On October 22, 2021 Chugai Pharmaceutical Co., Ltd. reported that has revised the full-year financial forecasts for the fiscal year ending December 2021 announced on February 4, 2021, considering the recent trend of business results, as follows (Press release, Chugai, OCT 22, 2021, View Source [SID1234591869]).

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!

1. Revised consolidated financial forecasts for the full fiscal year ending December 2021 (January 1 to December 31, 2021)

2. Reasons for the revisions
Forecast of Revenues has been revised to ¥970.0 billion, an increase of ¥170.0 billion from the previous forecast. For domestic sales, forecasted sales amount attributable to the supply of Ronapreve to the government for the fiscal year has been included, and the progress and revised assumptions for each product including Avastin and Tecentriq, which have progressed better than the previous forecast have been reflected. For overseas sales, exports of Actemra and Hemlibra to Roche have been forecasted to be higher than the previous forecast. For royalties and other operating income, the forecast of income and one-time income for Actemra and Hemlibra has also been updated.

Core operating profit forecast has also been revised to ¥400.0 billion, an increase of ¥80.0 billion from the previous forecast. In addition to the above-mentioned revision of revenue forecast, higher cost to sales ratio due to a change in the product mix from the original assumption, etc., foreign exchange effects and increases in some expenses attributable to increased sales and profits have been included.

Core EPS is forecasted to be ¥178.00, an increase of ¥37.00 from the previous forecast. Year-end dividend forecast and the forecast for the Core dividend payout ratio have not been determined at this time.

The revised full-year consolidated forecasts are based on updated foreign exchange rate assumptions: 1CHF = 117 JPY, 1EUR = 130 JPY, 1USD = 107 JPY, and 1SGD = 81 JPY.

On October 22, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the third quarter of fiscal year 2021 (Press release, Chugai, OCT 22, 2021, View Source [SID1234591824]).

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 achieved robust double-digit growth in both revenues and profits in the nine months under review. This was due to significant increases in both domestic and overseas sales, driven by new products and mainstay products, as well as continued growth in royalty income. As a result, we raised our full year forecast for FY2021. In terms of R&D, I am very pleased that our first mid-size molecule project, LUNA18, has entered the clinical development phase. Mid-size molecule drugs are expected to become the third modality to drive our future growth. Concerning COVID-19, we have filed Ronapreve for subcutaneous administration and the additional indications of treatment for asymptomatic patients and prophylaxis. In order to satisfy unmet medical needs through innovation, we will continue pursuing world-leading science and technology to contribute to patients awaiting treatments." said Dr. Osamu Okuda, Chugai’s President and CEO.

[Third quarter results for 2021]

Revenues and Core operating profit increased by approximately 17% and 25%, respectively, for the third quarter (nine months, Core-basis), mainly due to increases in sales and royalty and profit-sharing income.

Revenues increased by approximately 20% in total since sales, which recorded flat year-on-year growth for the first half, turned to double-digit growth. Domestic sales increased by double digits as the strong oncology field grew further with a mainstay product Tecentriq maintaining high growth despite the NHI price revision in August this year, and the contribution of a new product Polivy. In the primary field, sales increased approximately 30% after reporting a decrease by roughly 10% in the first half. The growth was driven by new products, with the supply of Ronapreve to the government and the market penetration of Enspryng, and double-digit growth of mainstay products, Actemra and Hemlibra. Overseas sales recovered growth and increased by approximately 10% as the further growth of Hemlibra outweighed the decrease in the sales of Actemra. Royalties and other operating income increased by approximately 20% mainly due to increases in royalty and profit-sharing income related to Hemlibra, despite a continued decrease in other operating income resulting from one-time income.

Cost to sales ratio improved by 1.2% points year-on-year to 41.9%, mainly due to an increase in the proportion of in-house products including Hemlibra. Operating expenses increased approximately by 10% as research and development, marketing and distribution, and general and administration expenses all increased. Research and development expenses increased by double digits, continued from the first half, associated with the progress of projects. The increases in marketing and distribution expenses were affected by last year’s decrease in activities due to the spread of the COVID-19. General and administration expenses increased owing to the enterprise tax and various expenses. As a result, core operating profit increased by approximately 25%.
The Company also made good progress in research and development. Its first mid-size molecule project, LUNA18, has entered the clinical development phase. Chugai has been focusing on mid-size molecule drugs as a new modality to constitute the mainstay for driving its medium-to long-term growth. LUNA18 is an oral cyclic peptide RAS inhibitor, and a Phase I clinical trial has been initiated for solid tumors. As for other in-house projects, Chugai has started development of Enspryng in generalized Myasthenia Gravis (gMG), initiating a Phase III clinical trial.

