On January 7, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported expected milestones for 2019, which highlight the company’s progress advancing its clinical development candidates: NiCord, an investigational universal bone marrow donor source in late-stage development for patients with hematologic malignancies (blood cancers), and NAM-NK, an investigational, cell-based cancer immunotherapy in development in patients with non-Hodgkin lymphoma and multiple myeloma (Press release, Gamida Cell, JAN 7, 2019, View Source [SID1234532542]).

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!

Gamida Cell expects to complete patient enrollment in its Phase 3 clinical study of NiCord in the second half of this year. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of NiCord compared to standard umbilical cord blood for allogeneic hematopoietic stem cell (bone marrow) transplant in approximately 120 patients with no available matched donor. The company also expects to report additional data from the ongoing Phase 1 study of NAM-NK at a medical meeting during the first half of 2019.

Today Gamida Cell also announced the appointment of Thomas Klima to the newly created role of chief commercial officer. Mr. Klima brings nearly 20 years of global experience in the pharmaceutical industry with expertise in cellular therapy, hematology, oncology and transplantation. During his career, he has played key roles in building commercial organizations and leading multiple successful product launches. Additionally, the company announced that Nurit Benjamini, an experienced financial executive, has been appointed to Gamida Cell’s board of directors and will chair the board’s audit committee.

"We are intently focused on advancing NiCord, which has potential to expand and improve bone marrow transplants, giving patients a chance for a cure. We are on track to complete enrollment by the end of this year, with topline data from the Phase 3 study anticipated in first half of 2020," stated Julian Adams, Ph.D., chief executive officer at Gamida Cell. "We are also continuing to build a pipeline based on our proprietary nicotinamide, or NAM, technology and plan to report additional data from the ongoing Phase 1 study of NAM-NK in the coming months."

"We have also augmented our team with two new experienced individuals. I’m pleased to welcome Tom Klima, who will join as chief commercial officer, and Nurit Benjamini, who will serve as an independent director on the company’s board. Their experience and contributions will be invaluable as we advance toward anticipated commercialization," Dr. Adams continued.

Mr. Klima most recently served as head of global commercial planning and operations at Atara Biotherapeutics, prior to which he played a key role as senior vice president and chief commercial officer at Navidea Biopharmaceuticals Ltd. Mr. Klima also served as head of sales and commercial operations at Algeta U.S. and led the successful commercial build-out and launch of Xofigo. Before Algeta, he held various commercial leadership positions at Dendreon. Mr. Klima began his pharmaceutical career at Eli Lilly where he held several positions of increasing responsibility and participated in the global launch of Cymbalta. Mr. Klima earned a B.A. in Business Administration and Marketing from Western State College.

Ms. Benjamini has served as chief financial officer of TabTale Ltd. since 2013. Previously, she held a number of chief financial officer positions, including at Wix.com Ltd., Sigma Designs Israel Ltd. and Compugen Ltd. She has served as a director at RedHill Biopharma Ltd., BioLine Rx and Allot Communications. Ms. Benjamini holds a BA in Economics and Business and an MBA in Finance from Bar Ilan University, Israel.

Expected 2019 Milestones

Gamida Cell expects to achieve the following milestones this year:

Present additional data from the Phase 1 study of NAM-NK in first half of 2019
Report early data from the Phase 1/2 study of NiCord in patients with severe aplastic anemia data in the first half of 2019
Complete patient enrollment in the Phase 3 study of NiCord in patients with high-risk hematologic malignancies in the second half of 2019
2019 Financial Outlook

Gamida Cell ended 2018 with cash, cash equivalents and available-for-sale securities of approximately $60 million (unaudited). The company expects that such funds will support the company’s capital needs through the data readout for the Phase 3 clinical study of NiCord, which is expected in the first half 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.

Gamida Cell intends to provide additional financial guidance for 2019 when it reports its fourth quarter and full-year 2018 financial results in March 2019.

About NiCord

NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 clinical study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.1 For more information on clinical trials of NiCord, please visit www.clinicaltrials.gov.

About NAM-NK

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. NAM-NK 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. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.2

NiCord and NAM-NK 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 7, 2019 NANOBIOTIX (Euronext : NANO – ISIN : FR0011341205) and The University of Texas MD Anderson Cancer Center reported a large-scale, comprehensive clinical research collaboration to evaluate innovative strategies for treating patients with head and neck, pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers (Press release, Nanobiotix, JAN 7, 2019, View Source [SID1234532558]).

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 collaboration will expand clinicaldevelopment of NBTXR3, a first-in-class agent designed to physically destroy cancer cells when activated by radiotherapy and to activate the immune system for both local control and systemic disease treatment.

