On November 2, 2022 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported financial results for the quarter ended September 30, 2022 (Press release, Allogene, NOV 2, 2022, View Source [SID1234622856]).

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The Company also announced an in-person and virtual Research & Development Showcase on Tuesday, November 29, 2022 in New York City. This event will discuss the Company’s CD19 program and data supporting the launch of ALPHA2, the first allogeneic CAR T Phase 2 clinical trial as well as updated data from the UNIVERSAL trial with single dose ALLO-715 and next steps for the program.

"We are incredibly proud of our teams and their strong execution which have enabled an industry first – the initiation of our potentially pivotal Phase 2 ALPHA2 trial," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our leading AlloCAR T programs ALLO-501A and ALLO-715 directed at CD19 and BCMA, respectively, have made substantial progress in 2022. We look forward to providing a comprehensive update on this progress at our upcoming R&D Showcase at the end of the month."

Pipeline Updates

CD19 Program

Allogene has initiated the industry’s first potentially pivotal allogeneic CAR T Phase 2 clinical trial of ALLO-501A (ALPHA2 trial) in patients with relapsed/refractory (r/r) large B-cell lymphoma (LBCL). The single-arm trial will utilize a single dose of ALLO-501A at 120 million CAR+ cells with a lymphodepletion regimen (FCA90) comprised of fludarabine (30 mg/m2/day x 3 days) and cyclophosphamide (300 mg/m2/day x 3 days) plus ALLO-647 (90 mg). The ALPHA2 trial will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. The primary endpoint of this trial is overall response rate (ORR) and the key secondary endpoint is duration of response (DoR).

The Company is in the process of initiating the EXPAND trial, a separate registrational trial that is intended to demonstrate the contribution of ALLO-647 to the standard fludarabine and cyclophosphamide lymphodepletion regimen. Patients will be randomized to receive the same single 120 million cell dose of ALLO-501A as in the ALPHA2 trial and either lymphodepletion with fludarabine and cyclophosphamide alone (control arm) or the same lymphodepletion regimen of the ALPHA2 trial (active arm). The trial is expected to enroll approximately 70 patients with r/r LBCL. The primary endpoint of this trial is progression free survival, and the key secondary endpoints are ORR, DoR, and the safety of ALLO-647.

Allogene’s Alloy manufacturing process will be deployed in the ALPHA2 and EXPAND trials. Updated clinical data from the CD19 program will be provided at the Company’s R&D Showcase on November 29, 2022.

BCMA Program

The Phase 1 UNIVERSAL trial of ALLO-715 continues enrolling patients with r/r multiple myeloma (MM). The Company will provide a clinical update from UNIVERSAL focused on a single dose ALLO-715 and discuss next steps for the program at the R&D Showcase.

Solid Tumor Program

ALLO-316, the Company’s first AlloCAR T candidate for solid tumors, targets CD70 and is being studied in patients with advanced or metastatic clear cell renal cell carcinoma (RCC) in the Phase 1 TRAVERSE trial.

Corporate Highlights

CAR T Together

The Company launched CAR T Together, a first-of-its-kind effort comprised of leading clinical trial investigators who represent the field of clinicians committed to supporting the development of off-the-shelf CAR T products to make CAR T therapy scalable and more accessible to patients with certain cancers. CAR T Together was created in response to several real-world access challenges that have emerged since the commercial introduction of autologous CAR T five years ago, highlighted by a new survey1 of U.S. based academic centers specializing in the administration of CAR T. This survey found that 82% of respondents agreed that CAR T therapies have changed how they manage aggressive cancers, but extensive wait times and manufacturing limitations keep many eligible patients from receiving treatment. Additional survey results revealed:

Only half of late-stage cancer patients who are eligible for currently FDA approved autologous CAR T therapies receive treatment.
Of those patients eligible for treatment, only 12% are able to receive treatment within one month, with approximately 40% waiting three to six months or longer to receive treatment as their disease worsens.
For eligible patients, disease progression, manufacturing capacity and comorbidities were the top barriers.
Increased patient demand, manufacturing capacity and time to treatment are cited by respondents as the three biggest challenges facing CAR T adoption in the future.
CAR T Together aims to support innovation and bring awareness to clinical trials that may ultimately lead to the availability of an allogeneic CAR T product for patients. Learn more at www.CARTTogether.com.

Allogene-Overland Biopharm

Allogene Overland Biopharm (Allogene Overland), the Company’s joint venture with Overland Pharmaceuticals, announced the completed buildout of a new, commercializable manufacturing facility in Shanghai, China. Allogene Overland is focused on the development, manufacturing and commercialization of AlloCAR T therapies for patients in greater China, Taiwan, South Korea and Singapore. The joint venture has exclusive license to develop, manufacture and commercialize specific Allogene candidates targeting BCMA, CD70, FLT3, and DLL3 in the licensed territories.

