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.

On November 10, 2021 Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, reported that its senior management will present and participate in 1-on-1 meetings with institutional investors at the Jefferies London Healthcare Conference on November 16 and 17, 2021 (Press release, Valneva, NOV 10, 2021, View Source [SID1234595079]).

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Valneva’s Chief Executive Officer Thomas Lingelbach and acting Chief Financial Officer David Lawrence will notably discuss the Company’s late stage vaccine candidates against Lyme disease (VLA15), chikungunya (VLA1553) and COVID-19 (VLA2001).

Valneva recently reported positive Phase 3 results for VLA20011, currently the only whole virus, inactivated, adjuvanted vaccine candidate against COVID-19 in clinical trials in Europe. The Company also announced last week the closing of a $102.0 million Global Offering2.

Valneva’s presentation will take place on November 16, 2021 at 4:20pm GMT and can be accessed via the following link: View Source To request a meeting at the event, please contact your representative at Jefferies.

On November 10, 2021 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat urothelial and specialty cancers, reported at its virtual Spotlight Event being held today that, following recent discussions with the U.S. Food and Drug Administration ("FDA"), it plans to conduct a new, single-arm Phase 3 pivotal study of UGN-102 for the treatment of low-grade, intermediate-risk, non-muscle invasive bladder cancer ("NMIBC") (Press release, UroGen Pharma, NOV 10, 2021, View Source [SID1234595078]). This new study, which is expected to initiate in early 2022, is expected to enroll approximately 220 patients across 90 sites.

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"We have worked diligently with the FDA over the past several years to define the unmet need in low-grade NMIBC, with a particular focus on the intermediate risk population that typically experiences multiple recurrences," said Liz Barrett, President and Chief Executive Officer of UroGen. "We are preparing to initiate a single-arm pivotal study of UGN-102 to form the basis for a New Drug Application for UGN-102 in the treatment of low-grade, intermediate-risk NMIBC. We believe this new study increases the probability of regulatory success for UGN-102 given its streamlined design in addition to the encouraging results observed from our Phase 2 OPTIMA II study."

UroGen is grateful to the investigators and patients who are participating in the ATLAS study and believes the data generated will represent an important component of the planned UGN-102 NDA submission, which remains on track for 2024. In light of the new planned Phase 3 trial, UroGen will stop enrollment in the ATLAS trial. Patients already enrolled in ATLAS will have the option to remain in the study until completion.

In addition to the updated clinical plan for UGN-102, the Spotlight Event features presentations on UroGen’s earlier-stage, locally administered immunotherapy candidates, UGN-301 and UGN-201, as well as two expert panel discussions on future directions in treating NMIBC, other types of bladder cancer and the potential benefits of UroGen’s immunotherapy pipeline products in non-urologic cancers.

Distinguished panelists discussing the current treatment landscape and potential future innovations in NMIBC include:

Dr. Sandip Prasad, a urologist in community practice with Atlantic Medical Group and the Morristown Medical Center;
Dr. Gary Steinberg, Professor of Urology and Director of the Bladder Cancer Program at NYU Langone Medical Center; and
Dr. William Huang, Professor of Urology and Radiology at NYU and Chief of Urology at Tisch Hospital.
The experts reviewing the unmet needs of patients with high-grade NMIBC and the potential for locally applied combination therapy include:

Dr. Karim Chamie, Associate Professor of Urology at UCLA Medical Center; and
Dr. Joshua Meeks, Associate Professor of Urology, Biochemistry and Molecular Genetics at Northwestern University Feinberg School of Medicine.
"Immunotherapy for the treatment of high-grade bladder cancer, with primary attention toward our TLR-7 agonist, UGN-201 and our anti-CTLA4 antibody, UGN-301, is a key area of focus of our earlier-stage pipeline," said Dr. Mark Schoenberg, Chief Medical Officer of UroGen. "We have pursued a series of pre-clinical studies to determine whether our RTGel platform might provide a method for delivering highly potent immunomodulators directly to the bladder surface, avoiding the toxicity associated with systemic administration. Our studies conducted to-date suggest bladder cancer treated with a combination of a TLR-7 agonist and an anti-CTLA4 antibody using our RTGel technology, produces improved survival relative to treatment with other checkpoint inhibitors in RTGel, either alone or in combination with UGN-201."

UroGen is currently conducting non-human primate toxicity studies to facilitate the initiation of a multi-arm Phase 1 study of UGN-301 in early 2022 to be followed by UGN-301 in combination with other agents. This approach leverages UroGen’s unique platform for drug delivery and provides an opportunity to evaluate intravesical delivery of its anti-CTLA4 monoclonal antibody in combination with other immuno-modulators, chemotherapies, gene therapy and innate immune stimulators, including UGN-201.