Intellia Therapeutics Announces First Patient Dosed in Phase 1/2a Clinical Trial of NTLA-5001 for the Treatment of Acute Myeloid Leukemia

On March 1, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, reported that the first patient has been dosed with NTLA-5001, the company’s ex vivo CRISPR/Cas9 genome editing candidate for the treatment of acute myeloid leukemia (AML) (Press release, Intellia Therapeutics, MAR 1, 2022, View Source [SID1234609321]). NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is found in AML and many other hematologic and solid tumors. It is the company’s first ex vivo candidate developed using Intellia’s advanced lipid nanoparticle cell engineering platform, designed to improve cell performance as compared to traditional ex vivo delivery technologies.

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"As Intellia’s first wholly-owned ex vivo candidate to be dosed in a patient, this NTLA-5001 milestone represents a significant step forward in our full-spectrum approach to genome editing," said Intellia President and Chief Executive Officer John Leonard, M.D. "AML is the most common type of acute leukemia in adults, where despite recent advancements, a significant therapeutic need still exists. We look forward to advancing this investigational engineered cell therapy as a treatment for people living with this aggressive cancer of the blood and bone marrow."

About the NTLA-5001 Clinical Program

The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Visit clinicaltrials.gov (NCT05066165) for more details.

About NTLA-5001

NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults in the U.S., with more than 20,000 estimated new cases in 2021. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.

Bicycle Therapeutics Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Corporate Update

On March 1, 2022 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the fourth quarter and full year ended December 31, 2021, and provided recent corporate updates (Press release, Bicycle Therapeutics, MAR 1, 2022, View Source [SID1234609228]).

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"We are encouraged by the progress we continue to make across our oncology pipeline," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "As we previously reported, BT8009 and BT5528 have demonstrated anti-tumor activity in two tumor types, and we look forward to presenting interim results from our Phase I trial of BT8009 at a medical meeting and initiating the expansion cohorts in our clinical trial of BT5528 this year. We also are moving our Bicycle TICA programs forward, including BT7480, which entered the clinic in the fourth quarter of 2021. Our plans to advance our clinical programs, as well as to expand our discovery efforts and early development pipeline, are on track and are supported by a strong balance sheet, with sufficient cash to fund these initiatives and support operations through 2024."

Fourth Quarter 2021 and Recent Highlights

Announced Continued Progress in Ongoing Phase I/II Clinical Trials of BT8009, BT5528 and BT7480. In January 2022, Bicycle announced progress updates for its wholly owned oncology pipeline, including BTCs BT8009 and BT5528 and Bicycle TICA BT7480. In the ongoing Phase I portion of the Phase I/II clinical trial of BT8009, a second-generation BTC targeting Nectin-4, four out of 11 patients were previously reported to have a partial response under Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, including one out of four (25%) in the 2.5mg/m2 dose and three out of seven (43%) at the 5.0 mg/m2 cohorts. All four patients previously reported as responders have since received at least one subsequent scan, and all have been confirmed as ongoing RECIST 1.1 responses. One patient in the 5.0 mg/m2 cohort, who previously was reported to have a partial response with an 89% tumor reduction, has now received two subsequent scans, which each showed that total tumor volume has been reduced by 100%, constituting a confirmed complete response.

The Phase I/II trial of BT5528, Bicycle’s second-generation BTC targeting EphA2, is also ongoing, with initiation of expansion cohorts planned for this year, with an expected recommended Phase II dose of 6.5mg/m2 every-other-week.

