On March 11, 2021 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported its fourth quarter and year end 2020 financial results and corporate highlights (Press release, Kezar Life Sciences, MAR 11, 2021, View Source [SID1234576488]).

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"Despite unprecedented circumstances, 2020 was a year of significant corporate and clinical accomplishments, thanks to the excellent execution by the Kezar team," said John Fowler, Kezar’s Co-Founder and Chief Executive Officer. "We advanced our lead product candidate, KZR-616, in multiple indications, and the early clinical data from MISSION strongly support its continued development for a wide range of severe immune-mediated diseases. We launched the open-label extension study for patients completing PRESIDIO, and KZR-616 was granted Orphan Drug Designations from the FDA for the treatment of polymyositis and dermatomyositis. This year we look forward to reporting the final results of the MISSION Phase 1b study and interim data from the MISSION Phase 2 study in systemic lupus erythematosus and lupus nephritis patients, respectively."

Fowler continued, "Additionally, we continue to be highly encouraged by the therapeutic potential of KZR-261 and look forward to initiating a Phase 1 trial in solid tumors later this year. The data we’ll be presenting from the protein secretion program in two posters during AACR (Free AACR Whitepaper) next month provides further support for inhibiting Sec61 as a target to treat various cancers."

Clinical Highlights & Updates

KZR-616: Selective Immunoproteasome Inhibitor

MISSION – Phase 1b/2 clinical trial in patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN), respectively (NCT03393013)

The Phase 2 open-label portion of the MISSION trial in patients with active, proliferative lupus nephritis opened for enrollment in August 2020 and is actively recruiting. The primary efficacy endpoint for the trial is the proportion of patients achieving a renal response measured by a 50% or greater reduction in urine protein to creatinine ratio (UPCR) at six months.
Interim data are expected in late 2021, and topline data are expected in the first half of 2022, subject to developments in the ongoing COVID-19 pandemic.
The MISSION Phase 1b 25-week safety and tolerability study of up to 75 mg weekly of KZR-616 in 47 patients with SLE completed enrollment in the fourth quarter of 2020.
Final data from the MISSION Phase 1b study are expected to be available in mid-2021.
In November 2020, additional data from this study were presented at the American College of Rheumatology Annual Meeting (ACR Convergence 2020). Positive early efficacy and biomarker data suggest that selective inhibition of the immunoproteasome with KZR-616 could have a meaningful clinical impact in patients with severe autoimmune diseases. No new safety signals were observed, and KZR-616 administered subcutaneously once weekly has been consistently well tolerated for up to 13 weeks.
PRESIDIO – Phase 2 clinical trial in patients with dermatomyositis (DM) and polymyositis (PM) (NCT04033926)

The PRESIDIO Phase 2, placebo controlled cross-over trial of KZR-616 in DM and PM is actively enrolling. Additionally, a 12-month open-label extension study is enrolling patients completing the 32-week placebo-controlled trial (NCT04628936).
Topline data are expected in the first half of 2022, subject to developments in the ongoing COVID-19 pandemic.
KZR-616 was granted Orphan Drug Designations (ODD) by the U.S. Food and Drug Administration in October 2020 for both DM and PM, the only investigative drug to have ODD in both indications. The estimated prevalence of DM and PM in the United States is up to 71,000 and 51,000, respectively.
During ACR Convergence 2020, preclinical results for KZR-616 were presented in CIM, the mouse model of polymyositis. The results demonstrated that treatment with KZR-616 was associated with significant improvement in muscle function and reduced levels of muscle tissue damage, providing a rationale for targeting selective immunoproteasome inhibition for the treatment of polymyositis.
Protein Secretion Program

KZR-261 is a first-in-class protein secretion inhibitor which targets the Sec61 translocon and has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies.
Submission of an Investigational New Drug (IND) application for KZR-261 is anticipated in mid-2021. The Phase 1 clinical trial will evaluate dose escalation and safety and tolerability in patients with solid tumors and is expected to commence shortly after the IND becomes effective.
Two abstracts featuring Kezar’s small molecule inhibitors of the Sec61 translocon have been selected for presentation at the upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Virtual Annual Meeting, taking place April 10-15, 2021 and May 17-21, 2021. Details for the two AACR (Free AACR Whitepaper) presentations are as follows:
Poster Presentations