MD Anderson will initially conductthe launch of nine new phase I/II clinical trials-involving around 340 patients-focused on evaluating the potential clinical benefit of NBTXR3 when activated by radiotherapy, either as a monotherapy or in combination with checkpoint inhibitors. The planned trials also will look at various disease stages, including low-risk/good prognostic patients, locally advanced disease, and metastatic disease. The trials will studyNBTXR3 across different radiation modalities such as radiation, re-irradiation, and compare different levels of intensity of radiation, to evaluate whether the addition of NBTXR3 to radiotherapy will improve progression-free survival, loco-regional control, quality of life and organ preservation.

Dr. Thomas Morris, Global Head of Development, Nanobiotix, said "It is important for Nanobiotix to collaborate with academic institutionsto develop a broad spectrum of clinical trials in an expedited fashion. This is a tremendous opportunity to strengthen our scientific and clinical understanding of the potential of NBTXR3 across the wide range of cancers treated with radiotherapy, with the goal of benefitting patients by applying novel research and bringing forward an innovative therapy. The collaboration between Nanobiotix and MD Anderson expands the number of NBTXR3 clinical trials to 16, illustrating our dedication to identify more effective treatments for cancer patients.

As part of the funding scheme for this collaboration, part of the payment will be made at the start of the collaboration. Other parts will be paid during development and the balance in case of FDA registration for a total of $12M investment.

This new collaboration between Nanobiotix and MD Anderson follows up the immunotherapeutic pre-clinical research collaboration, launched in April 2018, in lung cancer to explore NBTXR3 potential in immuno-oncology with checkpoint inhibitors, as well as its potential to control metastatic disease.

About NBTXR3 NBTXR3 is a first-in-class product designed to destroy, when activated by radiotherapy:
• tumors through physical cell death
• metastasis due to immunogenic cell death leading to activation of the immune system
NBTXR3 has a high degree of biocompatibility, requires one single administration before the whole radiotherapy treatment.

NBTXR3 is a late clinical stage product which has shown the potential for clinical benefit in advanced STS phase III randomized clinical trial.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity or 2 oropharynx in elderly and frail patients unable to receive chemotherapy. The other ongoing studies are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma.

Nanobiotix is also running an Immuno-Oncology development program. In the U.S., the Company received the FDA’s approval to launch a clinical study of NBTXR3 activated by radiotherapy in combination with anti-PD1 antibodies in lung, and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung cancer).

On January 7, 2019 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported it will provide a strategic business update to the investor community at the 37th Annual J.P. Morgan Healthcare Conference (Press release, Regeneron, JAN 7, 2019, View Source [SID1234532574]). Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer, and George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer, will discuss commercial and pipeline progress across the company’s portfolio. Slides and a webcast from the presentation may be accessed from the "Investors & Media" page of Regeneron’s website at View Source

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!

"Regeneron continues to advance an innovative, homegrown portfolio of marketed and investigational therapies for patients with a range of serious diseases," said Dr. Schleifer. "In 2018, we saw continued strong growth for EYLEA (aflibercept) Injection in retinal diseases and Dupixent (dupilumab) Injection in atopic dermatitis, as well as a positive initial reception to our two new launches — Libtayo (cemiplimab-rwlc) Injection for advanced cutaneous squamous cell carcinoma and Dupixent for asthma. On the development front, we now have more than 20 investigational candidates in human clinical trials and look forward to entering several more this year, as we continue to leverage our cutting-edge science and technology to bring new hope to patients in need."

EYLEA: Strengthening Market Leadership Position

·EYLEA† achieved $4.07 billion in 2018 U.S. net sales (based on preliminary, unaudited fourth quarter 2018 U.S. net sales of $1.07 billion), representing approximately 10 percent growth over 2017. (1)

·The U.S. Food and Drug Administration (FDA) has assigned an action date of May 13, 2019 for a new EYLEA indication in diabetic retinopathy.

·Regeneron is also advancing next-generation ophthalmology treatments, such as a high-dose formulation of EYLEA, which is expected to enter clinical trials in 2019.

Dupixent: Continued Growth and New Indications

· Dupixent*† uptake continues to accelerate in both its FDA-approved indications with positive trends in new-to-brand prescriptions following a direct-to-consumer television campaign for moderate-to-severe atopic dermatitis and the 2018 asthma launch.

· Additional important regulatory milestones are expected for Dupixent this year, including a March 11, 2019 FDA action date for adolescent atopic dermatitis (age 12-17), a European Medicines Association (EMA) decision for asthma and an FDA filing for chronic rhinosinusitis with nasal polyps.