In the efforts to develop treatments for COVID-19, Chugai filed an application for the antibody cocktail Ronapreve, which had received Special Approval in July. The filing is to obtain approval for additional indications for prophylaxis of COVID-19 and treatment of asymptomatic COVID-19, and additional subcutaneous administration. In addition, an application for approval was filed in Europe for Actemra for an additional indication of COVID-19 pneumonia.

[Revision of Full-Year Forecast for 2021]

Chugai raised forecasts (Core-basis) for FY2021 following the strong nine-month results. Regarding domestic sales, the sales forecast attributable to the supply of Ronapreve to the government, which was not expected at the beginning of the fiscal year, has been included, and the progress and revised assumptions have been reflected for each product including Avastin and Tecentriq, which are progressing ahead of the initial forecast. The forecast for overseas sales has been revised upward reflecting that sales of Actemra and Hemlibra should exceed the original forecast. Royalties and other operating income have been also revised upward. As a result, the revenues have been raised to ¥970 billion, an increase of ¥170 billion from the initial forecast. Operating profit forecast has been revised to ¥400 billion, up ¥80 billion from the initial forecast, taking into account a higher cost to sales ratio due to a change in the product mix, and increases in some expenses including the foreign exchange effects.

In accordance with the revision of the financial forecast, year-end dividends forecast was also revised to an undecided value. Reflecting the significant changes in the profit structures, year-end dividends will be decided based on the Company’s dividend policy* after the fiscal year ends.

*Regarding income distribution, taking into account the strategic funding needs and earning prospects, Chugai aims for a consolidated dividend payout ratio of 45% on average in comparison with Core EPS to provide a stable allocation of profit to all shareholders.

On October 22, 2021 OS Therapies, a research and clinical-stage biopharmaceutical company reported the dosing of the "First Patient In" for its lead OST-HER2 (OST31-164) program in a potentially pivotal Phase IIb clinical trial. OST-HER2 has already received Fast-Track and Orphan Designation from the EMA and FDA (Press release, OS Therapies, OCT 22, 2021, View Source [SID1234591795]). The nationwide open-label trial will enroll 39 to 45 Osteosarcoma patients whose cancer has metastasized to the lungs and has been surgically resected.

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!

OST-HER2 is targeting Osteosarcoma in children and young adults, and potentially other solid tumors. It is a Lm vector-based off-the-shelf Immunotherapy intended to prevent metastasis, delay recurrence, and increase overall survival. "The OST-HER2 treatment has been highly successful in multiple trials in canine osteosarcoma, demonstrating 3x improvement in Overall Survival and Disease Progression, with significant similarities between human and canine Osteosarcoma. We hope to demonstrate that it works as well – or even better – in kids," said Dr. Robert Petit, CMO/CSO of OS Therapies.

The clinical trial is being conducted across 20 Children’s Oncology Group (COG) affiliated institutions, starting at Seattle Children’s Hospital. "The Osteosarcoma Community and Clinicians have a great deal of Institutional Enthusiasm regarding the OST-HER2 trial," said COG Group Chair Dr. Douglas S. Hawkins, Professor of Pediatrics, Seattle Children’s Hospital and University of Washington. "It is our intent to enroll this trial as quickly and safely as possible to see if the results are as good as we have anticipated. This is potentially a very important new treatment option in Osteosarcoma."

"We are all very excited to get this trial started – the technology has shown great promise in Osteosarcoma," said Principle Investigator Dr. Damon Reed, Moffitt Cancer Center. "It is our goal to get the other sites up and enrolling the remaining patients over the next few weeks."

About Osteosarcoma
Osteosarcoma is a solid tumor of the bone that predominantly occurs in adolescent and young adults (AYA). Standard treatment includes surgery and chemotherapy. For patients with initially metastatic or recurrence after chemotherapy, there is a significantly poorer prognosis.

On October 22, 2021 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported its financial results and business highlights for the six months ending June 30, 2021, and provides an outlook for the rest of the year (Press release, NOXXON, OCT 22, 2021, View Source [SID1234591794]).

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!

