On November 10, 2021 Pharos iBio reported its PHI-501, a next-generation anticancer drug for acute myeloid leukemia (AML), has won orphan drug designation from the U.S. Food and Drug Administration (FDA) (Press release, Pharos iBio, NOV 10, 2021, View Source [SID1234595286]).

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It was the U.S. regulator’s second orphan drug designation of the Korean company’s product. FDA granted similar status to Pharos iBio’s main pipeline, PHI-101, a next-generation FMS-like tyrosine kinase 3 (FLT3) inhibitor for AML, in 2019.

PHI-501 is the first targeted anticancer therapeutics against neuroblastoma-RAS (NRAS) mutation using a more effective method for inhibition using synthetic lethality.

NRAS is a protein that plays a significant role in cell differentiation, proliferation, and survival in acute myeloid leukemia. However, NRAS mutants continuously send signals for cell growth which also leads to cancer growth.

According to Decision Resources Group, a global health industry research firm, AML is a rare disease that shows increased occurrence in the older populations above 65. It is particularly prevalent in the U.S. and Europe. The group expected the global market for AML treatments to reach $2.5 billion by 2025 with a 15 percent annual growth.

Pharos iBio holds eight new drug pipelines, including PHI-101, and originally built big data and AI-based platform technology.

Through open innovations, the company plans to promote joint research and development and technology transfer with global pharmaceutical companies and overseas organizations.

"We will benefit from various incentives, including shortened review period for clinical approval and commercialization, reduced application fee with tax exemption, and a seven-year marketing exclusivity," said Pharos iBio CDO Han Hye-jung, former principal scientist at Roche Sequencing Solutions. "We will speed up the clinical trials of PHI-501 to enter the global market."

The company has recently built a synthesis and bio laboratory at its headquarters in Anyang, Gyeonggi Province, establishing an infrastructure to discover new drug candidates independently.

Pharos iBio received approval for the first phase 1 clinical trial of PHI-101 in Korea and continued to strengthen its ability to develop new drugs for rare and incurable diseases.

On November 10, 2021 CureVac N.V. (Nasdaq: CVAC), a global biopharmaceutical company developing a new class of transformative medicines based on messenger ribonucleic acid ("mRNA"), reported promising data from the completed dose-escalation part of the Phase 1 clinical trial of CV8102, the company’s lead oncology candidate (Press release, CureVac, NOV 10, 2021, View Source [SID1234595052]). The data support the hypothesis that local injection of the RNA immuno-modulator into a single tumor lesion is able to induce a systemic response leading to immune attack against both injected and non-injected tumors. Extensive analysis of immune cell activation shows efficient stimulation of the immune system characterized by the induction of interferon alpha and interferon gamma signaling pathways. Serial tumor biopsies from individual patients demonstrated increased infiltration of tumor-fighting T cells in the microenvironment of injected as well as non-injected tumors. The study focuses on patients with advanced cutaneous melanoma, adenoid cystic carcinoma and squamous cell carcinoma of the skin or head and neck, treated with CV8102 alone and in combination with anti-PD-1 antibodies. The data are being presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference, held in Washington, D.C., and virtually from November 10–14, 2021.

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"The preliminary data from the Phase 1 dose escalation part of this study allow a much deeper understanding of the promising biologic effects of CV8102 observed in several patients," said Dr. Klaus Edvardsen, Chief Development Officer at CureVac. "CV8102 is designed to mimic a viral infection of the injected tumor. This potentially leads to a broad activation of tumor-specific T cells, which can kill tumor cells at the injected but also at distant sites. We expect that the data will be supplemented by upcoming results of the expansion study, which will provide further insight into which patients are more likely to experience a clinical response to CV8102."

An expansion part of the Phase 1 study, initiated in February and fully recruited in October 2021, aims to confirm the safety, tolerability and efficacy of CV8102 at the recommended 600μg dose in patients with advanced melanoma. The study involves 40 trial participants, with 10 in the single-agent cohort and 30 in the combination cohort. Data from the expansion part of the study is expected in the second half of 2022.

The dose-escalation part of the study included a total of 58 patients, 33 of which were treated in the in the single-agent cohort and 25 in the combination cohort. Data presented in September at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) conference found that as of the cutoff date of June 21, 2021, in the single-agent CV8102 dose-escalation cohort, one patient with a complete response and two patients with a partial response were observed. In addition, 12 patients experienced stable disease. In the PD-1 combination dose-escalation cohort, one PD-1 refractory melanoma patient experienced a partial response while three patients experienced stable disease.

