anywhere globally. Its efficacy and safety have not been established. More information about clinical trials with KTE-X19 is available at www.clinicaltrials.gov.

About MCL

MCL is a rare form of non-Hodgkin lymphoma (NHL) that arises from cells originating in the "mantle zone" of the lymph node and typically affects men over the age of 60.

About ZUMA-2

ZUMA-2 is a single-arm, multicenter, open-label Phase 2 study involving 74 enrolled/leukapheresed adult patients (≥18 years old) with MCL whose disease is refractory to or has relapsed following up to five prior lines of therapy, including anthracycline or bendamustine-containing chemotherapy, anti-CD20 monoclonal antibody therapy and the BTK inhibitors ibrutinib or acalabrutinib. The objectives of the study are to evaluate the efficacy (60 patients) and safety (68 patients) after a single infusion of KTE-X19 in this patient population. The primary endpoint for the study is objective response rate (ORR). ORR in this trial is defined as the combined rate of complete responses and partial responses as assessed by an IRRC.

Secondary endpoints include duration of response, progression-free survival, overall survival, incidence of adverse events, incidence of anti-CD19 CAR antibodies, levels of anti-CD19 CAR T cells in blood, levels of cytokines in serum, and changes over time in the EQ-5D scale score and visual analogue scale score. The study is ongoing.

About KTE-X19

KTE-X19 is an investigational, autologous, anti-CD19 CAR T cell therapy. KTE-X19 uses the XLP manufacturing process that includes T-cell selection and lymphocyte enrichment. Lymphocyte enrichment is a necessary step in certain B-cell malignancies with evidence of circulating lymphoblasts. KTE-X19 is currently in Phase 1/2 trials in acute lymphoblastic leukemia (ALL), mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).

On December 11, 2019 Omega Funds, a leading international investment firm focused on delivering impactful medicines to patients, reported the closing of Omega Fund VI, L.P. ("Fund VI"), with $438 million in capital commitments (Press release, Omega Fund Management, DEC 11, 2019, View Source [SID1234552274]). With Fund VI, the firm will continue to execute on its strategy of creating and investing in life sciences companies that target the most urgent medical needs. Since its inception in 2004, Omega Funds has raised more than $1 billion to invest in exceptional entrepreneurs developing innovative products across multiple therapeutic areas, including oncology, immunology, rare diseases, precision medicine and others.

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!

Omega Funds has a strong track record of identifying and supporting great management teams and companies by providing domain knowledge and perspective, network connectivity, and capital. Omega Funds’ portfolio companies have brought 37 new products to market and the firm’s investments have contributed to 24 successful portfolio company IPOs and 35 portfolio company exits via M&A.

"We appreciate the trust from our limited partners and their support of our distinctive investment style, which is guided by our conviction in people, products and breakthrough ideas, not by conventional categories," said Otello Stampacchia, Ph.D., Managing Director of Omega Funds. "Fund VI builds on our modus operandi aimed at investing in and, in many cases, actively combining innovative science with exceptional founders and company builders."

Fund VI is stage-agnostic and is expected to be deployed across companies in the U.S. and Europe. Similar to past funds, Fund VI investments will include a variety of investment approaches, from company founding and creation to early venture rounds and late-stage public investing, as well as direct secondary transactions.

"We believe this is the most exciting time to be investing in life sciences in a generation, but it requires diverse skills to capture the opportunity set," added Dr. Stampacchia. "At Omega, we have thoughtfully assembled a team that has the experience, insights and connections needed to successfully identify and harvest innovation from both sides of the Atlantic."

Omega Funds also announced today that industry veteran Bernard Davitian will be joining the firm as a Partner, beginning January 2020. Recent additions to the Omega Funds team also include Deirdre Cunnane, J.D., who joined as Chief Operating Officer and General Counsel in October 2019, as well as Paulina Hill, Ph.D., who joined as Principal, and Katie Kerfoot, who joined as head of Investor Relations, both of whom joined in January 2019.

Mr. Davitian has extensive experience in the life sciences and biotech industry, marked by a number of successful transactions involving financings and M&A. He previously served as SVP and Managing Director at Sanofi Ventures, having first joined Sanofi in 2010 as Vice President, Deputy Global Head, Business Development. Prior to Sanofi, Mr. Davitian held leadership roles at Fovea Pharmaceuticals, which was sold to Sanofi in 2009 in a €370 million cash transaction, Neurotech Pharmaceuticals, Transgene, which successfully raised $100 million in a 1998 IPO on the NASDAQ and EuroNext, as well as Institut Mérieux (now Sanofi Pasteur).

