On January 13, 2020 Illumina, Inc. (NASDAQ: ILMN), the global leader in DNA sequencing and array-based technologies, and Roche, a global pioneer in pharmaceuticals and diagnostics, reported a 15-year, non-exclusive collaboration agreement to broaden the adoption of distributable next-generation sequencing-based (NGS) testing in oncology (Press release, Illumina, JAN 13, 2020, View Source [SID1234553112]). As the understanding of genomic drivers of cancer evolves, NGS has the potential to transform cancer risk prediction, detection, diagnosis, treatment and monitoring.

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This agreement brings together complementary capabilities of each company to broaden global adoption of NGS in cancer care. As part of this agreement, Illumina will grant Roche rights to develop and distribute in-vitro diagnostic (IVD) tests on Illumina’s NextSeq 550Dx System, as well as on its future portfolio of diagnostic (Dx) sequencing systems, including the forthcoming NovaSeqDx. Roche will in turn collaborate with Illumina to complement Illumina’s comprehensive pan-cancer assay, TruSight Oncology 500 (TSO 500), with new companion diagnostic (CDx) claims. The financial terms of the deal were not disclosed.

Under the IVD terms of the agreement, Roche will develop, manufacture and commercialize AVENIO IVD tests for both tissue and blood for use on Illumina’s NextSeq 550Dx System. Illumina will continue to sell the NextSeq 550Dx Systems and core sequencing consumables. Under the CDx terms of the agreement, Illumina and Roche will develop tests and pursue CDx claims on TSO 500 for both existing and pipeline oncology targeted therapies on the NextSeq 550Dx System. Illumina will lead the development and regulatory approval process, and will continue to manufacture, supply and commercialize TSO 500. Roche will support the development of the claims and regulatory filings.

"We are excited Roche has selected Illumina’s sequencers as their platform of choice to accelerate the adoption and broaden the reach of oncology-based, distributable IVD tests into clinical care," said Francis deSouza, CEO of Illumina. "This partnership complements and strengthens our strategy to establish TSO 500 as a comprehensive NGS panel for cancer therapies by expanding the supported set of CDx claims on this universal panel. Building on the momentum of other recently established diagnostic and pharmaceutical partnerships, together we aim to advance critical access to NGS testing to improve patient outcomes."

Illumina’s TSO 500 is a comprehensive pan-cancer assay designed to identify known and emerging tumor biomarkers. TSO 500 utilizes both DNA and RNA from tumor samples to identify key somatic variants underlying tumor progression, such as small DNA variants, fusions and splice variants. When used to test for companion diagnostics claims, TSO 500 is poised to identify cancer patients globally who might benefit from targeted therapeutics. As the anchor tenant of Illumina’s oncology product portfolio, TSO 500 enables labs to adopt a comprehensive genomic profiling panel, in a decentralized testing model, leveraging the accurate and reproducible results of Illumina’s diagnostic sequencing systems.

On January 13, 2020 Median Technologies (Paris:ALMDT), The Imaging Phenomics Company reported its preliminary full year 2019 unaudited financial results (Press release, MEDIAN Technologies, JAN 13, 2020, View Source [SID1234553111]). These results fully validate the strategy adopted in 2018 .

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In 2019, Median strengthened its Research and Development efforts on the iBiopsy imaging phenomics platform. iBiopsy is based on the most advanced technologies in AI and data sciences. Using non-invasive imaging biomarkers, the platform enables the identification of certain chronic disease signatures including cancer signatures, to dramatically enhance early detection, severity quantification and monitoring of diseases. A prototype of the platform was presented during the Radiological Society of North America (RSNA) annual meeting in early December (Chicago), to showcase the potential of the iBiopsy technology. The prototype demo created a lot of traction and interest. The company’s objective is now to finalize ongoing discussions with partners regarding collaborations to conduct iBiopsy clinical validations on large patient cohorts.

On December 19th, 2019, the company signed a €35 million finance contract with the European Investment Bank (EIB). The financing is going to further accelerate the iBiopsy investment and recruitment program for the coming years. The disbursement of the first tranche of €15 million is expected during the first semester of 2020.

2019 has been a record year for Median’s iCRO business unit, which provides imaging solutions and services for oncology trials. As of December 31st, 2019, the annual revenue was €9 million, a 41% increase compared to 2018 revenue (€6.3 million). The company experienced a steady increase in its quarterly revenues all along 2019.

