On January 13, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today highlighted its key accomplishments in 2019 and updated its corporate priorities (Press release, Zymeworks, JAN 13, 2020, View Source [SID1234553109]). Zymeworks also announced multiple clinical program advances for its lead candidates, ZW25 and ZW49.

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Recent ZW25 and ZW49 Program Advances

Zymeworks initiated a Phase 2 clinical trial evaluating ZW25 in combination with Ibrance (palbociclib), an oral CDK4/6 inhibitor, and the hormone therapy fulvestrant with the goal of providing a chemotherapy-free treatment option to people with advanced HER2-positive, hormone receptor-positive breast cancer. Zymeworks entered into a clinical collaboration with Pfizer to advance the study.
ZW25 achieved two additional regulatory milestones, recently being granted Fast Track and Orphan Drug Designations by the FDA in refractory biliary tract cancer (BTC). These were ZW25’s second Fast Track and third Orphan Drug Designations. ZW25 already holds a Fast Track Designation for 1st line gastroesophageal adenocarcinoma (GEA) and Orphan Drug Designations for GEA and ovarian cancer.
An interim update from the ongoing ZW49 Phase 1 dose-escalation study highlighted that there have been no dose-limiting toxicities observed and the maximum tolerated dose has not been reached. With over ten patients treated, the majority of treatment-related adverse events have been grade 1 or 2, and were reversible and manageable on an outpatient basis. Importantly, preliminary results from these initial dose cohorts include anti-tumor activity.
"Our significant achievements in 2019 demonstrate that we are executing on our vision to establish ZW25 as the foundational HER2 therapy across the spectrum of HER2-expressing cancers and lines of therapy," said Ali Tehrani, Ph.D., President and CEO at Zymeworks. "As our second therapeutic candidate, ZW49, continues to advance in the clinic, we are excited about its transformative potential for both patients with HER2-expressing cancers and for the development of future antibody-drug conjugate therapies. With a visionary leadership team, key hires company-wide, and a strong financial position, we are ready to deliver on our clinical programs and partnering priorities for 2020."

2019 Achievements

ZW25 Single Agent and Chemotherapy Combination Data Presented at Multiple Medical Meetings; Data Support Plans for Multiple Registration-Enabling Studies

ZW25 demonstrated robust single agent anti-tumor activity and durable disease control across multiple HER2-expressing tumors that have progressed after standard‑of‑care (SOC) therapies, including HER2-targeted agents. These data support the initiation of a registration-enabling Phase 2 trial evaluating single agent ZW25 in refractory HER2‑positive BTC followed by additional HER2‑expressing tumor types.
ZW25 plus chemotherapy showed durable anti-tumor activity for patients with heavily pretreated GEA, which supports the ongoing Phase 2 trial of ZW25 plus SOC chemotherapy and a planned registration-enabling trial in 1st line HER2-posititve GEA vs Herceptin plus SOC chemotherapy.
ZW49 Enters Phase 1 Clinical Testing for HER2-Expressing Cancers

ZW49 is a HER2-targeted bispecific antibody-drug conjugate (ADC) that capitalizes on the unique geometry and antibody framework of ZW25, and is armed with Zymeworks’ proprietary ZymeLink cytotoxin. This design results in enhanced internalization and delivery of the cytotoxin to cancer cells. ZW49 is being evaluated in an ongoing Phase 1 clinical trial.
Partnerships Continue to Advance; Milestone Payments Received

Zymeworks’ pharmaceutical partner Lilly entered the clinic with a novel bispecific, Merck, Celgene, and Daiichi Sankyo advanced bispecific candidates toward clinical testing, GSK expanded its Azymetric partnership, and the first ZymeLink ADC collaboration was signed with Iconic Therapeutics. These events resulted in multiple milestone payments and increased future potential milestone payments and royalties for Zymeworks.
Expertise Added to Leadership Team

Extensive development and commercial experience was added to the executive management team and board of directors. In addition, a Chief People Officer was hired to strategically manage the growth of Zymeworks’ human resources.
Balance Sheet Strengthened

During the year, Zymeworks completed a public financing raising US $201.3 million, and added additional non-dilutive capital from multiple pharmaceutical partners.
Updated Corporate Priorities

Initiate ZW25 registration-enabling studies in 2nd line HER2-positive BTC and 1st line HER2-positive GEA
Report ZW49 Phase 1 dose-escalation data and initiate expansion cohorts
Expand ZW25 clinical development into additional HER2-expressing cancers
Report ZW25 Phase 2 chemotherapy combination data from 1st line HER2-positive GEA
Continue building a strong preclinical pipeline through internal R&D and external partnerships

On January 13, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported its expected milestones for 2020-2021, which highlight the company’s progress advancing its clinical development candidates: omidubicel, an advanced cell therapy in Phase 3 clinical development as a potential life-saving treatment option for patients in need of bone marrow transplant, and GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (Press release, Gamida Cell, JAN 13, 2020, View Source [SID1234553108]).

