On July 29, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported its half year 2021 financial results and provided a second quarter business update and outlook for the remainder of the year (Press release, argenx, JUL 29, 2021, View Source [SID1234585377]).

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"The first half of 2021 has been marked by clinical, financial and regulatory achievements for argenx. As we look toward 2022, we believe we are well-positioned to build on the impressive progress we have made with our first-in-class FcRn antagonist, efgartigimod. We are expanding our commercial organization to reach patients living with generalized myasthenia gravis this year and expect that these investments will benefit us in the future and support our growing, differentiated pipeline," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"Beyond myasthenia gravis, we are expanding the breadth of efgartigimod into our fifth and sixth indications, myositis and bullous pemphigoid, and simultaneously investing in potential scientific breakthroughs through our Immunology Innovation Program (IIP). Our first-in-class C2 inhibitor, ARGX-117, emerged from the IIP and has the potential to be our next pipeline-in-a-product opportunity. Collectively, the demonstrated execution this year supports our ‘argenx 2025’ vision and brings us closer than ever to becoming a global, integrated, immunology company," concluded Mr. Van Hauwermeiren.

SECOND QUARTER 2021 AND RECENT BUSINESS UPDATE

During its July 20th R&D Day, argenx introduced its long-term vision to becoming a global, integrated immunology organization. The ‘argenx 2025’ vision includes the following goals:

Efgartigimod being globally available to patients across its three expanding commercial franchises in neuromuscular diseases, hematology and dermatology
Efgartigimod either being commercially available or in clinical development in 15 active indications
Progress across broader immunology pipeline with ARGX-117 in multiple late-stage trials and ARGX-119 demonstrating proof-of-concept
Investment in continued expansion of differentiated pipeline through its Immunology Innovation Program (IIP), generating one new asset into pipeline each year
On track with buildout of global commercial organization in anticipation of potential approval of efgartigimod for treatment of generalized myasthenia gravis (gMG)

Biologics License Application (BLA) under review with U.S. Food and Drug Administration (FDA) with target action date of December 17, 2021 under Prescription Drug User Fee Act (PDUFA)
Marketing Authorization Application (J-MAA) under review with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) with anticipated approval in first half of 2022
MAA on track and expected to be filed with European Medicines Agency (EMA) in second half of 2021
Zai Lab on track with expected regulatory discussions with National Medical Products Administration (NMPA) for approval in China
ADAPT Phase 3 trial results of efgartigimod for treatment of gMG published in The Lancet Neurology
Hiring of salesforce expected to be completed in U.S. in third quarter of 2021 and in Japan in fourth quarter of 2021
Ongoing engagement with gMG patient community through awareness and advocacy efforts, including award-winning docuseries "A Mystery to Me", and continued enrollment into real-world evidence study, MyRealWorldMG

Efgartigimod is currently being evaluated in five ongoing registrational trials across four indications, including ADAPT-SC (gMG), ADHERE (chronic inflammatory demyelinating polyneuropathy or CIDP), ADVANCE (IV) and ADVANCE-SC (primary immune thrombocytopenia or ITP), and ADDRESS (pemphigus)

Completion of enrollment expected by end of 2021 in ADAPT-SC and ADVANCE (IV); topline data for both trials expected in first half of 2022
Broadened efgartigimod opportunity with announcement of new indications, idiopathic inflammatory myopathies (myositis) within neuromuscular franchise and bullous pemphigoid within dermatology franchise
Phase 2/3 trial of efgartigimod for treatment of myositis to start by end of 2021, pending interactions with FDA
Phase 3 registrational trial of efgartigimod for treatment of bullous pemphigoid to start by end of 2021
Phase 2 proof-of-concept trials of efgartigimod in additional indications to be evaluated as part of collaboration with Zai Lab

Phase 1 healthy volunteer data of C2-inhibitor, ARGX-117, support path forward into multifocal motor neuropathy (MMN)

Favorable safety profile demonstrated across single and multiple ascending doses and both IV and SC formulations
Pharmacokinetic/pharmacodynamic profiles demonstrate potential for infrequent dosing schedules
Phase 2 trial of MMN patients on track to start by end of 2021

