On April 22, 2021 ArcticZymes Technologies (OSE: AZT) reported sales of NOK 40.4 million (18.5) and an EBITDA of NOK 25.8 million (9.0) for the first quarter of 2021 (Press release, Biotec Pharmacon, APR 22, 2021, View Source [SID1234578334]).

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!

Highlights from Q1 2021

ArcticZymes Technologies (AZT) had Q1 sales of NOK 40.4 million growing by 119% (Q1 2020: NOK 18.5 million)
Coronavirus-related sales are estimated at NOK 16.5 million in Q1 2021 (NOK 3.8 million in Q1 2020)
Gross profit improved to NOK 39.9 million with 99% margin (Q1 2020: NOK 18.5 Million)
AZT delivered a positive EBITDA of NOK 25.8 million (Q1 2020: NOK 9.0 million)
Cash-flow for Q1 was positive NOK 23.2 million (Q1 2020: NOK 4.4 million) giving a cash balance of NOK 163.3 million (Q1 2020: NOK 35.7 million)
Successfully upscaled the SAN HQ production process
CEO Jethro Holter comments:

"We are delighted with the performance of our first quarter as a pure enzymes company. The ArcticZymes team has delivered the best ever quarterly performance achieving sales revenues of 40 million NOK and an EBITDA of 26 million NOK. All market segments have experienced quarterly growth including re-establishment of molecular research sales to pre-pandemic levels.

Furthermore, the SAN HQ enzyme production process was successfully upscaled 100-fold. The innovation pipeline and the infrastructure expansion project of R&D and operations are progressing. Such activities are key to leveraging the greater potential ArcticZymes Technologies can accomplish through organic growth."

On April 22, 2021 Amgen (NASDAQ:AMGN) reported that it will report its first quarter financial results on Tuesday, April 27, 2021, after the close of the U.S. financial markets (Press release, Amgen, APR 22, 2021, View Source [SID1234578352]). The announcement will be followed by a conference call with the investment community at 2:00 p.m. PT. Participating in the call from Amgen will be Robert A. Bradway, chairman and chief executive officer, and other members of Amgen’s senior management team.

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!

Live audio of the conference call will be simultaneously broadcast over the internet and will be available to members of the news media, investors and the general public. The webcast, as with other selected presentations regarding developments in Amgen’s business given by management at certain investor and medical conferences, can be found 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 April 22, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that the first patient has been dosed in an investigator-initiated Phase 2 study to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB)-associated locally advanced/metastatic squamous cell carcinoma (SCC) (Press release, Onconova, APR 22, 2021, View Source [SID1234578368]). The patient was dosed at the EB House Austria, a center of expertise for epidermolysis bullosa at the University Hospital Salzburg, Austria. Additional sites are anticipated to be opened in the UK and in the US to study this rare and genomically driven devastating disease.

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!

In this open-label investigator-initiated study, 12 patients will receive either oral or intravenous rigosertib at the clinician’s discretion given the various clinical manifestations of the disease, which may dictate the need for either oral or intravenous administration of rigosertib. These patients have skin desquamation making intravenous access difficult, or may form esophageal strictures, which make oral administration difficult. Patients will receive either oral rigosertib in four-week cycles (three weeks on, one week off) for up to 13 cycles, with 560 mg of oral rigosertib in the morning and again in the afternoon, for a total of 1,120 mg/day. Alternatively, patients will receive intravenous (IV) rigosertib as a 72-hour IV infusion on days 1, 2 and 3 of eight 2-week cycles, and on days 1, 2 and 3 of nine 4-week cycles thereafter, with each 24-hour infusion consisting of 1,800 mg of rigosertib.

The study has two primary endpoints. The first is to determine the anti-tumor activity of rigosertib in RDEB patients with advanced SCC who have failed prior standard of care through the overall response rate (ORR), defined as the proportion of patients who achieve either a complete response (CR) or a partial response (PR). The second primary endpoint is to evaluate the safety and tolerability of rigosertib in this population. Secondary study endpoints include quality of life and a biomarker analysis performed on archival tissue from all patients. Patients will be dosed for up to one year, with trial duration anticipated to be approximately two-and-a-half years.

"We are pleased with the advancement of our investigator-initiated programs with rigosertib, and to provide rigosertib in support of this important Phase 2 investigator-sponsored study," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova Therapeutics. "Recessive dystrophic epidermolysis bullosa is a genetic skin blistering disease that often results in squamous cell carcinoma in severe subtypes. In this patient cohort, squamous cell carcinoma is the leading cause of death. We have previously identified polo-like kinase 1 as a therapeutic target in skin SCC, including RDEB SCC, so we are encouraged by the start of this trial. We hope rigosertib can prove beneficial to this rare patient population with a tremendous unmet medical need."

In addition to Onconova Therapeutics, the study is being supported by DEBRA International. "The aggressive course and poor prognosis of skin cancer in our patients emphasize the urgent need for potent therapies," stated Professor Johann W. Bauer, M.D., Principal investigator of the trial. "We hope that rigosertib as an innovative approach provides benefit to this devastating illness that currently lacks effective therapies."