Third Quarter Financial Results

Research and development expenses were $63.6 million for the third quarter of 2022, which includes $11.0 million of non-cash stock-based compensation expense.
General and administrative expenses were $18.9 million for the third quarter of 2022, which includes $10.1 million of non-cash stock-based compensation expense.
Net loss for the third quarter of 2022 was $83.1 million, or $0.58 per share, including non-cash stock-based compensation expense of $21.1 million.
The Company had $637 million in cash, cash equivalents, and investments as of September 30, 2022.
2022 Financial Guidance
The Company expects full year GAAP Operating Expenses to be slightly below the low end of its prior guidance of $360 million and $390 million, including estimated non-cash stock-based compensation expense of $90 million to $100 million and excluding any impact from potential business development activities. Cash burn for 2022 is expected to be less than $250 million.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On November 2, 2022 Nanopharmaceutics, Inc., a clinical-stage pharmaceutical development company, reported completion of patient enrollment in the 450 patient Phase 3 clinical study sponsored by the NCI, part of the National Institutes of Health, and led by NCI funded network group, NRG Oncology, with participation of the NCI National Clinical Trials Network (NCTN), "A Randomized Phase III Trial of Radiation Therapy and Cisplatin Alone or in Combination with Intravenous Triapine in Women with Newly Diagnosed Bulky Stage IB2, Stage II, IIIB, or IVA Cancer of the Uterine Cervix or Stage II-IVA Vaginal," (NRG-GY006, ClinicalTrials.gov Identifier: NCT02466971) (Press release, Nanopharmaceutics, NOV 2, 2022, View Source;301666297.html [SID1234622847]). The randomized phase 3 trial is evaluating radiation therapy and cisplatin with Nanopharmaceutics’ proprietary formulation of Triapine compared to the standard radiation therapy and cisplatin, alone, in treating patients with newly diagnosed stage IB2, II, or IIIB-IVA cervical cancer or stage II-IVA vaginal cancer. Radiation therapy uses high energy protons to kill tumor cells and shrink tumors.

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Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, stopping them from dividing, or by stopping them from spreading. Triapine may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether radiation therapy and cisplatin are more effective with Triapine Injection in treating cervical or vaginal cancer. 450 patients were enrolled in the study, which was performed under a Cooperative Research and Development Agreement (CRADA) between NCI and Nanopharmaceutics.

About Triapine

Triapine is a synthetic heterocyclic carboxaldehyde thiosemicarbazone with potential antineoplastic activity being studied in the treatment of cancer. It is a type of ribonucleotide reductase inhibitor. Also called 3-aminopyridine-2-carboxaldehyde thiosemicarbazone and 3-AP, Triapine inhibits the enzyme ribonucleotide reductase, resulting in the inhibition of the conversion of ribonucleoside diphosphates to deoxyribonucleotides necessary for DNA synthesis.

On November 2, 2022 Curbside Health reported an official partnership with Memorial Sloan Kettering Cancer Center (MSK), with a plan to fully deploy its proprietary and scalable, clinical pathway platform in multiple aspects of MSK’s day-to-day clinical practice (Press release, Memorial Sloan-Kettering Cancer Center, NOV 2, 2022, View Source [SID1234622846]).

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MSK is among the highest-regarded, leading cancer centers in the world, and as such, the Curbside team is delighted to deliver industry-leading tools and resources to support MSK’s gold-standard.

Curbside is currently offering tailored consultations to qualified hospital groups and institutions.
Curbside is currently offering tailored consultations to qualified hospital groups and institutions.
For MSK, Curbside enhances digital tools and resources available to the clinical staff, while providing detailed clinical information and decision support. Based on similar instances, the Curbside team expects the usability goes up since Curbside is integrated inside of the Electronic Health Records (EHR), allowing the latest available, and approved pathways are delivered right at the point of care.

At a very granular level, Curbside will help MSK disseminate case-specific content to other sites so that a patient with a challenging cancer diagnosis, as an example, could still significantly benefit from world-class decision-making even if they’re not at MSK.

Regarding the complete solution from Curbside, Co-Founder Eric Leroux, MD MBA states, "The platform solution for MSK is all-inclusive, incorporating all of the content creation, maintenance, management access, analytics, reporting, it’s all in a single place. Curbside is where you go for any sort of clinical content you need to get pathways, guidelines, protocols, clinical calculators, and so on," he continues, "we see the usability go way up for an organization such as MSK".

Among Curbside’s core value propositions is that it provides a robust ecosystem helping to ensure patients are not limited in the quality of care they receive based on where they have to live or receive care — it’s a democratization of clinical pathways on all fronts.

Dr. Leroux adds, "It really starts to break down barriers to highest quality of care and that’s an important theme for Curbside and actually the reason for the company’s name — Curbside Health."

On November 2, 2022 Photocure ASA (OSE:PHO) reported Hexvix/Cysview revenues of NOK 96.9 million in the third quarter of 2022, up 12% compared to Q3 2021 (NOK 86.7 million), and positive EBITDA of NOK 4.7 million (NOK -0.2 million) for the Company (Press release, PhotoCure, NOV 2, 2022, View Source [SID1234622845]). With the launch of Karl Storz’s new high-definition blue light system in the U.S., Photocure expects that placements of new blue light rigid towers will begin to accelerate in the fourth quarter of this year. A recent ruling by the Centers for Medicare and Medicaid Services (CMS) improves U.S. reimbursement for Cysview, expanding access in the Ambulatory Surgery Center (ASC) segment.