·In 2019, Regeneron also expects to report results from a Phase 3 study of Dupixent in pediatric patients (age 6-11) with atopic dermatitis and initiate a Phase 2/3 program in Chronic Obstructive Pulmonary Disease. Phase 2 studies in grass allergy and peanut allergy are ongoing, as are combination studies with REGN3500* (IL-33 antibody) in atopic dermatitis and asthma.

* Collaboration program with Sanofi

Immuno-Oncology Platform

·Regeneron has seen encouraging early uptake from the U.S. launch of Libtayo*† for advanced cutaneous squamous cell carcinoma (CSCC). An EMA decision on Libtayo for advanced CSCC is expected in the first half of 2019, and pivotal and earlier studies in other cancers are continuing to enroll.

·Regeneron’s CD20xCD3 bispecific antibody, REGN1979, continues to progress with a potentially pivotal Phase 2 study in Follicular Lymphoma anticipated to begin in the first half of 2019 and a potentially pivotal Phase 2 study in Diffuse Large B-Cell Lymphoma anticipated to begin in the second half of 2019.

·Supported by Regeneron’s proprietary science and technology platforms, the company is advancing a new class of costimulatory bispecific antibodies for cancer, with two candidates expected to enter human clinical studies in 2019. These therapies have the potential to be used in combination with other Regeneron immuno-oncology therapies to address difficult-to-treat cancers.

·Earlier today, Regeneron and Sanofi announced a restructuring of their 2015 Immuno-oncology Discovery and Development Agreement. Regeneron and Sanofi have selected two investigational bispecific antibodies (MUC16xCD3 for mucin16-expressing cancers and BCMAxCD3 for multiple myeloma) for continued collaborative development. Regeneron will retain exclusive rights to all its other immuno-oncology programs, including additional xCD3 bispecifics and the new class of costimulatory bispecific antibodies. The bispecific antibody REGN1979 (CD20xCD3) remains exclusively owned by Regeneron.

"Over the last few years, we’ve made excellent progress with our immuno-oncology portfolio, which includes Libtayo, the first and only approved treatment for advanced cutaneous squamous cell carcinoma, as well as our first clinical-stage bispecific antibody, REGN1979," said Dr. Yancopoulos. "Regeneron now has one approved and five clinical-stage immuno-oncology therapies for a range of targets and modalities, which have the opportunity to be used as monotherapy or in combination with other agents. We’re particularly encouraged to be entering two new therapies into the clinic this year from our costimulatory bispecific portfolio. Building on our deep antibody engineering expertise, this new class of bispecific agents has the promise to treat certain cancers where other classes of immunotherapy have proven inadequate."

Additional Research and Development Updates

· In 2018, Regeneron entered four new molecules into the clinic: REGN4018, a MUC16xCD3 bispecific antibody for cancer; REGN4461, a leptin receptor (LEPR) agonist for lipodystrophy and obesity; REGN4659, a CTLA-4 antibody for cancer; and REGN5069, a GFRa3 antibody for pain.

· In 2019, four to six new molecules are expected to enter clinical development, including REGN5458, the BCMAxCD3 bispecific antibody which has already initiated a Phase 1 study, as well as two costimulatory bispecific antibodies for cancer.

· The Regeneron Genetics Center (RGC) continues to make important discoveries, including validating the genetic role of IL-33 in asthma and identifying a new genetic variant that protects against chronic liver disease. The RGC has now sequenced over 500,000 human exomes linked to detailed patient electronic health records and anticipates sequencing up to 500,000 more exomes in 2019.

Regeneron records net product sales of EYLEA in the United States. Outside the United States, EYLEA net product sales comprise sales by Bayer in countries other than Japan and sales by Santen Pharmaceutical Co., Ltd. in Japan under a co-promotion agreement with an affiliate of Bayer. The Company recognizes its share of the profits (including a percentage on sales in Japan) from EYLEA sales outside the United States within "Bayer collaboration revenue" in its Statements of Operations.

The Company’s 2019 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

This press release uses non-GAAP unreimbursed R&D and non-GAAP SG&A, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"). These non-GAAP financial measures are computed by excluding certain non-cash and other items from the related GAAP financial measure. Non-GAAP adjustments also include the estimated income tax effect of reconciling items.

The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company’s control (such as the Company’s stock price on the dates share-based grants are issued or changes in the fair value of the Company’s equity investments) or items that are not associated with normal, recurring operations (such as changes in applicable laws and regulations). Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company’s core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company’s non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP.

On January 7, 2019 Catalent, Inc. (NYSE: CTLT), the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, reported it has commenced a $200 million capital investment in its Biologics business to expand drug substance manufacturing capacity and drug product fill/finish capacity due to projected growth among existing and future customers (Press release, Catalent, JAN 7, 2019, https://www.catalent.com/index.php/news-events/news/Catalent-Invests-200-Million-to-Expand-Biologics-Capacity-and-Capabilities [SID1234532527]). The investments, phased over a three-year program, will be undertaken at the company’s biologics manufacturing sites in Madison, Wisconsin and Bloomington, Indiana. This follows a recent announcement to invest $14 million in packaging capabilities at the Bloomington site.