About CV8102 and the Phase 1 Clinical Trial

CV8102 is a single-stranded non-coding RNA optimized to maximize activation of cellular receptors that normally detect viral pathogens entering the cells, such as toll-like receptors 7 and 8 (TLR7/8), and retinoic acid inducible gene I (RIG-I), mimicking a viral infection of the tumor. CV8102 is designed to recruit and activate antigen-presenting cells at the site of injection to present tumor antigens released from tumor cells to T cells in the draining lymph node. This potentially leads to activation of tumor-specific T cells, which can kill tumor cells at the injected site, but also at distant non-injected tumor lesions or metastases.

The Phase 1, open-label, dose escalation and expansion study of CV8102 is fully recruited and includes patients with advanced melanoma, cutaneous squamous cell carcinoma, squamous cell carcinoma of head and neck or adenoid cystic carcinoma. The primary objective of the study is to assess safety and tolerability of CV8102.The dose escalation part tests escalating doses of single-agent CV8102 and CV8102 in combination with licensed anti-PD-1 antibodies in the range of 25-900µg. The expansion part of the study focuses on patients with advanced melanoma treated with a recommended dose of 600µg.

On November 10, 2021 F-star Therapeutics, Inc. (NASDAQ: FSTX), a clinical-stage biopharmaceutical company dedicated to developing next generation bispecific immunotherapies to transform the lives of patients with cancer, reported third quarter 2021 financial results and a corporate update (Press release, F-star, NOV 10, 2021, View Source [SID1234595068]).

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Eliot Forster, CEO of F-star Therapeutics, Inc., said, "A year on from listing on NASDAQ, we have delivered on our planned milestones, and through them value for patients, partners and our investors. Our agile, tenacious approach, working with world-leading investigators, continues to further F-star’s mission to bring our unique bispecific antibodies to patients who need them most. We continue to advance four clinical programs, initiate validating partnerships and execute our financial plan. This past quarter included a number of clinical updates and significant new partnerships with AstraZeneca and Janssen Biotech. I’m proud of the team paving the way with huge passion and dedication to make real the promise of next generation immunotherapies."

The Company continues to advance FS118, F-star’s first-in-class bispecific antibody targeting LAG-3 and PD-L1, in checkpoint inhibitor relapsed head and neck cancer and in checkpoint inhibitor naïve patients with non-small cell lung cancer (NSCLC) and diffuse large B cell lymphoma (DLBCL), with a clinical trial in the latter two populations currently being initiated. FS120, F-star’s first-in-class dual-agonist, bispecific antibody targeting CD137 and OX40, remains on track in the clinic, having completed the accelerated dose titration phase, with presentations at ESMO (Free ESMO Whitepaper) 2021 and SITC (Free SITC Whitepaper) 2021. SB 11285, a second-generation STimulator of INterferon Gene (STING) agonist, continues to advance well in the clinic, further to the update provided in the second quarter of 2021. FS222, the potentially best-in-class bispecific targeting PD-L1 and CD137, is also progressing well in the clinic.

The Company also announced during the third quarter significant new partnerships with both AstraZeneca PLC and Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, leveraging our platform technology. With four clinical-stage programs in progress, F-star is focused on the further development of its wholly-owned pipeline of tetravalent bispecific antibodies, as well as collaborations that have the potential to bring value to shareholders and patients alike.

THIRD QUARTER 2021 AND RECENT HIGHLIGHTS

FS118 development expanded following external clinical validation of the LAG-3 target: The expansion of the FS118 clinical development into checkpoint naïve, biomarker enriched NSCLC and DLBCL patients will broaden the clinical reach of this exciting LAG-3 & PD-L1 targeting bispecific antibody. This adds to the already ongoing checkpoint inhibitor relapsed head and neck cancer study that is anticipated to report data in mid-2022.

Combination of FS120 with KEYTRUDA: In August, F-star announced a clinical trial collaboration and supply agreement with Merck & Co., Inc., Kenilworth, NJ, USA (MSD) to evaluate the combination of FS120, F-star’s first-in-class dual-agonist bispecific antibody targeting CD137 and OX40, with KEYTRUDA (pembrolizumab), MSD’s anti-PD-1 therapy. FS120 has completed the accelerated dose titration phase in monotherapy with no safety concerns identified, and the pharmacokinetics were in line with expectations. The Company continues dose escalation to determine an optimal dosing regimen to initiate the KEYTRUDA combination.

AstraZeneca licenses STING inhibitors: In July, F-star entered into an exclusive licensing agreement with AstraZeneca plc under which AstraZeneca received global rights to research, develop and commercialize next generation STING inhibitor compounds. AstraZeneca was granted exclusive access to F-star’s novel preclinical STING inhibitors and will be responsible for all future research, development and commercialization of the STING inhibitor compounds. This forms part of the second CVR agreement with the former shareholders of Spring Bank Pharmaceuticals, Inc. (Spring Bank). F-star retains rights to all STING agonists currently in clinical development for patients with cancer.

