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 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period ended September 30, 2021 and provided a corporate update (Press release, Spectrum Pharmaceuticals, NOV 10, 2021, View Source [SID1234595083]).

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"The submission of the poziotinib NDA this quarter remains our top corporate priority. The front line data presented at ESMO (Free ESMO Whitepaper) and the preclinical combination data with KRAS inhibitors at the Triple meeting has the potential to significantly expand the poziotinib opportunity," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "Following a productive Type A meeting with the FDA on ROLONTIS, we anticipate the remediation efforts at Hanmi to be completed by the end of the year and would expect to resubmit our BLA for ROLONTIS shortly thereafter."

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Submission of the NDA, based on the positive results of Cohort 2 in patients with previously treated locally advanced or metastatic non-small cell lung cancer (NSCLC) with HER2 exon 20 insertion mutations is on track for this year under a Fast Track designation. There is currently no treatment specifically approved for this indication.
Encouraging results from Cohort 4 of the ZENITH20 clinical trial were presented at the European Society of Medical (ESMO) (Free ESMO Whitepaper) Congress 2021. The primary endpoint of objective rate of response (ORR) was 44% (95% CI:29.5-58.8%) in the first 48 treated patients including one complete response. 88% of patients showed tumor reduction with a disease control rate of 75%. Median duration of response was 5.4 months (range 2.8-19.1+). Median progression free survival was 5.6 months (range 0-20.2+). The most common treatment related Grade ≥ 3 adverse effects (AEs) were rash, stomatitis, diarrhea, and paronychia. In addition, only one patient experienced Grade ≥ 3 pneumonitis. Poziotinib demonstrated clinically meaningful anti-tumor activity in newly diagnosed NSCLC patients with HER2 exon 20 mutations with 16mg QD dosing. The safety profile was manageable and similar to those observed in previous studies and other second-generation tyrosine kinase inhibitors.
Preclinical data showed the synergistic impact of poziotinib when combined with KRAS inhibitors in KRASG12C mutant specific cell lines. Jacqulyne Robichaux, Ph.D., Assistant Professor, University of Texas, MD Anderson Cancer Center presented a poster titled "Pan-ErbB inhibition enhances activity of KRASG12C inhibitors in preclinical models of KRASG12C mutant cancers" at the Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) hosted by the American Association for Cancer Research (AACR) (Free AACR Whitepaper), the National Cancer Institute (NCI), and the European Organization for Research and Treatment of Cancer (EORTC). The preclinical data showed that inhibition of EGFR, HER2, HER3, and HER4 signaling was synergistic when combined with KRASG12C inhibitors. These results highlight the importance of a pan inhibitor of the Erb family of proteins.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

The company held a Type A meeting with the U.S. Food and Drug Administration (FDA) to better understand the issues identified in the Complete Response Letter (CRL). At that meeting, the company learned that the deficiencies at the fill finish site have been adequately addressed. Remediation of deficiencies at the drug substance facility are well under way and expected to be completed by the end of the year. The FDA confirmed that the reinspection of the drug substance facility would be in-person.
Three-Month Period Ended September 30, 2021 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $33.1 million, or $0.21 loss per basic and diluted share, in the three-month period ended September 30, 2021, compared to a net loss of $48.5 million, or $0.37 loss per basic and diluted share, in the comparable period in 2020. Total research and development expenses were $20.9 million in the quarter, as compared to $24.5 million in the same period in 2020. Selling, general and administrative expenses were $12.2 million in the quarter, compared to $15.1 million in the same period in 2020.

The company ended the quarter with cash, cash equivalents, and marketable securities of $133.6 million.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $25.8 million, or $0.16 loss per basic and diluted share, in the three-month period ended September 30, 2021, compared to a non-GAAP net loss of $35.2 million, or $0.27 loss per basic and diluted share, in the comparable period in 2020. Non-GAAP research and development expenses were $16.7 million, as compared to $23.3 million in the same period of 2020. Non-GAAP selling, general and administrative expenses were $9.2 million, as compared to $12.3 million in the same period in 2020.