Title: Prioritizing tumor types for clinical study of novel Sec61 inhibitors by searching for expression profiles of sensitive cell lines in tumor sample databases

Presenter/s: Eric Lowe, R. Andrea Fan, Henry W. B. Johnson, Christopher J. Kirk, Dustin McMinn, Yu Qian, Brian Tuch

Session: Genomic Profiling of Tumors – Abstract #2226

Date and time: Available on demand [8:30AM ET, Saturday, April 10, 2021]

Title: Quantitative proteomic profiling of novel anti-cancer small molecule inhibitors of Sec61: Mechanistic investigation and biomarker discovery

Presenter/s: Yu Qian, Jennifer Whang, Janet Anderl, Andrea Fan, Henry W. B. Johnson, Christopher J. Kirk, Eric Lowe, Dustin McMinn, Beatriz Millare, Tony Muchamuel and Jinhai Wang; Kezar Life Sciences

Session: Proteomics and Biomarker Discovery – Abstract #2816

Date and time: Available on demand [8:30AM ET, Saturday, April 10, 2021]

Additional preclinical data further detailing the ability of novel small molecule Sec61 inhibitors to target multiple checkpoint proteins on various cell populations, thereby offering the potential of combination therapy in a single compound, were presented during the 8th Annual Meeting of the International Cytokine & Interferon Society (Cytokines 2020) and the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in November 2020.
Corporate Update

Kezar was added to the NASDAQ Biotechnology Index (NASDAQ: NBI) on December 21, 2020.
Financial Results

Cash, cash equivalents and marketable securities totaled $140.4 million as of December 31, 2020, compared to $78.2 million as of December 31, 2019. The increase in cash, cash equivalents and marketable securities was primarily attributable to the net proceeds from the underwritten public offerings, net of cash used by the company in operations to advance its clinical stage programs and preclinical research and development.
Research and development expenses for the fourth quarter of 2020 increased by $0.7 million to $8.1 million compared to $7.4 million in the fourth quarter of 2019. Full year R&D expenses increased by $3.6 million in 2020 compared to 2019. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.
General and administrative expenses for the fourth quarter of 2020 increased by $0.4 million to $3.0 million compared to $2.6 million in the fourth quarter of 2019. Full year G&A expenses increased by $1.9 million in 2020 compared to 2019. The increase was primarily due to an increase in personnel expenses, professional fees and costs of directors and officers liability insurance.
Net loss for the fourth quarter of 2020 was $10.9 million, or $0.22 per basic and diluted common share, compared to a net loss of $9.6 million, or $0.50 per basic and diluted common share, for the fourth quarter of 2019. Net loss for 2020 was $41.7 million, or $0.95 per basic and diluted common share, compared to $35.1 million, or $1.84 per basic and diluted common share, in 2019.
Total shares of common stock outstanding were 46.4 million as of December 31, 2020. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 4.5 million shares of common stock at a weighted average exercise price of $6.14 per share as of December 31, 2020.
About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b clinical trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathways. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered by Kezar through a robust medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. An IND submission in solid tumors is expected to be filed in mid-2021.

About Lupus Nephritis

Lupus nephritis (LN) is one of the most serious complications of systemic lupus erythematosus (SLE). LN is a disease comprising a spectrum of vascular, glomerular, and tubulointerstitial lesions and develops in about 50% of SLE patients within 10 years of their initial diagnosis. LN is associated with considerable morbidity, including an increased risk of end-stage renal disease requiring dialysis or renal transplantation and an increased risk of death. There are limited approved therapies for the treatment of LN. Management typically consists of induction therapy to achieve remission and long-term maintenance therapy to prevent relapse.

About Dermatomyositis and Polymyositis

Polymyositis and Dermatomyositis are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). An approximate 30,000-120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

On March 11, 2021 Champions Oncology, Inc. (Nasdaq: CSBR), a leading global oncology technology solutions provider engaged in transforming drug discovery and development through data-driven research strategies and innovative pharmacology, biomarker and data platforms, reported its financial results for the third fiscal quarter ended January 31, 2021 (Press release, Champions Oncology, MAR 11, 2021, View Source [SID1234576487]).