Ms. Cunnane, COO & GC, has held similar roles both in venture funds and within a biotech company. She joins Omega Funds from Catabasis Pharmaceuticals, a publicly traded biotech company where she held various leadership positions over the past four years. Prior to joining Catabasis, Ms. Cunnane served as General Counsel at Advanced Technology Ventures and Lightstone Ventures.

Prior to joining Omega Funds, Dr. Hill was on the healthcare team at Polaris Partners since 2012. She completed her postdoctoral fellowship in Robert Langer’s lab in the Chemical Engineering department at the Massachusetts Institute of Technology and earned her Ph.D. in Molecular Medicine from the Wake Forest University School of Medicine.

Ms. Kerfoot joined Omega Funds to lead the firm’s communications strategy and investor relations initiatives, including marketing and fundraising. Prior to joining Omega Funds, she covered specialty pharmaceutical companies as an equity research analyst at Wells Fargo Securities and BMO Capital Markets in New York. Ms. Kerfoot has an extensive journalism background, including on-air reporting with an NBC-affiliate local news station and a national online news network.

On December 11, 2019 IASO Biotherapeutics (IASO Bio), a clinical-stage biotechnology company advancing the development of innovative therapies for cancer, reported that had the latest clinical data presented on their potential best-in-class therapy at the "Efficacy and Safety of Fully Human BCMA Targeting CAR T Cell Therapy in Relapsed Refractory Multiple Myeloma" session during the prestigious 61st Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting & Exposition, December 7-10 in Orlando, FL (Abstract #582) (Press release, IASO BioMed, DEC 11, 2019, View Source [SID1234552273]).

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!

In an IIT study conducted by Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, the oral presentation highlighted the impressive safety, efficacy and persistence of CT103A, an anti-BCMA CAR-T for the treatment of Relapsed/Refractory Multiple Myeloma. With 17 of the 18 patients evaluable, the objective response rate (ORR) was 100%. In addition, 70.6% of patients achieved a best response of stringent complete or complete response (sCR/CR), and 88.2% achieved a best response of very good partial response (VGPR) or better. CRS occurred in 17 of 18 patients (Grade 1&2- 72.2% (13), Grade 3- 16.7% (3), Grade 4- 5.6% (1)), but was generally manageable with no neurotoxicity. At the lowest dose (1 x106 cells/kg), CT103A still maintained 100% ORR, with 78% of these patients achieving a best response of very good partial response (VGPR) or better. As well, toxicity was manageable with 88% of these patients experiencing grade 2 CRS or less.

In addition, 4 patients participating in the study had relapsed from a prior murine CAR-T infusion. Their response, and the overall performance, suggests that CT103A may also provide patients, having relapsed from a prior CAR-T, a potential first-line treatment option

"Having presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) earlier this year, our presence at ASH (Free ASH Whitepaper) marks the end of a very exciting year for IASO BIO. We are very happy to see the prolonged patient response to this therapy and look forward to starting our phase II clinical trial early in the new year," said Hu Guang, Ph.D., Director of R&D at IASO BIO. "By applying highly innovative science in our pre-clinical programs, we believe our pipeline has potential to bring us closer to addressing critical unmet needs, not just for patients, but for the healthcare professionals that treat them."

Multiple Myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems and bone fractures. With a global annual incidence rate of 2/100,000 persons, it is one of the most commonly diagnosed blood cancers, second only to non-Hodgkin lymphoma.

This past September CT103A received IND approval for Ib/II chimeric protocol by the National Medical Products Association (NMPA), and phase II clinical trials are expected to begin in early 2020.

About Relapsed/Refractory Multiple Myeloma

For newly treated patients with multiple myeloma, common first-line treatment drugs include proteasome inhibitors, immunoregulatory drugs and alkane agents. For most patients, the commonly used first-line treatment can stabilize the patient’s condition for 3-5 years, but a small number of patients show primary drug resistance at the time of initial treatment, and the disease cannot be effectively controlled. Relapse patients are those who have a reoccurrence after complete remission of the disease. Refractory patients are those with primary drug resistance or those who have finished first-line treatment and do not achieve remission, or patients whose disease progress within 60 days after achieving minimal response. With effective treatment, the majority of patients will inevitably enter the stage of relapse and refractory after 3-5 years of disease stabilization. For these patients, the overall effective rate of existing second-line treatment is about 40% to 70%, with short remission time.