Immunology Innovation Program (IIP) continues to bring value to argenx through internal pipeline programs, partnerships and licensing agreements

ARGX-119, a SIMPLE Antibody aimed at boosting the neuromuscular junction in disease, emerging from IIP to be next pipeline candidate within neuromuscular franchise
Regained worldwide rights to anti-CD70 antibody cusatuzumab from Janssen; argenx to evaluate potential alternatives to advance cusatuzumab through partnership
15-20 discovery programs under evaluation at any point in time that have emerged from IIP

DETAILS OF THE FINANCIAL RESULTS

As of January 1, 2021, the Company changed its functional and presentation currency from euro to U.S. dollars, which results in reporting financial highlights in U.S. dollar as compared to euro in prior periods. Historical financials have been converted at the average exchange rate of the related period.

Cash, cash equivalents and current financial assets totaled $2,731.0 million as of June 30, 2021, compared to $1,996.5 million on December 31, 2020. The increase in cash and cash equivalents and current financial assets resulted primarily from (i) the closing of a global offering, which resulted in the receipt of $1,092.1 million in net proceeds in February 2021, (ii) the net receipt of a $73.1 million non-creditable, non-refundable development cost-sharing payment received from Zai Lab as part of the strategic collaboration for efgartigimod in Greater China, (iii) the payment of $98.0 million related to the purchase of the priority review voucher from Bayer HealthCare Pharmaceuticals, and other net cash flows used in operating activities.

Total operating income increased by $453.2 million for the six months ended June 30, 2021 to $487.5 million, compared to $34.3 million for the six months ended June 30, 2020. The increase was primarily due to the recognition of the transaction price as a consequence of the termination of the collaboration agreement with Janssen, resulting in the recognition of $315.1 million and the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue.

Research and development expenses increased by $84.7 million for the six months ended June 30, 2021 to $273.9 million, compared to $189.3 million for the six months ended June 30, 2020. The increase in the first six months of 2021 resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in various indications and other clinical and preclinical programs. Furthermore, the research and development personnel expenses increased due to a planned increase in headcount and the increased costs of the share-based payment compensation plans related to the grant of stock options.

Selling, general and administrative expenses totaled $129.6 million for the six months ended June 30, 2021, compared to $67.9 million for the six months ended June 30, 2020. The increase resulted primarily from higher personnel expenses, including the costs of the share-based payment compensation plans related to the grant of stock options, and consulting fees linked to the preparation of a possible future commercialization of argenx’s lead product candidate efgartigimod.

The change in fair value on non-current financial assets amounted to $11.2 million for the six months ended June 30, 2021, which is the result of the closing of a Series B financing round of AgomAb Therapeutics, for which argenx maintains a profit share in exchange for granting the license for the use of HGF-mimetic antibodies from the SIMPLE AntibodyTM platform.

Exchange losses totaled $18.4 million for the six months ended June 30, 2021, compared to an exchange gain of $0.2 million for the six months ended June 30, 2020. As a result of the change in the Company’s functional and presentation currency, the exchange losses for the six months ended June 30, 2021 are reflecting the unfavorable change in euro/U.S. dollar exchange rate, mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial asset position in euro.

FINANCIAL GUIDANCE

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx continues to expect its 2021 cash burn to approximately double from 2020. The increased spend will support the Company’s transition to an integrated immunology company, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including expected global trials of efgartigimod in six indications, and the continued investment in its Immunology Innovation Program.

EXPECTED 2021 FINANCIAL CALENDAR

October 28, 2021: Q3 2021 financial results and business update

CONFERENCE CALL DETAILS
The half year 2021 financial results and second quarter business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

On July 29, 2021 West Pharmaceutical Services, Inc. (NYSE: WST) reported its financial results for the second-quarter 2021 and updated full-year 2021 financial guidance (Press release, West Pharmaceutical Services, JUL 29, 2021, View Source [SID1234585376]).