Conference Call

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website View Source on November 10, 2021 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On November 10, 2021 Celyad Oncology SA (Euronext & Nasdaq: CYAD) (the "Company"), a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T cell (CAR T) therapies for cancer, reported an update on its financial results and recent business developments for the fiscal quarter ended September 30, 2021 (Press release, Celyad, NOV 10, 2021, View Source [SID1234595082]).

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"Our team continues to take major strides towards the development of a differentiated, next-generation CAR T pipeline based on novel non-gene edited technologies which we believe offer potential advantages over gene-editing approaches. During our R&D Day in the third quarter, we provided an in-depth review of our platforms, including our proprietary shRNA and TIM technologies, as well as our ‘armored’ CAR franchise focused on the co-expression of secreting IL-18. These key capabilities are reflected across our allogeneic clinical programs and highlight the advancements we’re making. Overall, we’ve treated more than 30 patients with our allogeneic CAR T product candidates without evidence of GvHD while observing encouraging early signals of clinical activity, which we believe is a promising validation that our non-gene edited strategy for allogeneic CAR T has the potential to be a game-changer for the field," commented Filippo Petti, Chief Executive Officer of Celyad Oncology. "There are several data readouts scheduled at this year’s ASH (Free ASH Whitepaper) and SITC (Free SITC Whitepaper) annual meetings, and we believe the updates from the Phase 1 IMMUNICY-1 trial of CYAD-211, as well as the pending initiation of the Phase 1b KEYNOTE-B79 trial will further position us as leaders in the allogeneic CAR T space."

Recent Highlights

Research & Development Day held on July 20, 2021, during which the management team provided:
Updates on the allogeneic CAR T clinical candidates CYAD-211 and CYAD-101.
Highlights from the latest research from the Company’s proprietary short hairpin RNA (shRNA) platform, including the introduction of CYAD-203 – a novel allogeneic, Interleukin-18 (IL-18)-armored CAR T candidate currently in IND-enabling studies.
Acquisition of an exclusive patent license from the University of Pennsylvania for an engager targeting Glypican 3 (GPC3), which will be tested with the Company’s proprietary shRNA technology platform.
Three abstracts to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting, which will include information about:
Celyad Oncology’s multiplexing capabilities using shRNA technology.
The Company’s first non-gene edited allogeneic, IL-18-armored CAR T candidate CYAD-203.
Phase 1b KEYNOTE-B79 trial of CYAD-101 followed by KEYTRUDA (pembrolizumab) in refractory metastatic colorectal cancer patients.
Two abstracts to be presented at the 63rd ASH (Free ASH Whitepaper) Annual Meeting to be held from December 11-14, 2021, which will include additional data for CYAD-211 and CYAD-02.
Third Quarter 2021 Financial Review

As of September 30, 2021, the Company had cash and cash equivalents of €6.1 million ($7.1 million), representing a decrease in cash and cash equivalents of €5.9 million as compared to June 30, 2021. The Company believes that its existing cash and cash equivalents combined with the remaining access to the equity purchase agreement established with Lincoln Park Capital Fund, LLC should be sufficient, based on the current scope of activities, to fund operating expenses and capital expenditure requirements to the end of the third quarter of 2022.