SB 11285 Phase 1 interim update: In July, F-star provided an interim update on the safety, tolerability and pharmacokinetics of its intravenously administered novel STING agonist, alone and in combination with atezolizumab. SB 11285 appeared to be well tolerated both alone and in combination across all dose levels tested to-date, including five dose levels as monotherapy and three dose levels as a combination. The Part 1a/1b study database lock (as defined in the first CVR agreement with Spring Bank’s former shareholders) has been completed. Based on the positive emerging clinical data, further dose escalations are ongoing, and a further clinical update is planned for the second half of 2022.

Johnson and Johnson licenses five new programs, based on platform technology: Under the terms of the license and collaboration agreement, F-star will grant Janssen Biotech a worldwide, exclusive royalty-bearing license to research, develop, and commercialize up to five novel bispecific antibodies directed to Janssen therapeutic targets using F-star’s proprietary Fcab and mAb2 platforms. Janssen will be responsible for all research, development, and commercialization activities under the agreement.

THIRD QUARTER 2021 FINANCIAL SUMMARY

Cash and cash equivalents as of September 30, 2021, were $71.1 million, compared to $18.5 million as of December 31, 2020. The up-front payment of $17.5 million in connection with the license and collaboration agreement with Janssen Biotech is expected to be received in the fourth quarter of 2021.

Research & Development (R&D) expenses were $5.1 million for the quarter ended September 30, 2021, compared to $5.3 million for the corresponding quarter in 2020, which included non-cash stock-based compensation expense of $1.1 million and $1.1 million, respectively.

General & Administrative (G&A) expenses were $5.2 million for the quarter ended September 30, 2021, compared to $7.3 million for the third quarter of 2020, which included non-cash stock-based compensation expense of $0.4 million and $59,000, respectively.

Net loss was $10.8 million, or a loss per share of $0.52 (basic and diluted), for the quarter ended September 30, 2021, compared to a net loss of $3.5 million, or a loss per share of $1.88 (basic and diluted), for the quarter ended September 30, 2020.

CONFERENCE CALL AND WEBCAST
F-star will host a conference call today, November 10, 2021, beginning at 9:00 a.m. EST.

To access the call, participants may join via a live webcast on the Investors & News section of the F-star Therapeutics website, under Events and Presentations. To join by phone, participants may dial the following numbers at least 10 minutes prior to the start of the call:

A replay of the conference call will be available for 90 days from the date of the call and may be accessed in the Investors & News section of the F-star Therapeutics website under Events and Presentations.

On November 10, 2021 Ascendis Pharma A/S (Nasdaq: ASND), reported financial results for the third quarter ended September 30, 2021 (Press release, Ascendis Pharma, NOV 10, 2021, View Source [SID1234595084]).

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"We are at a defining moment on our way to fulfilling Vision 3×3, our strategy to build a leading global biopharma company. In late August, we received U.S. FDA approval for TransCon hGH, and in mid-October we launched in the U.S.," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We believe this first FDA-approval for a TransCon product validates our technology platform, product innovation algorithm, and product development capabilities. Our robust, diverse pipeline, which today includes five unique clinical stage product candidates across endocrinology rare diseases and oncology, demonstrates our passion for following the science to address important unmet patient needs."

Company Highlights & Progress

TransCon hGH
Now commercially available in the U.S. for the treatment of pediatric GHD. Sales in the U.S. will be reported under the brand name.
In Europe, final decision anticipated from the European Commission on our Marketing Authorisation Application, for TransCon hGH in pediatric GHD by the end of 2021 or early 2022.
Enrollment continues in our foresiGHt Trial, a global Phase 3 trial in adult GHD and in the riGHt Trial, a Phase 3 trial in Japan in pediatric GHD.
TransCon PTH
Completed enrollment in the Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism, with topline results expected in Q1 2022.
58 subjects continue in the PaTH Forward Trial open-label extension as of November 7, 2021, with 84-week topline results expected in Q4 2021.
Enrollment continues in the PaTHway Japan Trial, a single-arm Phase 3 trial of TransCon PTH designed to enroll a minimum of 12 adult Japanese subjects.
TransCon CNP
Continued execution in the ongoing Phase 2 ACcomplisH Trial and ACcomplisH China Trial (through VISEN Pharmaceuticals) to evaluate the safety and efficacy of TransCon CNP in children ages 2-10 years with achondroplasia.
TransCon TLR7/8 Agonist
Patient enrollment continues in transcendIT-101, a Phase 1/2 study of TransCon TLR7/8 Agonist with or without pembrolizumab in patients with advanced or metastatic solid tumors.
Presenting new non-clinical data at this week’s SITC (Free SITC Whitepaper) 2021 (Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 36th annual meeting) in Washington D.C.
TransCon IL-2 β/γ
Initiated IL βelieγe ("I’ll Believe") Trial, a Phase 1/2 clinical trial to evaluate TransCon IL-2 β/γ in patients with advanced cancer.
Ended the third quarter of 2021 with cash, cash equivalents and marketable securities totaling €929.9 million.
Completed a successful public offering of American Depositary Shares raising net proceeds of approximately $436 million.
Completed $25 million share repurchase program of Ascendis’ American Depositary Shares in connection with the Company’s planned share-based incentive program. In total, 154,837 shares were repurchased with a weighted average purchase price of $161.43.
Virtual R&D update on Ascendis’ pipeline planned for mid-December.
Third Quarter 2021 Financial Results
For the third quarter, Ascendis Pharma reported a net loss of €80.3 million, or €1.47 per share (basic and diluted) compared to a net loss of €121.7 million, or €2.31 per share (basic and diluted) for the same period in 2020.