"Basic research has provided understanding into the etiology of Recessive Dystrophic Epidermolysis Bullosa-associated cancer," stated Andrew South Ph.D., Associate Professor, Department of Dermatology & Cutaneous Biology, Thomas Jefferson University. "I would like to thank both the Debra Foundations for funding this work as well as Onconova for providing a research drug that may target the life-threatening cancers arising in these patients."

On April 22, 2021 The Leukemia & Lymphoma Society Therapy Acceleration Program (LLS TAP) reported five new investments aimed at speeding the development of new and improved immunotherapies for the treatment of blood cancers (Press release, The Leukemia & Lymphoma Society, APR 22, 2021, View Source;lymphoma-society-therapy-acceleration-program-lls-tap-announces-five-new-investments-supporting-next-generation-cell-therapies-and-a-novel-immune-checkpoint-inhibitor-301274791.html [SID1234578384]. LLS has dedicated more than $100 million over the past several decades, through both grants and TAP investments, to advancing pioneering approaches that harness cellular immunotherapies to fight blood cancers .

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!

"We are very proud of our role in bringing groundbreaking immunotherapies to patients, including chimeric antigen receptor (CAR) T-cell therapy, which harnesses the power of T cells to kill tumor cells," said Louis DeGennaro, Ph.D., LLS President and Chief Executive Officer. "Our TAP venture philanthropy initiative is investing in the next generation of immunotherapies to find cancer treatments that are even more effective, safer, longer-lasting, easier to use, and active against more types of cancer."

LLS TAP is a strategic venture philanthropy funding initiative to accelerate high-risk, innovative blood cancer therapeutics and change the standard of care in leukemia, lymphoma, and multiple myeloma. LLS TAP makes individual investments of up to approximately $10 million and offers insights to funded companies from its extensive knowledge of blood cancer indications, the treatment landscape, and the expertise of LLS staff. LLS TAP due diligence and investment credibility can help companies raise needed funds to drive clinical-focused research forward. Today’s investments include three companies that have novel immunotherapies in early human clinical trials and two companies with promising programs in preclinical development for blood cancers:

Caribou Biosciences, Inc, co-founded by CRISPR pioneer and Nobel Prize winner Jennifer Doudna, Ph.D., uses next-generation CRISPR genome editing technology, referred to as chRDNA, to develop "off-the-shelf" CAR T-cell therapies for hard-to-treat blood cancers. Off-the-shelf CAR T-cell therapies use healthy donor T cells instead of reengineering a patient’s own cells. This approach could make treatment less costly and immediately available to patients with rapidly progressing disease.

The company’s lead therapeutic candidate, CB-010, which targets the CD19 protein, is in a phase 1 clinical trial (ANTLER) for treatment of patients with relapsed or refractory non-Hodgkin lymphoma. The company is working on additional off-the-shelf CAR T-cell therapies targeting BCMA and CD371 proteins, which play key roles in multiple myeloma and acute myeloid leukemia, respectively. Caribou is also working on adding a CAR to induced pluripotent stem cells to differentiate them into "natural killer" or NK cells to create a new type of immune cell therapy called CAR-NK, which holds promise for the treatment of solid tumors and metastases in addition to blood cancers.

NexImmune, a company that benefitted from an initial TAP investment nearly four years ago and now received a second investment as part of the company’s initial public offering, is taking a different approach. The company’s Artificial Immune Modulation, or AIM platform, does not rely on genetic manipulation of T cells. Instead, it uses nanoparticles—specific tiny molecules—to directly activate the body’s T cells to fight specific types of cancer. The company has two novel products in phase 1 clinical trials; NEXI-001 to treat advanced acute myeloid leukemia or myelodysplastic syndrome, and NEXI-002 for the treatment of advanced multiple myeloma.

Immune-Onc Therapeutics is developing a novel type of myeloid immune checkpoint inhibitor. Their lead candidate, called IO-202, a first-in-class antibody targeting the immune inhibitory receptor Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB) member 4 (LILRB4, also knowns ILT3), has entered a phase 1 clinical trial for the treatment of advanced acute myeloid leukemia and chronic myelomonocytic leukemia. The company also plans to evaluate this compound in solid tumors soon. The company’s work builds on early research by Chengcheng (Alec) Zhang, Ph.D. at The University of Texas Southwestern Medical Center that was also funded by LLS grants. Checkpoint inhibitors work by ‘releasing the brake’ from the body’s own immune cells so that they can effectively attack cancer cells. In addition to their lead drug candidate, Immune-Onc has active preclinical programs targeting other members of the LILRB family including IO-108, a novel antagonist antibody targeting LILRB2 (also known as ILT4) which is currently in the IND-enabling stage, IO-106, a first-in-class anti-LAIR1 antibody, and other undisclosed programs for solid tumors and blood cancers.