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Second-Quarter 2021 Summary (comparisons to prior-year period)

Net sales of $723.6 million grew 37.3%; organic sales growth was 30.6%.
Reported-diluted EPS of $2.47 increased 104%.
Adjusted-diluted EPS of $2.46 increased 97%.
Company is raising full-year 2021 net sales guidance to a new range of $2.760 billion to $2.785 billion, compared to a prior range of $2.630 billion to $2.655 billion.
Company is raising full-year 2021 adjusted-diluted EPS guidance to a new range of $8.05 to $8.20, compared to a prior range of $6.95 to $7.10.
"Adjusted-diluted EPS" and "organic sales growth" are Non-U.S. GAAP measurements. See discussion under the heading "Non-U.S. GAAP Financial Measures" in this release.

"Our strong second quarter performance was driven by continued momentum in organic sales growth in both our base business as well as increased demand for our products associated with COVID-19 vaccines," said Eric M. Green, President and Chief Executive Officer. "Guided by our mission, our dedicated team members remain focused on meeting the increased demand for our high-value product (HVP) components. Our market-led strategy coupled with our scientific leadership and regulatory insights in primary packaging and delivery of injectable drugs have resonated with our customers, resulting in a high participation rate on newly approved molecular entities, especially large molecule therapies. Over the past year and half, we have accelerated capital spending and expanded global HVP manufacturing capacity. Based on growing future demand from our customers, we are announcing another tranche of capital spending to expand HVP capacity that we expect will be ready for production in 2022. Given the strong first half of the year, we are raising our full-year financial guidance."

Proprietary Products Segment

Net sales grew by 47.0% to $587.3 million. Organic sales growth was 39.3%, with currency translation increasing sales growth by 770 basis points. HVP sales represented over 70% of segment sales and generated double-digit organic sales growth, led by customer demand for Westar, NovaPure, FluroTec, Daikyo and Envision components.

The Biologics and Pharma market units had strong double-digit organic sales growth, and the Generics market unit had low-single digit organic sales growth.

Contract-Manufactured Products Segment

Net sales grew by 6.7% to $136.4 million. Organic sales growth was 3.2% with currency translation increasing sales growth by 350 basis points. Segment performance was led by sales of healthcare-related injection and diagnostic devices.

Financial Highlights (first six months of 2021)

Operating cash flow was $233.1 million, an increase of 13.6%. Capital expenditures were $111.6 million, an increase of 61% over the same period last year. Free cash flow (operating cash flow minus capital expenditures) was $121.5 million, a decrease of 10.7%.

During the first-half 2021, the Company repurchased 479,000 shares for $137.1 million at an average share price of $286.23 under its share repurchase program.

Our capital and financial resources, including overall liquidity, remain strong. We believe that cash on hand and cash generated from operations, together with availability under our $300.0 million multi-currency revolving credit facility, will be adequate to address our foreseeable liquidity needs based on our current expectations of our business operations, capital expenditures and scheduled payments of debt obligations.

Full-Year 2021 Financial Guidance

Full-year 2021 net sales are expected to be in a range of $2.760 billion to $2.785 billion, compared to a prior guidance range of $2.630 billion to $2.655 billion.
Organic sales growth is expected to be in a range of 24% to 25%, compared to a prior range of 19% to 20%.
Net sales guidance includes an estimated full-year 2021 benefit of $80 million based on current foreign exchange rates, compared to a prior estimated benefit of $75 million.
Full-year 2021 adjusted-diluted EPS is expected to be in a range of $8.05 to $8.20, compared to a prior range of $6.95 to $7.10.
Full-year adjusted-diluted EPS guidance range includes an estimated benefit of approximately $0.27 based on current foreign currency exchange rates, compared to a prior estimated benefit of $0.23.
The revised guidance includes a $0.24 EPS positive impact from first-half 2021 tax benefits from stock-based compensation.
For the remainder of the year, our EPS guidance range assumes a tax rate of 23% and does not include potential tax benefits from stock-based compensation. Any tax benefits associated with stock-based compensation beyond those recorded in the first-half 2021 would provide a positive adjustment to our full-year EPS guidance.
Second-Quarter 2021 Conference Call
The Company will host a conference call to discuss the results and business expectations at 9:00 a.m. Eastern Time today. To participate on the call please dial 877-930-8295 (U.S.) or 253-336-8738 (International). The conference ID is 3089097.