Aram Mangasarian, CEO of NOXXON commented: "I am delighted to report on the strong progress NOXXON has made in 2021 so far by delivering on its unique approach to target the tumor microenvironment and enhance the way cancers are treated. We continue to develop our lead asset NOX-A12 in two indications with extremely high unmet medical needs, namely brain and pancreatic cancer. The impressive clinical data generated so far indicate the great potential of NOX-A12 to treat and improve the outcomes for patients in these extremely difficult indications."

"We have also significantly strengthened our Supervisory Board by adding three new board members with outstanding and highly relevant experience in business development, corporate law, and finance. On the business development front, I am delighted that MSD/Merck has renewed its confidence in NOXXON by entering a new collaboration agreement to develop NOX-A12 in combination with its market leader PD-1 inhibitor Keytruda. All these recent developments make us confident in our ability to deliver great value to shareholders in the months and years to come," added Aram Mangasarian.

H1 2021 Business Highlights

Scientific Advisory Board (SAB)
Top US and EU pancreatic cancer experts joined NOXXON’s SAB in February 2021:
Dr. Elena Gabriela Chiorean, Dr. Eileen O’Reilly, Prof. Dr. Thomas T. W. Seufferlein, Dr. Daniel D. Von Hoff and Dr. José Saro (Chair) have long-standing clinical expertise, cutting-edge scientific knowledge, and a track record of successfully developing new drugs.
Supervisory Board
Appointment of 3 highly distinguished new members to the Supervisory Board in June 2021:
Dr. Martine J. van Vugt, Senior Vice President Corporate Strategy and Development at Genmab, with 20 years of successful biotechnology industry experience, an inventor of two successfully commercialized cancer drugs (Darzalex and Tepezza) and an expert in corporate transactional and licensing operations.
Gregory Weaver, CFO of atai Life Sciences, with over 25 years in the life sciences industry ranging from start-ups to publicly traded commercial stage companies, has raised over US$1.5 billion in financing transactions, managed 3 IPOs, and has extensive M&A and business development experience.
Susan Coles, General Counsel and Head of Finance at Vivet Therapeutics, is a specialist in corporate law with over 25 years of experience in international collaborations and corporate/commercial activities, including more than 15 years in the life sciences sector.
H1 2021 Financial Highlights

For the reporting period, the Group – NOXXON Pharma N.V. and NOXXON Pharma AG – has not generated any revenues. The Group does not expect to generate any revenues from any product candidates that it develops until the Group either signs a licensing agreement or obtains regulatory approval and commercializes its products or enters into collaborative agreements with third parties.

In H1 2021, the Group generated K€142 in other operating income from the sale of raw materials and services as well as other income.

Operating costs increased by 213% in H1 2021 over the same period last year, most of this increase being driven by research and development (R&D) expenses, accounting for 81% of operating costs. The R&D expenses increased by 421% in H1 2021 over the same period last year as NOXXON continued to make progress with the clinical stage assets. General and administrative (G&A) expenses increased by 14% to support operational activities. These operating costs led to €5.9 million in net cash outflow from operations.

The Group was successful in strengthening its balance sheet by raising a total of €9.3 million in net proceeds from multiple sources, including €6 million via private placement.

These activities and fund raising in H1 2021 led the Group to report €13.7 million in cash and cash equivalents, in addition to having available secured financing of €10.45 million (nominal) drawable at the Company’s discretion. These available resources mean that NOXXON has sufficient financial visibility into June 2022.

H1 2021 and Year-to-Date Clinical Highlights

NOXXON had a busy and productive H1 2021 period on the R&D front with significant progress made on its lead asset, NOX-A12.

NOX-A12 + radiotherapy in glioblastoma (GBM) Phase 1/2 study (GLORIA) in patients newly diagnosed with aggressive brain cancer is ongoing. All patients have been recruited in the low, mid and high dose cohorts (200, 400 or 600 mg NOX-A12 per week), and will have completed 6 months of therapy in Q1 2022. Data from two dose cohorts (low and medium) showed that:
Five out of six patients showed reductions in tumor size with maximal reductions baseline ranging from 2% to 71%, including two objective responses (reduction larger than 50%);
Five of six patients achieved reduced blood flow to the tumor compared with baseline;
Comparison of pre-treatment to on-therapy tumor tissue from one patient in the low dose cohort revealed that NOX-A12 effectively suppresses the target.
Based on the data obtained from the initial dose cohorts, an expansion of the study with patients who would not benefit from standard of care chemotherapy is planned. NOXXON believes that the next step in development should be a pivotal trial following the current Phase 1/2 study and targets first market approval in 2025.