Median’s iCRO activity was already operationally at its break-event point as of June 30th, 2019. Full year performance demonstrated its capacity for sustainable growth and profitability.

As of December 31st, 2019, the order backlog reached €38.3 million, representing a €7.6 million increase compared to the backlog as of June 30th, 2019 and a €14.6 million increase compared to the backlog as of December 31st, 2018 (+61.6%). At the end of 2019, Chinese business represented 56% of the total order backlog compared to 39.2% at the end of 2018. As far as Europe and the US are concerned, Median largely exceeded all expectations, thanks to a dedicated sales strategy based on recurrent business. Strategy success shows a great deal of trust in Median’s execution, competitiveness and quality. In 2019, Median’s service quality was successfully validated through 12 client audits and one Food and Drug Administration (FDA) audit on a major Phase III study, sponsored by one of the top 3 pharma companies.

Considering the company’s performance, as of December 31st, 2019, the company cash and cash equivalents was €7.6 million, compared to €7.9 million as of June 30th, 2019 and €12.7 million as of December 31st, 2018. The company cash burn rate was €0.4 million per month over the year, dropping from €0.8 million per month over the first semester down to €0.3 million per month during the second semester, excluding the Research Tax Credit positive impact.

"Our excellent iCRO performance validates the strategy adopted in 2018 and we are pleased to see that the activity is now sustainable and profitable. We anticipate keeping up this momentum in 2020. Additionally, the extent of our order backlog makes us confident about our revenues for 2020", said Fredrik Brag, Median’s co-founder and CEO. "With the finance contract signed with EIB, we are now going to accelerate our iBiopsy investment program. 2020 is going to bring us partnerships and clinical data which will enable iBiopsy validations on large cohorts of patients and for different therapeutic indications", he added.

The preliminary results set forth above are based on management’s initial review of the Company’s operations for the year ended December 31, 2019 and are subject to revision based upon the Company’s year-end closing procedures and upon the completion and external audit of the Company’s year-end financial statements. Actual results may differ materially from these preliminary results as a result of the completion of year-end closing procedures, final adjustments and other developments arising between now and the time that the Company’s financial results are finalized, and such changes could be material. In addition, these preliminary results are not a comprehensive statement of the Company’s financial results for the fourth quarter or full year ended December 31, 2019, should not be viewed as a substitute for full, audited financial statements prepared in accordance with generally accepted accounting principles, and are not necessarily indicative of the Company’s results for any future period.

On January 13, 2020 Adimab, LLC, the global leader in the discovery and optimization of fully human monoclonal and bispecific antibodies, reported that 11 new partner programs entered clinical development in 2019 (Press release, Adimab, JAN 13, 2020, View Source [SID1234553110]). This brings the total number of Adimab partner programs that have entered the clinic to 32. Partners initiating clinical trials in 2019 include Alector, Dragonfly Therapeutics, Hanmi Pharmaceutical, IASO Therapeutics, Innovent Biologics, Inc., Roche, Surface Oncology, and others.

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"This is the second year in a row that we have seen 11 or more new programs enter the clinic and we don’t expect that trend to slow anytime soon. In particular, we have seen younger companies backed by leading venture capital firms show remarkable success in translating new biological insight into clinical programs. On average, these venture-backed partners have advanced two out of every three discovery programs towards the clinic – that’s an incredible success rate compared to the industry at large," said Tillman Gerngross, Chief Executive Officer and Co-Founder of Adimab. "People aren’t very good at pricing failure. We see many antibody discovery shops push their cheap alternatives, but too many of those programs fail for technical or developability reasons."

In 2019, Adimab partners exercised ten commercial licenses to advance programs into product development, bringing the total number of optioned programs to more than 60. Partners exercising commercial options in 2019 include Acceleron, Innovent Biologics, Inc., Scholar Rock, Surface Oncology, and others.

"While the majority of the programs in the clinic came from Adimab’s core antibody discovery platform, there are now several programs in the clinic that came from new applications of Adimab’s technology. Some programs are antibodies that originally came from other technologies that we subsequently engineered to enhance their properties, or simply ‘fixed’ to reduce developability liabilities. We are also seeing a growing number of bispecific and CAR programs entering the clinic, and we expect that trend to continue in the coming years," said Guy Van Meter, Chief Business Officer of Adimab.

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.