Update on Clinical and Preclinical Programs

CYAD-101 – Allogeneic TIM-based, NKG2D CAR T for mCRC

CYAD-101 is the Company’s allogeneic CAR T candidate engineered to co-express a chimeric antigen receptor (CAR) based on the NKG2D receptor and the novel inhibitory peptide TCR Inhibitory Molecule (TIM).
To the Company’s knowledge, CYAD-101 is the first investigational allogeneic CAR T candidate to generate evidence of clinical activity for the treatment of a solid tumor indication.
The Phase 1b KEYNOTE-B79 trial, which will evaluate CYAD-101 following FOLFOX preconditioning chemotherapy, with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in refractory mCRC patients with microsatellite stable (MSS) / mismatch-repair proficient (pMMR) disease, is on track to begin by year-end 2021.
This KEYNOTE-B79 trial is part of a clinical trial collaboration involving Celyad Oncology and MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., USA, through a subsidiary.
CYAD-211 – Allogeneic shRNA-based, anti-BCMA CAR T for r/r MM

CYAD-211 comprises a B cell maturation antigen (BCMA) targeting CAR co-expressed with a shRNA that targets the CD3ζ component of the T-cell receptor (TCR) complex.
The dose-escalation, Phase 1 IMMUNICY-1 trial is evaluating the tolerability and clinical activity of a single infusion of CYAD-211 following preconditioning with cyclophosphamide and fludarabine given for three consecutive days.
In July 2021, preliminary data from the Phase 1 IMMUNICY-1 trial was presented from a total of seven patients (three patients at dose level one, three patients at dose level two and one patient at dose level three) demonstrating no evidence of Graft-versus-Host disease (GvHD) along with initial evidence of clinical response in the form of two partial responses. Importantly, CYAD-211 cells were detected within the peripheral blood of all patients analyzed with an apparent stepwise engraftment across the three dose levels.
Enrollment in the trial is ongoing with plans to explore higher doses of preconditioning regimens in future cohorts. A clinical update will be presented at the upcoming ASH (Free ASH Whitepaper) meeting in December 2021.
CYAD-203 – Allogeneic shRNA-based, IL-18-armored NKG2D CAR T for Solid Tumors

CYAD-203 is the Company’s first armored CAR T candidate engineered to co-express the cytokine IL-18 with the NKG2D CAR receptor. To the Company’s knowledge, CYAD-203 is the first allogeneic IL-18 secreting CAR T candidate.
Investigational New Drug (IND)-enabling studies are currently in progress alongside production of the clinical grade vector.
Submission of the IND application for CYAD-203 is expected in mid-2022.
At the SITC (Free SITC Whitepaper) 36th Annual Meeting, preclinical data will be presented demonstrating the enhanced anti-tumor activity of NKG2D CAR T cells armored with IL-18.
CYAD-02 – Autologous NKG2D receptor CAR T for r/r AML / MDS

CYAD-02, the Company’s autologous CAR T candidate with shRNA technology that targets the NKG2D ligands MICA and MICB, is currently being evaluated for the treatment of r/r AML / MDS in the Phase 1 CYCLE-1 dose-escalation trial.
Additional safety and efficacy data from the trial will be presented at the upcoming ASH (Free ASH Whitepaper) meeting in December 2021.
Upcoming Anticipated Milestones

Initiation of the Phase 1b KEYNOTE-B79 trial evaluating CYAD-101 with KEYTRUDA in mCRC patients with MSS/pMMR disease is anticipated by year-end 2021.
Additional data from the Phase 1 IMMUNICY-1 trial of CYAD-211 and the Phase 1 CYCLE-1 trial of CYAD-02 are expected at the upcoming ASH (Free ASH Whitepaper) meeting in December 2021.
Submission of the IND application for CYAD-203 for treatment of solid tumors is expected in mid-2022.

On November 10, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel irreversible small molecules to treat and improve the lives of patients with genetically defined cancers, reported that Thomas Butler, Chief Executive Officer and Chairman of the Board, will participate in two upcoming virtual conferences (Press release, Biomea Fusion, NOV 10, 2021, View Source [SID1234595081]).

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Jefferies London Healthcare Conference
Fireside chat on Thursday, November 18th at 3:00am ET

Piper Sandler 33rd Annual Virtual Healthcare Conference
Fireside chat on Monday, November 22nd at 10:00am ET

Biomea Fusion will participate in 1×1 investor meetings during the conferences hosted by Jefferies and Piper Sandler, respectively. Video webcasts will be available for viewing on the News & Events section of biomeafusion.com. Archived webcasts will be available for viewing for 30 and 90 days, respectively, following the webcasts.