Revenue for the third quarter was €1.1 million compared to €2.8 million in the same quarter of 2020. The decrease was due to lower sale of clinical supplies to VISEN Pharmaceuticals compared to the same period last year.

Research and development (R&D) costs for the third quarter were €58.8 million compared to €64.1 million during the same period in 2020. The decline in R&D costs in 2021 reflect a one-time reversal of pre-launch inventories, which had been recognized as research and development costs in current and previous periods. The reversal of pre-launch inventories followed the U.S. FDA approval of SKYTROFA (lonapegsomatropin-tcgd) on August 25, 2021.

Selling, general and administrative expenses for the third quarter were €39.3 million compared to €17.5 million during the same period in 2020. The increase is primarily due to higher personnel-related and IT costs.

Net loss of associate for the third quarter was €3.9 million compared to a net loss of €3.1 million in the same quarter of 2020. The net loss of associate represents our share of the net result from VISEN Pharmaceuticals.

As of September 30, 2021, Ascendis Pharma had cash, cash equivalents and marketable securities of €929.9 million compared to €641.3 million as of June 30, 2021. As of September 30, 2021, Ascendis Pharma had 56,877,723 ordinary shares outstanding.

Conference Call Details

A live webcast of the conference call will be available on the Investors & News section of the Ascendis Pharma website at View Source A webcast replay will be available on the site shortly after conclusion of the event and will stay available for 30 days.

On November 10, 2021 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported financial results for the third quarter and nine months ended September 30, 2021 (Press release, RAPT Therapeutics, NOV 10, 2021, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-reports-third-quarter-2021-financial-results [SID1234595100]).

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"This has been an important year for RAPT as we advance the development of RPT193 in atopic dermatitis and FLX475 in cancer," said Brian Wong, M.D., Ph.D., President and Chief Executive Officer of RAPT Therapeutics. "In the third quarter, we presented additional incremental data for RPT193 at two separate dermatology meetings, allowing us to share our early positive data in atopic dermatitis with key members of the medical community. We are well positioned to advance RPT193 into Phase 2 clinical trials in atopic dermatitis and asthma in 2022. In addition, we have begun to focus development of FLX475 in key indications showing early promise, including EBV+ lymphoma, nasopharyngeal cancer and head and neck cancer. Our goal is to report data from ongoing cohorts in the Phase 1/2 trial for FLX475 at a medical meeting in 2022."

Financial Results for the Third Quarter Ended September 30, 2021

Third Quarter ended September 30, 2021

Net loss for the third quarter of 2021 was $18.7 million, compared to $14.6 million for the third quarter of 2020.

Research and development expenses for the third quarter of 2021 were $15.7 million, compared to $12.9 million for the same period in 2020. This increase was primarily due to increased clinical trial costs for FLX475 and RPT193 and increases in stock-based compensation, personnel costs and facilities costs.

General and administrative expenses for the third quarter of 2021 were $3.8 million, compared to $3.2 million for the same period of 2020. The increase was primarily due to increases in stock-based compensation expense, insurance expense, personnel costs and facilities costs.

Nine Months Ended September 30, 2021

Net loss for the nine months ended September 30, 2021 was $51.3 million, compared to $40.2 million for the same period in 2020.

Research and development expenses for the nine months ended September 30, 2021 were $42.7 million, compared to $34.6 million for the same period in 2020. The increase was primarily due to increases in costs related to the clinical trials of FLX475 and RPT193, as well as increases in stock-based compensation, personnel expenses, facilities costs and laboratory supplies spend.

General and administrative expenses for the nine months ended September 30, 2021 were $11.5 million, compared to $9.3 million for the same period of 2020. The increase in general and administrative expenses was primarily due to increases in stock-based compensation expense, insurance expense and personnel costs.

As of September 30, 2021, the Company had cash and cash equivalents and marketable securities of $210.8 million.