Bicycle also initiated a Phase I clinical trial of BT7480, a novel, fully synthetic Bicycle TICA targeting Nectin-4 and agonizing CD137, in the fourth quarter of 2021, and dose escalation in that trial remains ongoing.
Announced Expansion of and Transition in Management Team. In January 2022, Michael Skynner, Ph.D., previously the company’s Chief Operating Officer (COO), was appointed to the newly created position of Chief Technology Officer, to focus on leading and overseeing the growth of Bicycle’s proprietary phage display discovery platform in oncology, as well as on creating innovative opportunities for the platform outside of oncology. Dr. Skynner joined the company in January 2016 as Vice President, Operations and Discovery and had served as COO since March 2018. Alistair Milnes, who served as the company’s Vice President, Human Resources and Communications since January 2021, has assumed the COO role. Mr. Milnes previously led human resources and communications at multinational energy and mineral companies. Both Dr. Skynner and Mr. Milnes are based in the United Kingdom.
Presented Preclinical BT7480 and BT7455 Data at the SITC (Free SITC Whitepaper) 36th Annual Meeting. In November 2021, Bicycle presented preclinical data for BT7480 and BT7455, an EphA2/CD137 Bicycle TICA. Results supported Bicycle’s decision to initiate a Phase I/II clinical trial of BT7480 and its prioritization of potential indications to target. Additionally, Bicycle has developed a pharmacokinetic/pharmacodynamic (PK/PD) modelling framework intended to predict preclinical biomarker level and tumor growth inhibition in response to changes in the BT7480 dose and dosing schedule. Bicycle found that plasma and tumor drug concentration levels may be associated with tumor growth inhibition.
Financial Results

Cash was $438.7 million as of December 31, 2021, compared to $136.0 million as of December 31, 2020. The net increase in cash is primarily due to financing activities during 2021, including net proceeds of $188.4 million from a public offering of Bicycle’s ADSs, net proceeds of $102.6 million from Bicycle’s at-the-market (ATM) offering program and net proceeds of $15.0 million from Bicycle’s debt facility with Hercules Capital, as well as $45.0 million received from Ionis Pharmaceuticals under the 2021 collaboration and license agreement, and $10.0 million received from Genentech for exercising an option to initiate an additional program under the 2020 collaboration agreement, offset by cash used for operating activities.
Research and development expenses were $13.0 million for the three months ended December 31, 2021 and $44.9 million for the year ended December 31, 2021, compared to $10.1 million for the three months ended December 31, 2020 and $33.1 million for the year ended December 31, 2020. The increase in expense of $2.9 million for the three months ended December 31, 2021 as compared to the same period in the prior year was primarily due to increased Bicycle TICA clinical program spend and other discovery and platform related expenses, as well as increased personnel related expenses, including $0.7 million of incremental non-cash share-based compensation expense. The increase in expense of $11.7 million for the year ended December 31, 2021 as compared to the same period in the prior year was primarily due to increased clinical program expenses for BT8009, Bicycle TICA and other discovery and platform related expenses and increased personnel-related expenses, including $2.4 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $8.8 million for the three months ended December 31, 2021 and $32.4 million for the year ended December 31, 2021, compared to $10.9 million for the three months ended December 31, 2020 and $29.2 million for the year ended December 31, 2020. The decrease of $2.1 million for the three months ended December 31, 2021 as compared to the same period in the prior year was primarily due to the settlement and license agreement with Pepscan Systems B.V. ("Pepscan") entered into in November 2020, offset by increased costs to support operations as a public company and increased personnel-related costs, including $0.7 million of incremental non-cash share-based compensation expense. The increase of $3.2 million for the year ended December 31, 2021 as compared to the same period in the prior year was primarily due to increased personnel-related costs, including $3.2 million of incremental non-cash share-based compensation expense and increased costs to support operations as a public company, offset by a decrease in professional and consulting costs, and the settlement and license agreement with Pepscan entered into in November 2020.
Net loss was $18.0 million, or $(0.63) basic and diluted net loss per share, for the three months ended December 31, 2021, and net loss was $66.8 million, or $(2.67) basic and diluted net loss per share, for the year ended December 31, 2021, compared to net loss of $17.4 million, or $(0.83) basic and diluted net loss per share, for three months ended December 31, 2020, and net loss of $51.0 million, or $(2.66) basic and diluted net loss per share, for the year ended December 31, 2020.

G1 Therapeutics to Participate in the 42nd Annual Cowen Health Care Conference and the 34th Annual Roth 2022 Conference

On March 1, 2022 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported that the Company will participate in two upcoming investor conferences in March 2022 (Press release, G1 Therapeutics, MAR 1, 2022, View Source [SID1234609261]).