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Third Quarter and Recent Highlights:

•Delivered record quarterly revenue of $10.8 million, an increase of 20% year over year
•Reported non-GAAP income from operations, excluding stock-based compensation, depreciation and amortization, of $1.3 million
•Expanded Lumin Bioinformatics proprietary SaaS platform with proteomic module integration

Ronnie Morris, CEO of Champions, commented, "We are pleased with our third quarter results and we continue to execute our long term strategy of investing in the development of new products and services to increase our revenue streams."

David Miller, CFO of Champions, added, "We delivered another record quarter with revenue increasing to $10.8 million. In addition to the growth in our product and service offerings, we’re bringing the outsourced ex-vivo work in-house to our labs which we expect will impact margins positively in the future."

Third Fiscal Quarter Financial Results

For the third quarter of fiscal 2021, revenue increased 20.0% to $10.8 million compared to $9.0 million for the third quarter of fiscal 2020. The increase in revenue was due to a continued increase in sales,

Exhibit 99.1
both in number and size of studies, and the expansion of both our platform and product lines. Our contract amounts have increased as we perform more complex studies and end point analysis testing. Total costs and operating expenses for the third quarter of fiscal 2021 were $10.0 million compared to $8.6 million for the third quarter of fiscal 2020, an increase of $1.5 million or 17.1%.

For the third quarter of fiscal 2021, Champions reported income from operations of $763,000, including $232,000 in stock-based compensation and $297,000 in depreciation and amortization expenses, an increase of $330,000 compared to income from operations of $433,000, inclusive of $229,000 in stock-based compensation and $219,000 in depreciation and amortization expenses, in the third quarter of fiscal 2020. Excluding stock-based compensation, depreciation and amortization expenses, Champions reported non-GAAP income from operations of $1.3 million for the third quarter of fiscal 2021 compared to non-GAAP income from operations of $881,000 in the third quarter of fiscal 2020, an increase of $411,000.
Cost of oncology solutions was $4.8 million for the three-months ended January 31, 2021, an increase of $0.5 million, or 12.0% compared to $4.3 million for the three-months ended January 31, 2020. For the three- months ended January 31, 2021, gross margin was 55.2% compared to 52.0% for the three-months ended January 31, 2020. The increase in cost of oncology services for the three-month period was mainly due to an increase in compensation and lab supply expenses. The increase in cost of sales is generally in line with the expected contribution based on the growth in revenue, study volume, and expansion into new services. Gross margin varies based on timing differences between expense and revenue recognition and was boosted by the revenue recognized from outsourced work where costs were partially recognized in prior quarters.
Research and development expense for the three-months ended January 31, 2021 was $1.9 million, an increase of $488,000 or 35.1%, compared to $1.4 million for the three-months ended January 31, 2020, respectively. The increase was due to increased compensation and lab supply expense as we continued to develop new service capabilities and endpoint analysis testing. Additionally, we incurred proteomic sequencing costs as our investment in characterizing our TumorBank continued, adding valuable data to our platform. Sales and marketing expense for the three-months ended January 31, 2021 was $1.5 million, an increase of $185,000, or 14.2%, compared to $1.3 million for the three-months ended January 31, 2020. The increase was primarily due to compensation expense driven by the continued investment in expanding our business development team. General and administrative expense for the three-months ended January 31, 2021 was $1.8 million, an increase of $280,000, or 18.0%, compared to $1.6 million for the three-months ended January 31, 2020, respectively. The increase was primarily due to compensation and IT related expenses.
Net cash used in operating activities was $107,000 for the three-months ended January 31, 2021 compared to net cash provided by operating activities of $280,000 for the same period last year. The decrease in cash flow from operations was primarily due to a significant reduction in our accounts payable balance along with timing differences in other working capital accounts in the ordinary course of business.

The Company ended the quarter in a strong cash position with a $7.4 million cash balance compared to $3.3 million at the end of the same period last year. The Company has no debt.

