On May 4, 2021 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune diseases, reported financial results for the first quarter 2021, recent business highlights and key catalysts over the next 18 months (Press release, Atara Biotherapeutics, MAY 4, 2021, View Source [SID1234579064]).

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"Atara is off to a strong start in 2021, advancing all three strategic priorities to deliver on key value drivers," said Pascal Touchon, President and Chief Executive Officer of Atara. "We look forward to several milestones in 2021, including the expected tab-cel BLA and MAA filings, progress on the ATA188 program – especially the presentation of clinical and translational data from the Phase 1 OLE study, and the first clinical data on our mesothelin CAR T franchise."

Tabelecleucel (tab-cel) for Post-Transplant Lymphoproliferative Disease (PTLD)

Atara is in active discussions with the FDA and progressing toward alignment on the content of CMC Module 3, including methodologies to assess comparability between the product used in the pivotal ALLELE study and the intended commercial product
A recent analysis shows that duration of response (DoR) in its ALLELE study is maturing as anticipated with a larger number of responders followed for at least six months and a safety profile consistent with previously published data with no new safety signals
Atara is working toward completing a BLA submission in Q3 2021 pending alignment with the FDA
The Company has submitted a letter of intent to the European Medicines Agency (EMA), starting the process for a submission of an EU Marketing Authorization Application (MAA) for tab-cel in patients with EBV+ PTLD expected in Q4 2021
Atara has data at two medical congresses from a combined long-term overall survival (OS) analysis from three clinical studies of tab-cel demonstrating that patients with EBV+ PTLD, following both HCT (hematopoietic cell transplantation) and SOT (solid organ transplant), that is relapsed or refractory (R/R) to initial treatment, derived similar OS benefit greater than 80 percent at two years whether they achieved complete or partial response (CR or PR) with tab-cel
Data from the Phase 3 ALLELE study will be presented at an appropriate congress in Q4 2021
The Company is continuing to invest in U.S. commercial readiness activities in anticipation of tab-cel approval and planned launch in H1 2022. In addition, Atara is in discussions with potential partners for the commercialization of tab-cel in Europe
Tab-cel for Potential Additional Indications

Atara is actively opening sites in the Phase 2 multi-cohort study for patients with other EBV-driven cancers
ATA188 for Progressive Forms of Multiple Sclerosis (MS)

Atara continues to make progress enrolling the ATA188 Phase 2 randomized, double-blind, placebo-controlled trial (RCT) evaluating the efficacy and safety of ATA188 in patients with progressive forms of MS (PMS)
Atara plans to conduct an interim analysis (IA) in H1 2022 including efficacy and safety from the Phase 2 RCT in patients with PMS, and following the IA, expects to complete enrollment of the study in H1 2022
The Company plans to present long-term, two-year clinical data from the Phase 1 open-label extension (OLE) and translational data from the Phase 1 study in H2 2021
In the first quarter of 2021, Atara filed and received approval of a Clinical Trial Application (CTA) for the Phase 2 RCT in Canada
CAR T Programs

ATA2271/ATA3271 (Solid Tumors Over-Expressing Mesothelin)

The global strategic collaboration with Bayer including ATA2271 and ATA3271 is progressing well with successful launch of joint governance and activities
Enrollment of the first cohort in the Phase 1 clinical study of ATA2271 for patients with advanced mesothelioma has completed and the Company anticipates presentation of first clinical data in an appropriate forum in Q4 2021
Atara is continuing to make progress on IND-enabling studies for ATA3271, an off-the-shelf, allogeneic CAR T therapy targeting mesothelin using a PD-1 DNR and 1XX CAR co-stimulatory signaling domain through its EBV T-cell platform, and expects an IND filing in Q2/Q3 of 2022
ATA3219 (B-cell Malignancies)

Atara expects to submit an IND for ATA3219, its next-generation off-the-shelf, allogeneic CAR T using a 1XX CAR co-stimulatory signaling domain through its EBV T-cell platform for patients with B-cell malignancies, in Q4 2021 / Q1 2022
First Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments as of March 31, 2021 totaled $435.2 million, as compared to $500.7 million as of December 31, 2020
Atara believes that its cash as of March 31, 2021 together with projected revenue from U.S. tab-cel sales is sufficient to fund its operations into 2023, including expenses related to the BLA filing and commercial launch of tab-cel in the U.S.
License and collaboration revenue was $3.6 million for the first quarter 2021 and consisted of revenue from activities performed under the Bayer Collaboration Agreements. Atara did not recognize any license and collaboration revenue for the same period in 2020
Net cash used in operating activities was $65.7 million for the first quarter 2021, as compared to $67.0 million for the same period in 2020
Atara reported net losses of $78.3 million, or $0.86 per share, for the first quarter 2021, as compared to $73.5 million, or $1.20 per share, for the same period in 2020
Total operating expenses include non-cash expenses of $14.4 million for the first quarter 2021, as compared to $14.5 million for the same period in 2020
Research and development expenses were $64.1 million for the first quarter 2021, as compared to $57.7 million for the same period in 2020
The increase in the first quarter 2021 was primarily due to higher employee-related costs from increased headcount, increased spending on the Company’s ATA188 and CAR T programs and increased facilities and information technology expenses allocated to research and development
Research and development expenses include $7.5 million of non-cash stock-based compensation expenses for the first quarter 2021, as compared to $7.7 million for the same period in 2020
General and administrative expenses were $17.7 million for the first quarter 2021, as compared to $17.0 million for the same period in 2020
General and administrative expenses include $4.7 million of non-cash stock-based compensation expenses for the first quarter 2021, as compared to $5.0 million for the same period in 2020