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On Tuesday March 8, 2022, G1’s Chief Executive Officer Jack Bailey will participate in a New Drug Launches panel at 2:10PM ET during the virtual 42nd Annual Cowen Health Care Conference.

On Tuesday March 15, 2020, Jack Bailey and Raj Malik, M.D., G1’s Chief Medical Officer, will participate in a fireside chat at 8:00AM PT during the 34th Annual Roth Conference.
The webcast and replay of both events will be accessible on the Events & Presentations page of View Source

Avidity Biosciences Reports Fourth Quarter and Year-End 2021 Financial Results and Recent Highlights

On March 1, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the fourth quarter and year ended December 31, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAR 1, 2022, View Source [SID1234609281]).

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"2021 was a pivotal year for Avidity, the field of RNA therapeutics and for the DM1 community as the first patient was dosed with an AOC as part of the AOC 1001 MARINA trial," said Sarah Boyce, president and chief executive officer. "This milestone transitioned us into a clinical-stage biopharmaceutical company and we continue to make great strides with our advancing and expanding pipeline and AOC platform technology. By the end of 2022, we plan to have three programs in three different rare diseases in the clinic with AOC 1001, AOC 1020 and AOC 1044."

"We are in a strong financial position with $406 million at year-end bolstered through approximately $175 million raised in 2021, inclusive of $155 million of net proceeds from our first follow on financing. We are well funded into 2024 which we expect will allow us to complete the MARINA trial, advance the AOC 1044 and AOC 1020 programs into clinical development and to continue to invest in expanding our pipeline and the AOC platform in muscle and beyond," said Mike MacLean, chief financial officer.

2021 Key Highlights

AOC 1001 Achievements

The Company initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).
The FDA and the European Medicines Agency (EMA) granted AOC 1001 Orphan Designation and the FDA granted AOC 1001 Fast Track Designation.
The MARINA trial is on track for a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the trial participants in Q4 2022.
Pipeline Advancements

AOC 1044 was named as the clinical development candidate for the Duchenne Muscular Dystrophy (DMD) program targeting Exon 44. AOC 1044 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
AOC 1020 was named as the clinical development candidate for the facioscapulohumeral muscular dystrophy (FSHD) program. AOC 1020 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
Avidity also entered a collaboration with the FSHD Clinical Trial Research Network (FSHD CTRN) to support a natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can potentially accelerate and support future clinical trial design.
Organizational Highlights

Avidity recently appointed Steve Hughes, M.D. as chief medical officer. Dr. Hughes brings over 20 years of experience in the biotechnology industry and has extensive experience in RNA-based treatments and rare diseases.
Fourth Quarter and Year-End 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $405.5 million as of December 31, 2021, which reflects $174.7 million raised in 2021, inclusive of $155.1 million of net proceeds from our first follow on financing.

Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $1.9 million for the fourth quarter of 2021 compared with $2.1 million for the fourth quarter of 2020, and $9.3 million for the full year 2021 compared with $6.8 million for the full year 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $33.0 million for the fourth quarter of 2021 compared with $13.6 million for the fourth quarter of 2020, and $101.2 million for the full year 2021 compared with $37.6 million for the full year 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $7.4 million for the fourth quarter of 2021 compared with $4.8 million for the fourth quarter of 2020, and $26.2 million for the full year 2021 compared with $13.5 million for the full year 2020. The increases were primarily due to higher personnel costs. The full year increase was also due to higher professional fees and insurance costs.

BeiGene to Present at the Cowen 42nd Annual Health Care Virtual Conference

On March 1, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, reported that the Company will participate in the Cowen 42nd Annual Health Care Conference on Monday, March 7, 2022 at 9:50 a.m. ET (Press release, BeiGene, MAR 1, 2022, View Source [SID1234609299]).

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A live webcast can be accessed from the investors section of BeiGene’s website at View Source or View Source An archived replay will be available for 90 days following the event.