Year-to-Date Financial Results

For the first nine months of fiscal 2021, revenue increased 30.4% to $30.5 million, as compared to $23.4 million for the first nine months of fiscal 2020. For the first nine months of fiscal 2021, total

Exhibit 99.1
operating expenses increased 27.6% to $29.7 million, as compared to $23.3 million for the first nine months of fiscal 2020. The increase in revenue was due to increased sales, both in number and size of studies, an increase in demand for our services, the growth of the platform, and the expansion of our product line. Our customers are seeking more complex study designs and end point analysis testing, contributing to the larger contract sizes.

For the first nine months of fiscal 2021, Champions reported income from operations of $795,000, which includes $437,000 in stock-based compensation and $881,000 in depreciation and amortization expenses, an increase of $686,000 or 629%, compared to income from operations of $109,000, inclusive of $437,000 in stock-based compensation and $579,000 depreciation, for the first nine months of fiscal 2020. Excluding stock-based compensation and depreciation, Champions reported non-GAAP operating income of $2.1 million for the first nine months of fiscal 2021 compared to non-GAAP operating income of $1.1 million in the same period last year.

Cost of oncology solutions was $15.8 million for the first nine months of fiscal 2021 compared to $12.0 million for the first nine months of fiscal 2020, an increase of $3.9 million or 32.3%. Gross margin was 48.1% for the first nine months of fiscal 2021 compared to 48.8% for the first nine months of fiscal 2020. The increase in cost of oncology services for the nine month period was mainly due to an increase in compensation, lab supply, and outsourced lab service expenses. Gross margin varies based on timing differences between expense and revenue recognition and was driven lower by the increase in costs on growing study volume in advance of revenue recognition. The cost of outsourced lab services amplified this impact.

Research and development expense was $5.1 million for the first nine months of fiscal 2021 an increase of $1.1 million, or 27.0% compared to $4.0 million for the first nine months of fiscal 2020. The increase was due to increased compensation and lab supply expense as we continued to develop new service capabilities and endpoint testing analysis, and incurred sequencing costs as we continued to characterize our TumorBank. Sales and marketing expense for the first nine months of fiscal 2021 was $4.0 million, an increase of $893,000, or 28.3% compared to $3.2 million for the first nine months of fiscal 2020. The increase was primarily due to compensation expense driven by the continued investment in expanding our sales force. General and administrative expense was $4.7 million for the first nine months of fiscal 2021, an increase of $568,000, or 13.8% compared to $4.1 million for the first nine months of fiscal 2020. General and administrative expenses are primarily comprised of compensation, insurance, accounting fees, and depreciation expenses and have increased to support the overall infrastructure growth of the company.

Net cash provided by operations was $299,000 for the first nine months of fiscal 2021 compared to net cash provided by operations of $360,000 in fiscal 2020, a decrease of $61,000 or 16.9%. The decrease in cash from operations was primarily due to timing differences in working capital accounts in the ordinary course of business.

Conference Call Information:
The Company will host a conference call today at 4:30 p.m. EST (1:30 p.m. PST) to discuss its third quarter financial results. To participate in the call, please call 877-407-8035 (domestic) or 201-689-8035 (international) ten minutes ahead of the call and give the verbal reference "Champions Oncology."
Full details of the Company’s financial results will be available by Wednesday, March 17, 2021 in the Company’s Form 10-Q at www.championsoncology.com.

On March 11, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today provided a business update and reported fourth quarter and full year 2020 financial results (Press release, Oncternal Therapeutics, MAR 11, 2021, View Source [SID1234576486]).

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"In 2021, we are advancing a deep pipeline of differentiated oncology assets. We have now initiated early-stage work in ROR1-targeting immunotherapies, including CAR-T and CAR-NK cell therapies, while moving forward the later-stage clinical development of cirmtuzumab, an antibody targeting ROR1, in MCL, which has generated encouraging data in this difficult to treat cancer. At the same time, we continue to evaluate TK216, an ETS inhibitor which has generated promising results in Ewing sarcoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We have also strengthened our balance sheet by raising $125 million in 2020, which provides us with the runway to advance our promising programs into 2023. We have several key data read-outs pending in the second quarter this year."