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"This is an incredibly important year for Gamida Cell. With patient enrollment completed, we are expecting topline data from the Phase 3 study of omidubicel in the first half of this year. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and has the potential to be the first FDA-approved bone marrow transplant graft. Positive data from our study would represent an important step toward bringing potentially curative medicines to patients," stated Julian Adams, Ph.D., chief executive officer at Gamida Cell. "We are also very excited by the progress of GDA-201, an investigational therapy in a class that we believe has the potential to be the next generation of cell therapies to dramatically improve the treatment of blood cancers. GDA-201 has shown promise for the treatment of non-Hodgkin lymphoma, including an aggressive form of lymphoma called diffuse large B cell lymphoma, and we anticipate announcing additional data in the first half of 2020."

Today Gamida Cell also announced the appointment of Jas Uppal, Ph.D. to the newly created role of chief regulatory and quality officer. Dr. Uppal brings more than 25 years of global experience in the pharmaceutical industry, including expertise in hematology, immunology and neurology. During her career, she has played key roles in building regulatory organizations and leading multiple successful product launches.

"We are delighted to welcome Jas to Gamida Cell. Her strategic, global experience in building teams and securing multiple product approvals for novel biologics will be invaluable as we move omidubicel and GDA-201 through critical regulatory milestones," stated Dr. Adams.

Dr. Uppal most recently served as vice president, global head of regulatory affairs of oncology, endocrinology and rare diseases at Ipsen Biopharmaceuticals, where she held worldwide responsibility for Ipsen’s oncology, endocrinology and rare diseases portfolio. In this role, she led all areas of product development and managed a team of regulatory professionals. Prior to Ipsen, she served as vice president, global head of regulatory affairs at Karyopharm Therapeutics, where she was responsible for developing a global regulatory strategy and approach for multiple Phase 2 and Phase 3 programs that were being developed to treat hematological malignancies and solid tumors. Earlier in her career, Dr. Uppal held several regulatory-related positions over the course of 12 years at Biogen Idec (now Biogen) that culminated in her role as director of global emerging markets and head of development sciences. Dr. Uppal has participated in over 30 new drug approvals worldwide and has more than 30 publications in peer reviewed journals. She holds a Ph.D. in biochemistry from Kings College, University of London.

Expected 2020-2021 Milestones

Gamida Cell targets achieving the following milestones during 2020-2021:

Omidubicel

Report topline data from the Phase 3 study in the first half of 2020
Present data from the Phase 3 study at a medical meeting in the second half of 2020
Submit the biologics license application to the FDA in the second half of 2020, assuming positive data
Report additional data from the Phase 1/2 study in patients with severe aplastic anemia in the second half of 2020
Launch omidubicel in 2021, contingent upon FDA approval
GDA-201

Present additional data from the Phase 1 study in the first half of 2020
Submit company-sponsored investigational new drug application to FDA in the second half of 2020
Initiate a Phase 1/2 clinical study in patients with non-Hodgkin lymphoma in 2021
2020 Financial Outlook

Gamida Cell ended 2019 with cash, cash equivalents and available-for-sale securities of approximately $56 million (unaudited). The company expects that its current cash, cash equivalents and available-for-sale securities will support the company’s ongoing operating activities into the fourth quarter of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

About Omidubicel

Omidubicel, the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.4

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On January 13, 2020 Human Longevity, Inc., an innovator in providing data-driven health intelligence and precision health to physicians and patients reported that the Oncology Division was sold to NeoGenomics (NASDAQ NEO) for $37 Million (Press release, Human Longevity, JAN 13, 2020, View Source [SID1234553107]). The financing, a cash transaction, closed on Friday, January 10, 2020.

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David Karow, MD, PhD, President and Chief Innovation Officer of Human Longevity, Inc., said, "The sale of this division allows us to focus entirely on longevity and extending the healthy, high-performance human lifespan. Additionally, it will provide the firm with additional working capital, along with the recent Series A2 round of financing, to grow domestically and internationally."