A live broadcast of the conference call will be available at the Company’s website, www.westpharma.com, in the "Investors" section. Management will refer to a slide presentation during the call, which will be made available on the day of the call. To view the presentation, select "Presentations" in the "Investors" section of the Company’s website.

An online archive of the broadcast will be available at the website three hours after the live call and will be available through Thursday, August 5, 2021, by dialing 855-859-2056 (U.S.) or 404-537-3406 (International) and entering conference ID 3089097.

On July 29, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported the appointment of Chris H. Takimoto, M.D., Ph.D., F.A.C.P., to the role of Chief Medical Officer, effective today (Press release, IGM Biosciences, JUL 29, 2021, View Source [SID1234585375]). Dr. Takimoto will be responsible for global development of IGM’s clinical pipeline of proprietary IgM antibodies. He joins IGM with 30 years of experience in cancer research and development, most recently as Senior Vice President, Oncology, Gilead Sciences, Inc. Daniel S. Chen, M.D., Ph.D., will continue to assist the Company in a consulting capacity.

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"Dr. Takimoto has a proven track record of shepherding novel oncology product candidates through clinical development together with broad expertise in oncology and pharmacology gained through a distinguished industry, academic, and public service career," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We look forward to working with him and our clinical team in continuing to advance our growing clinical pipeline of IgM antibodies. We would also like to thank Dr. Chen for the significant contributions he has made to the Company, including managing the initial dose escalation portion of our Phase 1 clinical trial of IGM-2323, our CD20 x CD3 T cell engager IgM antibody for non-Hodgkin’s lymphoma, and successfully launching our Phase 1 clinical trial of IGM-8444, our Death Receptor 5 agonist IGM antibody for solid and liquid tumors, and we sincerely wish him continued success in his career."

"As the pioneers of a groundbreaking new technology, IGM Biosciences has the potential to make a real difference for patients in therapeutic areas from oncology to infectious diseases to immunology and inflammation by harnessing the power of nature’s strongest antibodies, IgMs," said Dr. Takimoto. "IGM Biosciences has solved many of the challenges historically associated with engineering and manufacturing these complex molecules, and I look forward to participating in their clinical development and helping to demonstrate their full potential."

Prior to Gilead, Dr. Takimoto served as Chief Medical Officer, since February 2016, of Forty Seven, Inc., a biotechnology company formed out of Stanford University and acquired by Gilead Sciences in 2020. From September 2010 to January 2016, Dr. Takimoto served as Vice President of Experimental Medicine Early Development, Oncology Therapeutic Area for Janssen Research and Development, LLC. From 2008 to 2010, Dr. Takimoto served as Senior Director of Translational Medicine of Ortho Biotech Oncology Research and Development. He has over thirty years of experience in industry and academia, including academic positions at the University of Texas Health Science Center at San Antonio, the National Cancer Institute, and the Uniformed Services University of the Health Sciences. He also served as a Commissioned Officer in the U.S. Public Health Service. Dr. Takimoto received a B.S. in Chemistry from Stanford University, a Ph.D. in Pharmacology from Yale University, and an M.D. from Yale University School of Medicine.

On July 29, 2021 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, CRISPR Therapeutics, JUL 29, 2021, View Source [SID1234585374]).

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"We concluded an important quarter in which we reported notable data from our hemoglobinopathies program while rapidly advancing our entire clinical and pre-clinical portfolio and our capabilities," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Updated clinical data on CTX001 presented at EHA (Free EHA Whitepaper) demonstrate consistency and durability, further validating the promise of a functional cure for sickle cell disease and beta thalassemia. We expect to report clinical data from our immuno-oncology programs later this year, and to file multiple INDs for our regenerative medicine and in vivo programs in the next 18 to 24 months. In addition, we continue to expand our portfolio and access best-in-class capabilities through collaborations, such as those recently announced with Capsida Biotherapeutics and Nkarta Therapeutics."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

In April, CRISPR Therapeutics and Vertex announced an amendment to their collaboration for CTX001. In connection with the completion of the transaction in June, Vertex made a $900 million upfront payment to CRISPR Therapeutics.