About CT103A

CT103A is an innovative therapy co-developed by IASO BIO and Innovent. Previous studies indicate patients with relapsed/refractory multiple myeloma (RRMM) who received high-dose BCMA-targeting CAR-T cells may achieve better remission but have worse adverse events. Moreover, once the disease progresses again, the re-infusion of CAR-T cells is not effective. To solve this dilemma, CT103A has been developed. A lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3z activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, the construct of the CT103A CAR-T is potent and persistent.

On December 11, 2019 NeoImmuneTech, Inc., a clinical-stage T cell-focused biopharmaceutical company, reported it has entered into a clinical collaboration agreement with Merck, known as MSD outside of the United States and Canada, through a subsidiary, to evaluate the combination of NeoImmuneTech’s Hyleukin-7 (NT-I7) and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in a basket study in patients with relapsed/refractory (R/R) advanced solid tumors (Press release, NeoImmuneTech, DEC 11, 2019, View Source [SID1234552272]).

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 goal of the Phase 1b/2a study is to establish a recommended dosing regimen and explore the preliminary anti-tumor activity of the combination in patients with both checkpoint inhibitor (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.

"Although immunotherapy has become the new paradigm in cancer treatment, the majority of patients fail to respond. For tumors that are considered non-responsive to CPIs, such as microsatellite stable colorectal cancer and pancreatic cancer, the response to single-agent CPI has been low," said NgocDiep Le, M.D., Ph.D., NIT’s Executive VP and Chief Medical Officer. "Since immunotherapies rely heavily on the anti-tumor activity of T cells, Hyleukin-7’s demonstrated ability to increase multiple T-cell subsets potentially enhances the breadth and depth of the response to CPIs such as KEYTRUDA."

Se Hwan Yang, Ph.D., NIT’s Co-President and Chief Executive Officer, added: "We are excited to partner with Merck in our endeavor to provide new treatment options for patients whose tumors do not respond to CPIs or have progressed after CPI treatment. This combination approach could open the door to expanding the use of immunotherapy to these patients and ultimately improve clinical outcomes."

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 December 11, 2019 Pyxis Oncology, an immuno-oncology company focused on developing a new family of antibody-based immunotherapies derived from novel insights into the biology of the tumor microenvironment, reported the appointment of Lara Sullivan, M.D. as Chief Executive Officer and Director (Press release, Pyxis Oncology, DEC 11, 2019, View Source [SID1234552271]). She succeeds Longwood General Partner and founding Chief Executive Officer, David Steinberg, who assumes the role of Chairman of the Board of Directors while John Flavin, the founding Chairman, will remain on the Board as an independent Director.

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!

"This is a very exciting time in immuno-oncology and Pyxis has a unique platform to understand the tumor microenvironment that will enable the company to make major contributions to the field," said Lara. "I am looking forward to working closely with our scientific co-founders and the Pyxis scientific team to unlock new avenues to restore the potency of dysfunctional immune cells. Building off this solid foundation with the support of our investors, I believe Pyxis will be able to make a real difference in the lives of cancer patients as we move our antibody programs into the clinic."

"We are excited to welcome Lara to Pyxis, and look forward to her leadership as we build and advance our pipeline. Lara brings a remarkable range of skills and experience spanning drug development, corporate strategy, finance and business development to the role of CEO at Pyxis," said David Steinberg, Chairman, Pyxis Oncology. "In addition, I would like to thank our co-founder and outgoing Chairman John Flavin for his exceptional early leadership and pivotal role in conceptualizing and establishing Pyxis. We look forward to his ongoing engagement and further contributions as an independent Director."

Prior to joining Pyxis, Lara was Founder and President of SpringWorks Therapeutics where she conceived of and executed the clinical stage spin-out from Pfizer and raised the Series A. In her executive roles at Pfizer, she led early stage R&D portfolio operations and strategy, and developed novel business models for early stage financing. Previously, Lara was an Associate Partner at McKinsey & Co. and a Principal at Paul Capital Partners, and earlier in her career worked in healthcare finance at CS First Boston. She is an independent Director of Rexahn Pharmaceuticals. Lara holds an M.D. from the University of Pennsylvania School of Medicine, an MBA from The Wharton School at the University of Pennsylvania, and a B.A. in Comparative Literature from Cornell University.