On May 4, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that the company will participate at the BofA Securities 2021 Virtual Health Care Conference (Press release, Ascendis Pharma, MAY 4, 2021, View Source [SID1234579063]). Company executives will provide a business overview and an update on the Company’s pipeline programs.

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Details

Event BofA Securities 2021 Virtual Health Care Conference
Location Virtual
Date Tuesday, May 11, 2021
Time 4:15 p.m. Eastern Time
A live audio webcast of the presentation will be available on the Investors and News section of the Company’s website at www.ascendispharma.com. A webcast replay will also be available on the Company’s website shortly after conclusion of the event for 30 days.

On May 4, 2021 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal second quarter ended March 31, 2021 (Press release, Arrowhead Research Corporation, MAY 4, 2021, View Source [SID1234579062]). The company is hosting a conference call today, May 4, 2021, at 4:30 p.m. ET to discuss the results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 3080187.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 3080187.

Selected Recent Events

Announced positive interim 48-week liver biopsy results from the AROAAT2002 study, an open-label Phase 2 clinical study of ARO-AAT, the company’s second generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). The interim readout demonstrated that ARO-AAT treatment led to:
Consistent and substantial reduction in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer
Consistent decrease in histological globule burden
Improvements in fibrosis
Improvements in other relevant biomarkers of liver health
Arrowhead intends to present additional interim AROAAT2002 data at an upcoming medical congress, pending abstract acceptance.
Announced ARO-DUX4, Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform. ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).
Submitted an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA) for a Phase 2b dose-finding clinical study of ARO-ANG3, the company’s investigational RNAi therapeutic being developed as a treatment for patients with mixed dyslipidemia.
Submitted an IND to the FDA for a Phase 2b dose-finding clinical study of ARO-APOC3, the company’s investigational RNAi therapeutic being developed as a treatment for patients with hypertriglyceridemia.
Selected Fiscal 2021 Second Quarter Financial Results

On May 4, 2021 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the first quarter ended March 31, 2021 and provided pipeline updates (Press release, AnaptysBio, MAY 4, 2021, View Source [SID1234579061]).

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"We look forward to advancing imsidolimab into a Phase 3 trial for GPP following our recent end-of-Phase 2 meeting with the FDA," said Hamza Suria, president and chief executive officer of AnaptysBio. "AnaptysBio continues to pursue a capital-efficient business model where partnership revenues continue to support advancement of our wholly-owned pipeline poised to generate multiple clinical data catalysts through 2021 and 2022."

Imsidolimab (Anti-IL-36 Receptor) Program

We held an end-of-Phase 2 meeting with the FDA during Q2 2021 to review an orphan disease Phase 3 development plan for imsidolimab for the treatment of GPP and anticipate announcing key aspects of our Phase 3 trial design upon its initiation in mid-2021. Our worldwide registry of GPP patients, named RADIANCE, is ongoing, and we expect that this study will improve our understanding of the patient journey and support enrollment of our Phase 3 clinical trial. While initial GPP epidemiology studies suggested at least 3,000 GPP patients in the United States, medical claims analyses conducted by IQVIA indicate approximately 37,000 unique patients were diagnosed with GPP at least once, and approximately 15,000 unique patients were diagnosed with GPP at least twice, by a physician between 2017 and 2019 using the International Classification of Diseases 10th Revision (ICD-10) billing code pertaining to GPP (L40.1).
We initiated a Phase 2 clinical trial of imsidolimab in hidradenitis suppurativa, named HARP, where 120-patients are randomized equally between two dose levels of imsidolimab and placebo, and top-line data is anticipated in H2 2022. We also commenced an imsidolimab Phase 2 trial in moderate-to-severe acne, named ACORN, where 120-patients are randomized equally between two dose levels of imsidolimab and placebo, and top-line data is anticipated in H1 2022. We continue to anticipate top-line data at the end of 2021 from our EMERGE Phase 2 trial of imsidolimab in EGFR/MEK-mediated skin toxicities and top-line data from our Phase 2 INSPIRE trial in ichthyosis during 2022.
We announced in Q1 2021 top-line data from our POPLAR phase 2 clinical trial of imsidolimab monotherapy in PPP, which failed to meet the trial’s primary endpoint. While we continue to review secondary endpoints to further understand the activity of imsidolimab in various PPP patient subsets, we do not currently plan to pursue further clinical development of imsidolimab in PPP.
ANB030 (Anti-PD-1 Agonist) Program