About BMF-219

BMF-219 is an irreversibly binding inhibitor of menin, a protein that is known to play an essential role in oncogenic signaling in genetically defined leukemias. Preclinically, BMF-219 has demonstrated robust downregulation of key leukemogenic genes in addition to menin itself (via MEN1) in well-established menin dependent cell lines. Additionally, BMF-219 has shown efficacy in multiple in vivo and in vitro models of acute leukemias and lymphomas. BMF-219 will be evaluated in a first-in-human trial in patients with relapsed or refractory acute leukemia with MLL/KMT2A gene rearrangement or NPM1 mutation.

On November 10, 2021 "The U.S. FDA approval for TIVDAK represents an important milestone both for the treatment of cervical cancer as well as for Genmab as a company," reported Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab (Press release, Genmab, NOV 10, 2021, View Source [SID1234595080]). "TIVDAK is the first and only approved antibody-drug conjugate (ADC) for adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy, providing a new treatment option for patients impacted by this devastating disease. The decision by the U.S. FDA also marks the first regulatory approval for an ADC combining Genmab’s antibody with Seagen’s ADC technology and is the first approval for any therapy owned at least 50% by Genmab. This achievement was only possible because of the efforts of our dedicated and talented team, the excellent collaboration with our partner for TIVDAK, Seagen, and the patients, families and caregivers as well as the nurses, physicians and study teams who participated in our clinical trials."

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Financial Performance First Nine Months of 2021

Net sales of DARZALEX by Janssen Biotech Inc. (Janssen) were USD 4,378 million in the first nine months of 2021 compared to USD 2,937 million in the first nine months of 2020, an increase of USD 1,441 million, or 49%.
Royalty revenue was DKK 4,698 million in the first nine months of 2021 compared to DKK 3,090 million in the first nine months of 2020, an increase of DKK 1,608 million, or 52%. The increase was driven by higher net sales of DARZALEX, TEPEZZA and Kesimpta resulting in higher royalties.
Total revenue was DKK 5,863 million in the first nine months of 2021. In addition to the royalty revenue described above, Genmab also recognized DKK 794 million of milestone revenue during the first nine months of 2021. Revenue for the first nine months of 2020 was DKK 8,067 million and included the one-time upfront payment of DKK 4,398 million recognized as license revenue from AbbVie Inc. (AbbVie) pursuant to our collaboration announced in June 2020.
Operating expenses were DKK 3,654 million in the first nine months of 2021 compared to DKK 2,641 million in the first nine months of 2020. The increase of DKK 1,013 million, or 38%, was driven by the continued advancement of multiple pipeline projects, the increase in new employees to support the launch of TIVDAK and expansion of our product pipeline, as well as the continued development of commercialization capabilities and Genmab’s broader organizational infrastructure.
Operating result was DKK 2,209 million in the first nine months of 2021 compared to DKK 5,426 million in the first nine months of 2020. The decrease of DKK 3,217 million, or 59%, was driven by lower revenue as a result of the non-recurring license revenue in 2020 associated with the upfront payment from AbbVie and increased operating expenses.

Outlook
As announced in Company Announcement No. 66, Genmab is improving its 2021 financial guidance published on August 11, 2021, driven primarily by increased royalty revenue related to the net sales of DARZALEX.

Genmab will hold a conference call in English to discuss the results for the first nine months of 2021 today, Wednesday, November 10, at 6:00 pm CET, 5:00 pm GMT or 12:00 pm EST. To join the call dial
+1 631 913 1422 (U.S. participants) or +44 3333000804 (international participants) and provide conference code 90392669.

A live and archived webcast of the call and relevant slides will be available at www.genmab.com/investors.