Lyell Immunopharma to Participate in Credit Suisse Healthcare Conference

On November 2, 2022 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical‑stage T-cell reprogramming company dedicated to developing curative cell therapies for patients with solid tumors, announced today that members of its senior management team will present at the Credit Suisse 31st Annual Healthcare Conference Wednesday, November 9, at 2:00 p.m. ET (Press release, Lyell Immunopharma, NOV 2, 2022, View Source [SID1234622863]).

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A live webcast of the presentation can be accessed through the investor relations section of the Company’s website at www.lyell.com. A replay of the webcast will be available on the Company’s website for 90 days following the presentation.

Ascendis Pharma Reports Third Quarter 2022 Financial Results

On November 2, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the third quarter ended September 30, 2022 and provided a business update (Press release, Ascendis Pharma, NOV 2, 2022, View Source [SID1234622858]).

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"With our improved commercial execution and emphasis on SKYTROFA’s unique patient-focused product strengths, I believe we are advancing our goal to build SKYTROFA into the leading brand and expand the pediatric growth hormone market," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "With our expanding global commercial activities, potential U.S. approval of TransCon PTH in April 2023, and new data expected in achondroplasia and oncology, I am confident in our ability to deliver multiple high-value products to drive long-term sustainable growth."

Select Highlights

TransCon hGH:
SKYTROFA (lonapegsomatropin-tcgd) U.S. revenue more than doubled quarter-to-quarter again to €12.3 million in the third quarter. As of September 30, 2022, the cumulative number of new patient prescriptions increased to over 2,400.
On track to complete enrollment in the global Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD) during the fourth quarter of 2022.
Endocrinology Rare Disease commercial center for Germany planned in Munich; Skytrofa (lonapegsomatropin) launch in Germany expected mid-2023.
TransCon PTH:
U.S. FDA accepted for Priority Review NDA submission in adult patients with hypoparathyroidism; PDUFA target action date is April 30, 2023.
MAA submission to EMA on track for the fourth quarter of 2022.
Topline results from Phase 3 PaTHway Japan Trial expected during fourth quarter of 2022.
After more than two years of treatment, 57 out of 59 patients continue in the open-label extension portion of the Phase 2 PaTH Forward Trial as of September 30, 2022.
In the Phase 3 PaTHway Trial, 79 out of 79 patients completed one-year follow-up; 77 out of 79 patients continue in the open-label extension portion of the trial as of September 30, 2022.
TransCon CNP:
Topline and initial open-label extension data from ACcomplisH, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in children ages 2-10 years with achondroplasia, expected during the fourth quarter of 2022; 57 out of 57 patients continue in the open-label portion of the trial as of September 30, 2022.
Submitted protocol to initiate ApproaCH, a new global randomized, double-blind, placebo-controlled Phase 2b trial in children ages 2-11 years with achondroplasia. The trial is expected to enroll approximately 80 patients.
TransCon TLR7/8 Agonist:
Completed the dose-escalation portion and announced selection of recommended Phase 2 dose in transcendIT-101. Topline data accepted for oral presentation at SITC (Free SITC Whitepaper) 2022, the annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) being held in Boston November 8-12.
The next phase of transcendIT-101 will evaluate the recommended Phase 2 dose of investigational TransCon TLR7/8 Agonist in combination with pembrolizumab in patients in four different indication-specific cohorts: head and neck squamous cell carcinoma (HNSCC); other human papillomavirus (HPV)-associated cancers; melanoma; and cutaneous squamous cell carcinoma (cSCC).
TransCon IL-2 β/γ
The Phase 1/2 IL-βelieγe Trial evaluating investigational TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. The Phase 1/2 IL-βelieγe Trial topline data from monotherapy dose escalation are expected during the fourth quarter of 2022.
Ended the third quarter of 2022 with cash, cash equivalents, and marketable securities totaling €935 million.
Third Quarter 2022 Financial Results

Total revenue for the third quarter was €15.3 million compared to €1.1 million in the same quarter of 2021. Revenue included U.S. revenue from SKYTROFA, as well as license, clinical supply and services provided to third parties, primarily VISEN Pharmaceuticals. The increase in revenue compared to the same period last year was primarily attributable to the €12.3 million commercial revenue from SKYTROFA.

Research and development (R&D) costs for the third quarter were €97.4 million compared to €58.8 million during the same period in 2021. Lower R&D costs in the third quarter of 2021 reflect a one-time €53.7 million reversal of pre-launch inventories, which had previously been recognized as research and development costs. The third quarter 2022 R&D costs also reflect the manufacturing of pre-launch inventories for TransCon PTH and an increase in employee and other costs attributable to organizational growth.

Selling, general, and administrative (SG&A) expenses for the third quarter were €60.7 million compared to €39.3 million during the same period in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel following the launch of SKYTROFA in the U.S. and preparation for future product launches.

Our share of net loss of associate was €3.7 million in the third quarter, compared to a net loss of €3.9 million during the same period in 2021.

Net finance loss was €20.9 million in the third quarter compared to a net finance income of €20.4 million in the same period in 2021.

For the third quarter of 2022, Ascendis Pharma reported a net loss of €169.0 million, or €3.03 per share (basic and diluted) compared to a net loss of €80.3 million, or €1.47 per share (basic and diluted) for the same period in 2021.

As of September 30, 2022, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €935.1 million compared to €789.6 million as of December 31, 2021. As of September 30, 2022, Ascendis Pharma had 57,027,240 ordinary shares outstanding.

Conference Call and Webcast Information

Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its third quarter 2022 financial results.

Those who would like to listen to the live webcast can access it through the following link here. To access the live teleconference, register online here. Participants are encouraged to register at least 15 minutes prior to the call.

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

Allogene Therapeutics Reports Third Quarter 2022 Financial Results and Announces Investor R&D Showcase

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.

Nanopharmaceutics, Inc. Announces the End of Patient Recruitment for the National Cancer Institute (NCI) Sponsored Phase 3 Clinical Study in Advanced-Stage Cervical and Vaginal Cancers with Cisplatin During Radiation Therapy with and without Triapine®

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.

Memorial Sloan Kettering Cancer Center Partners with Curbside Health to Advance Clinical Pathways in Oncology

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."