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!

Mammalian cell culture capacity will be increased at Madison with the build out of two new suites, each with a 2 x 2,000 liter single-use bioreactor system, providing additional clinical and commercial production capacity at the 2,000 or 4,000-liter batch scale. Work is expected to be completed by mid-2021 and will more than double Catalent’s commercial biomanufacturing capacity.

Additionally, fill/finish capacity at the Bloomington site will be expanded by 79,000 sq. ft., with both GMP and non-GMP capabilities. A high-speed flexible vial line, utilizing both ready-to-use (RTU) components and bulk filling, at a filling speed of 300 units per minute, will be installed along with a high-speed flexible syringe/cartridge line with a filling speed of over 300 units per minute, and a fully automated vial inspection machine.

"The expansions at both sites will support our customers’ development programs and commercial launches," commented Barry Littlejohns, President, Catalent Biologics and Specialty Drug Delivery. He added, "Catalent’s continued investments in innovative technologies and flexible capacity allow us to offer the most comprehensive solutions to bring important and innovative treatments to market faster."

Opened in April 2013 and recently expanded with the addition of a 2 x 2,000-liter single-use bioreactor suite and new laboratories, Catalent Biologics’ Madison facility specializes in development, manufacturing and analytical services for new biological entities and biosimilars. It is the home of the company’s proprietary GPEx cell line development technology, used to create high-yielding mammalian cell lines. This year, Catalent celebrated a significant milestone when the tenth biologic therapy utilizing GPEx technology was approved for commercial use. The Madison facility was designed for cGMP production from 10 to 4,000-liter scale, providing flexibility in batch size to meet client needs.

Catalent’s 875,000-square-foot biologics development and manufacturing facility in Bloomington employs a growing staff of 900 employees, with deep expertise in sterile formulation and extensive biomanufacturing and drug product fill/finish capacity across liquid and lyophilized vials, prefilled syringes, and cartridges. The site also recently achieved regulatory approval for a twentieth commercial product.

On January 7, 2019 Adimab, LLC, the global leader in the discovery and optimization of fully human monoclonal and bispecific antibodies, reported that its partner Innovent Biologics has received BLA approval in China for an antibody against PD-1 to treat Hodgkin’s Lymphoma (Press release, Adimab, JAN 7, 2019, View Source [SID1234532543]).

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!

Adimab and Innovent initiated a partnership in 2013 to discover antibodies against multiple targets selected by Innovent. The partnership has been expanded multiple times to add additional programs and to access Adimab’s bispecifics capabilities. Innovent currently has 12 programs in development derived from the Adimab Platform. The PD-1 program, which Innovent has partnered with Lilly, was initiated in the spring of 2013.

On December 27th, 2018, Innovent and its co-development partner, Eli Lilly, announced that Tyvyt (fully human anti-PD-1 therapeutic monoclonal antibody, generic name: sintilimab injection) has been granted approval for market authorization by the National Medical Products Administration of China ("NMPA", formerly the China Food and Drug Administration) for the treatment of patients with classical Hodgkin’s lymphoma (cHL) that has relapsed or refractory (r/r) after two or more lines of systemic chemotherapy (r/r cHL).

"Innovent has been an outstanding partner. Their ability to go from inception to an approved drug in five years is impressive by any standard. Innovent has a deep pipeline of quality programs and we expect them to be a major player in the Chinese pharmaceutical industry for years to come," said Tillman Gerngross, Chief Executive Officer and Co-Founder of Adimab.

"Adimab’s technology helped generate one of the most competitive PD-1 programs in the industry and the quality of the antibody allowed Innovent’s PD-1 program to go from initiating discovery to approval in China in less than six years," said Dr. Michael Yu, the Founder, Chief Executive Officer and Chairman of Innovent. "Adimab has been one of our most important partners. The quality of their work and professionalism are exceptional."

About Tyvyt (sintilimab injection)

Tyvyt (sintilimab injection) is an innovative drug co-discovered by Innovent and Adimab, jointly developed by Innovent and Eli Lilly and Company in China. Tyvyt (sintilimab injection) is a type of immunoglobulin G4 monoclonal antibody, which binds to the PD-1 molecule on the surface of T-cells, blocks the PD-L1 (Programmed Cell Death-1 Ligand-1, PD-L1 pathway) and reactivates T-cells to kill cancer cells. Tyvyt (sintilimab injection) is the only PD-1 antibody in China branded by both a local biopharmaceutical company and a global pharmaceutical company.