CARISMA Therapeutics is a spin out company from the University of Pennsylvania (Penn), founded by Saar Gill, M.D., Ph.D. and Michael Klichinsky, PharmD, Ph.D., SVP of Research. Early work at Penn was supported in part by LLS grants. CARISMA is developing CAR-engineered macrophages, white blood cells that exert broad effects on the immune system, to infiltrate solid tumors, eat away at cancer and activate the adaptive immune system. Based on preclinical studies, the company’s highly differentiated CAR-macrophage (CAR-M) platform may have the potential to overcome challenges encountered by other cell therapies such as trafficking limitations to the tumor site, immunosuppressive tumor microenvironments and the heterogeneous expression of tumor-associated antigens. CARISMA is actively enrolling patients for its phase 1 clinical trial for CT-0508, a HER2-targeted CAR-M. CARISMA is also working closely with LLS TAP to develop one or more CAR-M therapies for blood cancers.

Abintus Bio is developing cutting-edge in vivo CAR therapies that allow for powerful CAR T cells to be generated directly in a patient’s body, eliminating the need for time-consuming and costly collection, engineering and re-infusion of patient T cells. The innovative Abintus approach aims to reprogram T cells inside the body to attack and eliminate tumors. This technology is currently in preclinical testing and could, if successful, improve patient access with an off-the-shelf profile while advancing patient outcomes.
"Funding from leaders like LLS TAP helps fuel the progress of our clinical development program, bringing next-generation treatments that much closer to patients," said Rachel Haurwitz, Ph.D., President and Chief Executive Officer of Caribou Biosciences. "We’re particularly excited to be working with LLS TAP because it gives us access to their deep knowledge of blood cancer and their network of patients and drug development experts."

"The LLS track record in accelerating lifesaving blood cancer treatments is unparalleled," said Lee Greenberger, Ph.D., LLS Chief Scientific Officer. "The continuity of our funding from preclinical through clinical research demonstrates our commitment to supporting innovative therapies as they move from the laboratory into practice."

"Since 2017, three TAP-supported therapies have been approved in the U.S.," added Lore Gruenbaum, Ph.D., VP of TAP. "TAP partnership provides companies with deep blood cancer expertise and increases their credibility with the investment community. We look forward to our ongoing partnerships with these five companies as we work together to provide new hope for patients."

On April 22, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the first quarter of fiscal year 2021 (Press release, Chugai, APR 22, 2021, View Source [SID1234578335]).

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!

"In the first quarter of 2021, despite growth in royalties and other operating income, both revenues and profits declined due to a decline in domestic and overseas sales. Still, there is no change to the full-year forecast for growth in revenues and profits as sales of mainstay products Tecentriq and Kadcyla are progressing significantly higher than expected at the beginning of the fiscal year, and we are also anticipating increases in exports to Roche and in royalty and profit-sharing income from the overseas growth of in-house products such as Hemlibra. In terms of R&D, we have received regulatory approval for our new anti-cancer drug Polivy and the liquid biopsy test FoundationOne Liquid CDx Cancer Genomic Profile, and we have begun a domestic Phase I clinical trial for the antibody cocktail casirivimab and imdevimab for COVID-19. We will continue striving to deliver innovation to patients as quickly as possible," said Dr. Osamu Okuda, Chugai’s President and CEO.

[First quarter results for 2021]

Chugai reported declines in revenues and operating profit for the first quarter (Core-basis) by approximately 6% and 12% year-on-year, respectively.

Revenues decreased as a whole due to a decrease in domestic and overseas sales, despite an increase in royalties and other operating income. Domestic sales declined by approximately 7% overall. Despite the sales increase in Oncology field driven by the double-digit growths of Tecentriq and Kadcyla, sales in the Primary field decreased by 20%, significantly impacted by the NHI drug price revision in April 2020 as well as generic competition. Overseas sales decreased by less than 20%, due to a decrease in exports of Actemra and other products to Roche. On the other hand, royalties and other operating income increased by double digits mainly due to an increase in royalty and profit-sharing income of Hemlibra, despite a decrease in other operating income from one-time income.

Cost to sales ratio remained at the same level as the same period last year. Operating expenses increased by approximately 9% as research and development expenses increased approximately by 15% due to steady progress in development projects while marketing and distribution expenses and general and administration expenses were almost flat year-on-year. As a result, operating profit declined by double digits.

The Company also made good progress in research and development. Chugai obtained regulatory approval in March 2021 for Polivy in relapsed or refractory diffuse large B-cell lymphoma, and FoundationOne Liquid CDx Cancer Genomic Profile as the first blood-based comprehensive genomic profiling test for solid tumors in Japan. A Phase III clinical trial evaluating Tecentriq in early-stage lung cancer achieved its primary endpoint, making a steady progress toward the regulatory application for line extension in 2021.

Regarding the clinical development of Actemra for COVID-19 pneumonia, Chugai announced in March 2021 that the Phase III REMDACTA study in combination with remdesivir did not meet its primary endpoint. Chugai is continuing further evaluation of overall risk-benefit profile based on the results of J-COVACTA, REMDACTA, COVACTA, EMPACTA trials and other studies of Actemra for COVID-19 pneumonia. In addition, a Phase I clinical trial in Japan was initiated in March 2021 for the investigational antibody cocktail casirivimab and imdevimab for COVID-19 in-licensed from Roche.