Data from 22 patients with at least three months of follow-up after CTX001 infusion were presented at the Annual European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress (EHA) (Free EHA Whitepaper) in June and continued to build the profile of a functional cure for patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), showing consistent and durable benefit with longer term data from a larger population of patients.

Enrollment and dosing are ongoing in the clinical studies for CTX001 and more than 45 patients have been dosed across the programs to date. The companies anticipate achieving target enrollment in both studies in the third quarter of 2021, with regulatory filings possible in the next 18 to 24 months.
Immuno-Oncology

The Company expects to report additional clinical data in 2021 from its ongoing Phase 1 CARBON trial assessing the safety and efficacy of several dose levels of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR-T) investigational therapy targeting CD19, for the treatment of relapsed or refractory B-cell malignancies.

CRISPR Therapeutics’ Phase 1 clinical trial assessing the safety and efficacy of several dose levels of CTX120, its wholly-owned allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma, is ongoing. The Company expects to report top-line data from this trial in 2021.

CRISPR Therapeutics received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70, for the treatment of T-cell lymphoma. CRISPR Therapeutics’ two independent Phase 1 clinical trials assessing the safety and efficacy of several dose levels of CTX130, for the treatment of both solid tumors and certain hematologic malignancies, are ongoing. The Company expects to report top-line data from these trials in 2021.

In May, CRISPR Therapeutics and Nkarta Therapeutics announced a research and development collaboration to co-develop and co-commercialize two chimeric antigen receptor (CAR) NK cell product candidates, one targeting CD70, and a product candidate combining NK and T cells (NK+T), each enhanced with genome engineering.

Regenerative Medicine and In Vivo Programs:

CRISPR Therapeutics and its partner ViaCyte remain on track to initiate a Phase 1/2 trial of their allogeneic stem cell-derived therapy for the treatment of Type 1 diabetes in 2021. The combination of ViaCyte’s stem cell capabilities and CRISPR’s gene editing capabilities has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring immune suppression.

The Company continues to make progress with its in vivo approaches for liver gene editing. Additionally, earlier this month, Nature Communications published an independently peer-reviewed article entitled "Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases." The publication includes information on the Company’s development of proprietary small Cas9 variants which may allow for more efficient delivery in vivo using viral delivery vehicles. The Company expects to move multiple programs utilizing in vivo approaches into the clinic in the next 18 to 24 months.

In June, CRISPR Therapeutics and Capsida Biotherapeutics announced a strategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.
Other Corporate Matters

In June, CRISPR Therapeutics announced the election of H. Edward Fleming Jr., M.D. to its Board of Directors. Dr. Fleming is a Senior Partner at McKinsey and Company and the global leader of McKinsey’s R&D practice.
Second Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $2,589.4 million as of June 30, 2021, compared to $1,806.2 million as of March 31, 2021. The increase in cash of $783.2 million was primarily driven by an upfront payment of $900.0 million in connection with the Amended and Restated Joint Development and Commercialization Agreement with Vertex, offset by continuing operating expenses.

Revenue: Total collaboration revenue was $900.2 million for the second quarter of 2021, compared to less than $0.1 million for the second quarter of 2020. Collaboration revenue primarily consisted of the $900.0 million upfront payment from Vertex, as well as charges to partners for research activities.

R&D Expenses: R&D expenses were $108.3 million for the second quarter of 2021, compared to $59.4 million for the second quarter of 2020. The increase in expense was driven by development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs, as well as increased headcount and supporting facilities related expenses.

G&A Expenses: General and administrative expenses were $29.8 million for the second quarter of 2021, compared to $21.4 million for the second quarter of 2020. The increase in general and administrative expenses for the year was primarily driven by headcount-related expense.

Net Income (loss): Net income was $759.2 million for the second quarter of 2021, compared to a net loss of $79.7 million for the second quarter of 2020.
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex Collaboration
Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBON
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120
CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

On July 29, 2021 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that it will host a conference call and webcast to report its second quarter financial results on Thursday, August 5, 2021, at 8:30 a.m. ET (Press release, GlycoMimetics, JUL 29, 2021, View Source [SID1234585373]).

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The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 9977599. Participants are encouraged to connect 15 minutes in advance of the call to ensure they are able to connect. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, with participant code 9977599.