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"Photocure delivered Hexvix/Cysview revenue growth of 12%, driven by higher unit sales in both North America and Europe in addition to the net favorable effect of foreign exchange. I am very pleased to report that our North American business segment achieved the highest quarterly unit sales volume in our history, and we are now in a strong position to significantly expand the installed base of blue light capital equipment in the U.S. and get back to the high growth trajectory that we were achieving before the pandemic," says Daniel Schneider, President & Chief Executive Officer of Photocure.

Photocure reported total group revenues of NOK 106.8 million in the third quarter of 2022 (NOK 87.4 million), and EBITDA* of NOK 4.7 million (NOK -0.2 million), driven by the increase in product revenue and a milestone payment from Asieris. Hexvix/Cysview revenues ended at NOK 96.9 million in the quarter (Q3 2021: NOK 86.7 million), due to higher unit sales in both North America and Europe, and net favorable foreign exchange. EBIT was NOK -1.4 million (-6.3 million) and the cash balance at the end of Q3 2022 was NOK 283.9 million.

Photocure announced the commercial availability of Karl Storz’s new high-definition blue light rigid system in the United States late in the quarter. The Company reports that 10 new blue light towers were installed during the period; 6 new Saphira cystoscopes and 4 flexible units. The installed base of blue light cystoscopes in the U.S. was 339 (302) at the end of the third quarter, a 12% increase from the same period last year. This includes 62 flexible cystoscopes, an increase of 38%.

"The pipeline for new blue light cystoscopy towers remains stronger than ever. With the new system now officially launched, we are currently assisting Karl Storz with the placement and activation of towers for accounts that participated in the obsolescence protection program to replace the old standard definition system. We anticipate broader installations of rigid BLC equipment in new accounts this quarter," Schneider adds.

With the launch of Karl Storz’s new high-definition blue light system in the U.S. at the end of the third quarter 2022, Photocure expects that placements of new blue light rigid towers will accelerate in the fourth quarter of this year and beyond. Additionally, the post-period ruling by CMS to improve Medicare reimbursement is anticipated to help increase sales in the Hospital-Based Outpatient Department (HOPD) setting and enable Photocure’s U.S. commercial team to penetrate accounts in the ASC site of care.

"With account access now open in our major markets, Karl Storz’s new blue light system launching, and an improvement in Medicare access and reimbursement expected to take effect January 1 of next year, I believe that Photocure’s business is at an inflection point. We remain focused on positioning the Hexvix/Cysview franchise as the standard of care in the management of bladder cancer," Schneider concludes.

Please find the full financial report and presentation enclosed.

EBITDA* and other alternative performance measures (APMs) are defined and reconciled to the IFRS financial statements as a part of the APM section of the third quarter 2022 financial report on page 22.

The quarterly report and presentation will be published at 08:00 CET and will be publicly available at www.photocure.com. Daniel Schneider, CEO and Erik Dahl, CFO, will host a live webcast at 14:00 CET.

The presentation will be held in English and questions can be submitted throughout the event. The streaming event is available through https://channel.royalcast.com/hegnarmedia/#!/hegnarmedia/20221102_7

On November 2, 2022 Omega Therapeutics, Inc. ("Omega"), a clinical-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OTX-2002, the company’s first-in-class epigenomic controller engineered to downregulate c-Myc (MYC), for the treatment of hepatocellular carcinoma (HCC) (Press release, Omega Therapeutics, NOV 2, 2022, View Source [SID1234622844]).

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The company recently announced that the first patient had been dosed in its Phase 1/2 MYCHELANGELO I clinical trial investigating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy and in combination with standard of care therapies in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene.

"HCC is a devastating illness that often develops resistance to current standard of care therapeutics. The FDA’s decision to grant orphan drug designation for OTX-2002 underscores the need for novel therapies to address HCC and the potential of epigenomic programming to transform the treatment landscape," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "We look forward to continuing to work with clinical investigators, patients and the FDA as we advance our MYCHELANGELO clinical program and evaluate the potential of OTX-2002 to bring a new treatment option to the liver cancer patient community."

About Orphan Drug Designation
The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is a leading cause of cancer deaths worldwide and represents an unmet clinical need with few therapeutic options. Tyrosine kinase inhibitors (TKIs) have been used as a systemic therapy for HCC, but patients frequently develop resistance with oncogenic MYC identified as a correlating prognostic factor. The MYC oncogene is associated with aggressive disease in up to 70% of patients with HCC.

About OTX-2002
OTX-2002 is a first-in-class Omega Epigenomic Controller in development for the treatment of hepatocellular carcinoma (HCC). OTX-2002 is an mRNA therapeutic delivered via lipid nanoparticles (LNPs) and is designed to downregulate MYC expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. MYC is a master transcription factor that regulates cell proliferation, differentiation and apoptosis and plays a significant role in more than 50% of all human cancers. OTX-2002 is currently being evaluated in the Phase 1/2 MYCHELANGELO I trial in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene; visit clinicaltrials.gov (NCT05497453) for more details.