Recent Highlights

In January 2021, we announced an agreement with Lentigen Technology, Inc., a wholly-owned subsidiary of Miltenyi Biotec B.V. & Co. KG, to manufacture lentiviral vectors to support Oncternal’s investigational ROR1-targeting CAR-T cell therapy program.
In January 2021, we announced a research and development collaboration with Karolinska Institutet in Stockholm, Sweden, to advance novel ROR1-targeting CAR-T and CAR-NK cell therapies from the laboratory into the clinic.
In December 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial of cirmtuzumab, an investigational anti-ROR1 monoclonal antibody, in combination with ibrutinib in patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Virtual Annual Meeting. Best objective response rate of 87%, including complete response (CR) rate of 47%, was reported for 15 evaluable patients with relapsed/refractory (r/r) MCL. Median progression-free survival (PFS) was not reached for patients with MCL, with the 95% confidence interval above 17.5 months, after a median follow-up of 12.1 months. The median PFS was not reached for patients with treatment-naïve CLL (n=19) after a median follow-up of 16.6 months, and median PFS was 29.5 months for patients with r/r CLL (n=30) after a median follow-up of 17.1 months. The combination of cirmtuzumab and ibrutinib was well tolerated in this trial.
In November 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial evaluating TK216, an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with r/r Ewing sarcoma at the Connective Tissue Oncology Society 2020 Virtual Annual Meeting. The reported disease control rate (CR, partial response or stable disease) was 43%, including two patients with durable complete responses that were ongoing at over 1.5 years and 8 months on treatment.
In October 2020, we announced that the U.S. Food and Drug Administration granted Rare Pediatric Disease designation for TK216 for the treatment of Ewing sarcoma.
In November and December 2020, we raised an aggregate of approximately $109 million in gross proceeds from two underwritten offerings.
Expected Upcoming Milestones

Cirmtuzumab (ROR1 antibody) programs
Clinical data update for patients with MCL and CLL treated with cirmtuzumab plus ibrutinib in the ongoing Phase 1/2 study in Q2 2021
Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study in Q2 2021
Preclinical data in additional ROR1 expressing tumors in Q2 2021
ROR1 CAR-T program
First-in-human dosing in China in the second half of 2021
TK216 (ETS inhibitor) program
Clinical data for patients with Ewing sarcoma treated in the ongoing Phase 1/2 expansion cohort in Q2 2021
Preclinical data in additional ETS-driven tumors in Q2 2021
Fourth Quarter and Full Year 2020 Financial Results
Our grant revenue was $1.6 million for the fourth quarter ended December 31, 2020. Our grant revenue is derived from a subaward under a grant from the California Institute for Regenerative Medicine (CIRM) to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL. For the full year 2020, grant revenue was $3.4 million.

Our total operating expenses for the fourth quarter ended December 31, 2020 were $4.4 million. Research and development expenses for the quarter totaled $3.0 million, and general and administrative expenses for the quarter totaled $1.5 million. Net loss for the fourth quarter was $2.6 million, or a loss of $0.09 per share, basic and diluted. For the full year 2020, total operating expenses were $20.9 million. Net loss for the full year 2020 was $17.2 million, or a loss of $0.85 per share, basic and diluted.

As of December 31, 2020, we had $116.7 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into 2023. As of December 31, 2020, we had approximately 48.8 million shares of common stock outstanding.

Management Webcast
As previously announced, Oncternal will host a webcast today, March 11, 2020, at 5:00 p.m. ET. The live webcast will be available online and may be accessed from the "Investors" page of the company website at View Source A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

On March 11, 2021 Amgen (NASDAQ:AMGN) reported that it will present at the virtual Oppenheimer 31st Annual Healthcare Conference at 2:30 p.m. ET on Tuesday, March 16, 2021 (Press release, Amgen, MAR 11, 2021, View Source [SID1234576485]). David M. Reese, M.D., executive vice president of Research and Development at Amgen will present at the conference. Live audio of the conference call will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public.

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The webcast, as with other selected presentations regarding developments in Amgen’s business given at certain investor and medical conferences, can be accessed on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.