We anticipate top-line data in H2 2021 from our ongoing Phase 1 healthy volunteer clinical trial of ANB030, our wholly-owned PD-1 agonist antibody, designed to assess the safety, pharmacokinetics and pharmacodynamics of ANB030 in single and multiple ascending dose cohorts.
We plan to initiate Phase 2 clinical trials of ANB030 in alopecia areata and vitiligo in Q4 2021.
Preclinical translational data using ANB030 was presented in March 2020 at the Festival of Biologics Meeting.
ANB032 (Anti-BTLA Modulator) Program

We achieved first-in-human dosing of ANB032, our wholly-owned BTLA modulator antibody, upon initiation of a healthy volunteer Phase 1 trial in the first quarter of 2021, under an Australian Clinical Trial Notification (CTN), and anticipate top-line data from this trial during the first half of 2022.
We presented preclinical data regarding ANB032 at the 2020 Federation of Clinical Immunology Societies (FOCIS) Virtual Annual Meeting in October 2020.
GSK Partnered Programs

A BLA for our most advanced partnered program, which is an anti-PD-1 antagonist antibody called JEMPERLI (dostarlimab), was approved by the FDA in April 2021 for the treatment of advanced or recurrent deficient mismatch repair endometrial cancer (dMMREC). This is the first AnaptysBio-generated antibody, of eight currently under clinical development, to obtain FDA approval. We earned a $20.0 million milestone payment as a result of this FDA approval.
In April 2021 the European Medicines Agency (EMA) granted conditional marketing authorization in the European Union for JEMPERLI for use in women with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer who have progressed on or following prior treatment with a platinum containing regimen, which approval makes JEMPERLI the first anti-PD-1 therapy available for endometrial cancer in Europe. We earned a $10.0 million milestone payment as a result of this approval.
A second BLA submitted by GSK was accepted by the FDA during the first quarter of 2021 for JEMPERLI in pan-deficient mismatch repair tumors (PdMMRT). We received a $10.0 million cash milestone payment upon the FDA acceptance of GSK’s second FDA BLA for JEMPERLI and anticipate an additional $20.0 million cash milestone payment upon FDA approval of this second FDA BLA of JEMPERLI during the second half of 2021. We anticipate an additional $15.0 million and $165.0 million in milestone payments upon achievement of certain JEMPERLI regulatory and commercial milestones, respectively.
During Q1 2021, we recognized $1.2 million in royalty revenue related to GSK’s Zejula product sales, which we estimated based on GSK’s historical sales. In October 2020, we amended our GSK collaboration which resulted in increased royalties on global net sales of JEMPERLI to 8-25%, a 1% royalty rate on GSK’s global net sales of Zejula and a one-time cash payment of $60.0 million.
First Quarter Financial Results

Cash, cash equivalents and investments totaled $387.4 million as of March 31, 2021 compared to $411.2 million as of December 31, 2020, for a decrease of $23.8 million. The decrease relates primarily to cash used for operating activities.
Collaboration revenue was $11.2 million for the three months ended March 31, 2021, $10.0 million related to milestone revenue for the FDA accepted BLA filing of the second dostarlimab indication and $1.2 million related to royalties on GSK’s Zejula product sales, compared to $15.0 million of milestone revenue for the three months ended March 31, 2020.
Research and development expenses were $24.2 million for the three months ended March 31, 2021, compared to $21.0 million for the three months ended March 31, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.4 million for the three months ended March 31, 2021, compared to $4.3 million for the three months ended March 31, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $18.2 million for the three months ended March 31, 2021, or a net loss per share of $0.66, compared to a net loss of $8.3 million for the three months ended March 31, 2020, or a net loss per share of $0.30.
Financial Guidance

AnaptysBio expects its net cash burn in 2021 will be close to $100 million. We anticipate that our cash, cash equivalents and anticipated revenues will fund our current operating plan at least into 2024.

On May 4, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that it has entered into a joint research agreement with BioArctic AB, a Swedish research-based biopharma company focusing on disease-modifying treatments and reliable biomarkers and diagnostics for neurodegenerative diseases, such as Alzheimer’s disease and Parkinson’s disease (Press release, Alligator Bioscience, MAY 4, 2021, View Source [SID1234579060]). Under the agreement, Alligator will employ its proprietary antibody generation technologies in collaboration with BioArctic to develop new product candidates.

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"We are very pleased that BioArctic has recognized the power of Alligator’s proprietary phage display libraries which have been successfully used to generate Alligator’s suite of immuno-oncology programs. The application of this technology to the neurodegenerative field demonstrates the broad applicability of our powerful platform," said Malin Carlsson, interim CEO of Alligator Bioscience.

"BioArctic’s focus is to develop new treatments to help patients with neurological diseases. We are looking forward to working together with Alligator, utilizing their phage display platform, in the development of new antibody therapeutic candidates with novel mechanisms of action," said Johanna Fälting, Vice President and Head of Research at BioArctic.