On January 13, 2020 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that its Chief Executive Officer and President, Alan H. Auerbach, will be presenting at the 38th Annual J.P. Morgan Healthcare Conference at 12:00 p.m. PST on Wednesday, January 15, 2020 (Press release, Puma Biotechnology, JAN 13, 2020, View Source [SID1234553106]). Mr. Auerbach will be providing a corporate update that will include, among other things, the company’s preliminary estimate that it sold approximately 4,900 bottles of NERLYNX in the United States in the fourth quarter of 2019. This preliminary estimate is subject to completion of the Company’s customary closing and review procedures and could change based on that process. The slides to be discussed during the presentation are currently available on the Investors section of Puma’s website at View Source A live webcast of the presentation will also be available on January 15, 2020, at www.pumabiotechnology.com.

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On January 13, 2020 TriSalusTM Life Sciences ("TriSalus"), a company committed to transforming outcomes for patients with solid tumors, reported the launch of its TriNav Infusion System ("TriNav") (Press release, TriSalus Life Sciences, JAN 13, 2020, View Source [SID1234553105]). Powered by its proprietary Pressure-Enabled Drug DeliveryTM (PEDD) approach with SmartValveTM technology, TriNav is designed to help overcome the infusion barriers that limit therapeutic uptake in solid tumors, including hepatocellular carcinoma (HCC) and liver metastases.2,3

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The TriNav System can provide interventional radiologists intravascular tumor access with improved trackability, compatibility with standard angiographic catheters and workflow comparable to standard interventions.4

The tumor microenvironment creates pressure barriers that limit flow into solid tumors.5,6 PEDD with SmartValve creates a high-pressure gradient that improves delivery and penetration of therapy into tumors.7,8 The porous expandable SmartValve is designed to allow antegrade flow and leverages blood flow to carry the dose deep into the solid tumor.1,7 SmartValve enables therapy delivery to the intended target while minimizing non-target delivery that has been shown to damage healthy tissue.2,9

In a clinical study, PEDD with SmartValve demonstrated the ability to overcome tumor infusion barriers and significantly improve response rates in HCC.2 Outcomes from a retrospective, single-center study of patients with solitary HCC tumors who underwent treatment utilizing either PEDD or standard end-hole microcatheters, showed 100% Objective Response (OR) with PEDD versus 76.5% with standard end-hole microcatheters (p=0.019).2* Additionally, after first treatment, Pathological Response (PR) as shown by tumor necrosis percentage was significantly greater with PEDD (88.8%) vs. standard end-hole microcatheters (33.8%), (p=0.026).2* Improving response rates could potentially help more patients meet transplant criteria, lead to successful downstaging, bridging, and post-transplant survival.10

"Tumor-directed delivery of therapeutics is an exciting opportunity to help improve outcomes across a wider range of procedures by overcoming intratumoral pressure that can prevent drugs from adequately penetrating the tumor," said Mary Szela, President and CEO of TriSalus. "The new TriNav Infusion System utilizes SmartValve, a first-in-kind, proprietary technology that has been shown to modulate pressure and flow with the goal for improved therapeutic delivery and deeper penetration into the tumor while helping to protect healthy tissue."1-3,7,8

Acknowledging its unique technology for therapeutic delivery, the Centers for Medicare and Medicaid Services (CMS) granted TriNav transitional pass-through payment as part of the 2020 Medicare Hospital Outpatient Prospective Payment System and Ambulatory Surgical Center Payment System (CMS-1717-FC), effective January 1, 2020.12

This supplemental payment is intended to "facilitate access for [Medicare] beneficiaries to the advantages of new and innovative devices"13. The TriNav Infusion System met the required criteria to receive transitional pass-through status, including the demonstration of substantial clinical improvement, with the CMS saying it believes there is no existing pass-through payment category for this device because its SmartValve technology offers a unique mechanism for therapy delivery to selected sites in the peripheral vascular system, including solid tumors in the liver.12

*Study design: A retrospective, single-center study included 88 treatment-naive patients with solitary HCC tumors <6.5 cm who underwent treatment utilizing either PEDD (n = 18) or standard end-hole (EH) microcatheters (n = 70). Explant liver assessment of pathological response after first treatment: PEDD n=4; EH n=12.

About the TriNav Infusion System
The TriNav Infusion System is a 0.021-inch lumen microcatheter with SmartValve self-expanding tip. SmartValve supports pressure generation, which enables delivery of therapeutic agents to select sites in the peripheral vascular system, including solid tumors in the liver.2,3,8,14

The TriNav SmartValve has demonstrated the potential to overcome intratumoral pressure in solid tumors to improve distribution and penetration of therapy during arterial embolization procedures per clinical studies performed to date. 2,3,14

Intended Use: The TriNav Infusion System is intended for use in angiographic procedures. It delivers radiopaque media and therapeutic agents to selected sites in the peripheral vascular system.15

Contraindications: TriNav is not intended for use in the vasculature of the central nervous system (including the neurovasculature) or central circulatory system (including the coronary vasculature).15