On March 11, 2021 PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapies for cardiopulmonary diseases, and BioVectra Inc., an innovative global contract development and manufacturing organization (CDMO), reported a commercial scale supply agreement for the production of bentracimab, PhaseBio’s lead product candidate currently in a global Phase 3 clinical trial (Press release, PhaseBio Pharmaceuticals, MAR 11, 2021, View Source [SID1234576484]). Bentracimab is a novel, human monoclonal antibody fragment that in earlier clinical trials has shown immediate and sustained reversal of the antiplatelet effects of Brilinta (ticagrelor).

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Under the terms of the agreement, BioVectra will provide its integrated CDMO services for the manufacturing of the active pharmaceutical ingredient (API) of bentracimab for use in PhaseBio’s ongoing global clinical trials and for global commercial use upon regulatory approval. Utilizing a cost-efficient, E.coli-based manufacturing process at its manufacturing site in Windsor, Nova Scotia, BioVectra recently completed the first GMP run of bentracimab at commercial scale. Going forward, PhaseBio plans to integrate API manufactured at commercial scale at BioVectra into the ongoing Phase 2b and Phase 3 REVERSE-IT clinical trials to support global regulatory filings.

"As we prepare for the next phase of growth for bentracimab, we’re pleased to sign this agreement with BioVectra for the commercial supply and development of our novel reversal agent," said Jonathan P. Mow, Chief Executive Officer of PhaseBio Pharmaceuticals. "Having recently expanded our global Phase 3 REVERSE-IT trial into Canada and the European Union, and with other countries on the horizon, the signing of this agreement and the completion of our first commercial-scale manufacturing run are important steps as we continue preparing our regulatory filings and commercialization efforts for bentracimab. We believe BioVectra will be an excellent partner as we move down the path of developing and potentially commercializing the first specific antiplatelet reversal agent for ticagrelor."

Pending approval of the drug by regulators, the partnership will enable PhaseBio to supply bentracimab at launch in the U.S. to key trauma and critical care centers. Based on data from IQVIA, PhaseBio believes that providing adequate bentracimab supply for initial stocking and demand-based reorders for these key centers will make bentracimab accessible to approximately 80% of the population in the U.S. who are prescribed P2Y12 inhibitors like ticagrelor.

"We’re excited to be partnering with PhaseBio under this new commercial agreement for the high-volume scale-up and global supply of bentracimab," said Oliver Technow, Chief Executive Officer of BioVectra. "BioVectra is an expert CDMO with proven, specialized capability in scaling the most complex biologic drug substances produced from fermentation. Leveraging our fully integrated approach and 50 years of experience, our highly-skilled team looks forward to continuing to make a difference in patients’ lives by delivering on and supporting PhaseBio’s global commercialization of bentracimab from our new, large-scale Microbial Biomanufacturing facility."

About Bentracimab (PB2452)

Bentracimab is a novel, recombinant, human monoclonal antibody antigen-binding fragment designed to reverse the antiplatelet activity of ticagrelor in major bleeding and urgent surgery situations. In a Phase 1 clinical trial, bentracimab demonstrated the potential to bring life-saving therapeutic benefit through immediate and sustained reversal of ticagrelor’s antiplatelet activity, mitigating concerns regarding bleeding risks associated with the use of antiplatelet drugs. The Phase 1 clinical trial of bentracimab in healthy volunteers was published in the New England Journal of Medicine in March 2019. In April 2019, bentracimab received Breakthrough Therapy Designation from the FDA. Breakthrough Therapy Designation may be granted by the FDA when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapy. In September 2019, PhaseBio completed a Phase 2a trial in which bentracimab was investigated in older and elderly subjects on dual antiplatelet therapy of ticagrelor and low-dose aspirin. Additionally, the Phase 2a trial investigated a bentracimab regimen for the reversal of supratherapeutic doses of ticagrelor in healthy younger subjects. In both arms of the trial, bentracimab achieved immediate and sustained reversal of the antiplatelet effects of ticagrelor and was generally well-tolerated, with only minor adverse events reported. These results are consistent with the results observed in healthy younger subjects treated with ticagrelor in the previously published Phase 1 trial. PhaseBio initiated the REVERSE-IT trial, a pivotal Phase 3 clinical trial of bentracimab, in March 2020 to support a Biologics License Application for bentracimab in both major bleeding and urgent surgery indications. There are currently no approved reversal agents for ticagrelor or any other antiplatelet drugs.