NOX-A12 + immunotherapy. The Journal for ImmunoTherapy of Cancer published in October 2021 the final peer-reviewed results of the Phase 1/2 study (OPERA) of NOX-A12 and pembrolizumab in metastatic pancreatic and colorectal cancer patients who failed standard therapy.
Outlook for the Remainder of 2021 and 2022

NOXXON continues to progress its ongoing Phase 1/2 clinical trial of NOX-A12 plus radiotherapy in first-line brain cancer (glioblastoma) patients who are shown by biomarker analysis of their tumor tissue to be resistant to the current standard of care chemotherapy. The clinical trial remains on track to report data from the third cohort (highest dose) in Q1 2022. The Company also continues to advance the regulatory filings for the expansion cohorts to explore additional patient populations and combination therapies in brain cancer.

The operational environment continues to be uncertain due to the COVID pandemic and its consequences and NOXXON remains dependent upon its service providers for drug manufacturing and conducting clinical trials.

NOXXON’s long-term strategic plans now include the following trials by indication:

NOX-A12 + radiotherapy in Brain Cancer:

Completion of the ongoing Phase 1/2 dose escalation trial is planned for Q1 2022. Thereafter, additional data are expected from expansion arms testing NOX-A12 in another population of brain cancer patients or in combination with other agents.
Anticipating that the ongoing Phase 1/2 trial data supports further development, NOXXON plans to initiate in 2022 a pivotal trial of NOX-A12 combined with radiotherapy in first-line MGMT promoter-unmethylated glioblastoma patients vs. standard of care, with first market authorization application and approval targeted for 2025.
NOX-A12 + immunotherapy in Pancreatic Cancer:

Following the promising results from the OPERA trial, NOXXON has decided to pursue the NOX-A12 + immunotherapy combination in second-line pancreatic cancer with a dosing regimen of NOX-A12 optimized to induce anti-tumor immune responses.
A two-step approach is planned with a Phase 2 study (OPTIMUS) comparing two NOX-A12 combinations with anti-PD-1 antibody and two different standard of care chemotherapy regimens in second-line patients followed by a pivotal trial comparing the best combination to the standard of care.
To conduct the OPTIMUS study, NOXXON and MSD (Merck & Co., Inc., Kenilworth, N.J. USA) have entered into a collaboration by which MSD will provide pembrolizumab (Keytruda) and expert advice for the study protocol.
As previously announced, first patients are expected to be dosed in Q2 2022. The study completion is expected in early 2024.
This strategic approach will enable NOXXON to choose the optimal regimen to move forward into a randomized, controlled pivotal study targeting marketing authorization application and approval in 2027.
NOX-E36 in Oncology:

The second clinical stage asset, NOX-E36, is also being readied for the first clinical trial of NOX-E36 in oncology. Manufacturing of clinical supply has been contracted and clinical trial supply is projected to be available in Q1 2022, and trial initiation is targeted for mid-2022.

The Half-Year Financial Report 2021 can be downloaded from the NOXXON website.

On October 22, 2021 CEL-SCI Corporation (NYSE American: CVM) reported it has completed the expansion of its existing dedicated Multikine* (Leukocyte Interleukin, Injection) cGMP manufacturing facility (Press release, Cel-Sci, OCT 22, 2021, View Source [SID1234591793]). The expansion was undertaken in anticipation of filing a Biologics License Application (BLA) which, if approved, will allow Multikine produced in the facility to be commercially distributed. The construction, which began in 2020, expanded the facility and added various upgrades to ensure it will be in compliance with all requirements of the FDA’s Current Good Manufacturing Practice (GMP) regulations. The facility’s production capacity has been doubled to meet anticipated market demand for Multikine once it is licensed. The renovations also anticipate that additional personnel will be required to staff a second manufacturing shift to meet the eventual market demand for Multikine. Following an $11 million investment to increase production, CEL-SCI staff recently moved back into the renovated facility.

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 now validating the facility and will begin preparing our facility license application for Multikine. We continue to hire and train additional personnel required to efficiently operate the manufacturing facility in compliance with all federal and state requirements," stated CEL-SCI CEO, Geert Kersten.

CEL-SCI reported topline results in June 2021 from its landmark pivotal Phase 3 study of Multikine as a neoadjuvant treatment of advanced primary previously untreated head and neck cancer, which is believed to be the largest study in the world for this indication.