On April 25, 2022 AstraZeneca reported it’s Biologics License Application (BLA) for tremelimumab has been accepted for Priority Review in the US, supporting the indication of a single priming dose of the anti-CTLA4 antibody added to Imfinzi (durvalumab) for the treatment of patients with unresectable hepatocellular carcinoma (HCC) (Press release, AstraZeneca, APR 25, 2022, View Source [SID1234612891]). A supplemental BLA (sBLA) has also been submitted for Imfinzi in this indication. This novel dose and schedule of the combination is called the STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab).

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The Prescription Drug User Fee Act date, the Food and Drug Administration (FDA) action date for their regulatory decision, is during the fourth quarter of 2022 following the use of a priority review voucher.

Liver cancer, of which HCC is the most common type, is the third-leading cause of cancer death and the sixth most commonly diagnosed cancer worldwide.1,2 Approximately 26,000 people in the US present with advanced, unresectable HCC each year.3

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "The HIMALAYA Phase III trial showed an unprecedented three-year overall survival in this setting with a single priming dose of tremelimumab added to Imfinzi, highlighting the potential for this regimen to improve longer-term survival outcomes. Patients with advanced liver cancer are in great need of new treatment options, and we are working closely with the FDA to bring this novel approach to patients in the US as soon as possible."

The BLA for tremelimumab and sBLA for Imfinzi are based on final results from the HIMALAYA Phase III trial presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium.

In this trial, patients treated with the STRIDE regimen experienced a 22% reduction in the risk of death versus sorafenib (based on a hazard ratio [HR] of 0.78, 96.02% confidence interval [CI] 0.65-0.93; p=0.0035).4 Nearly one in three (31%) patients were still alive at three years versus one in five (20%) for sorafenib.4

The safety profiles of the STRIDE regimen and for Imfinzi alone were consistent with the known profiles of each medicine, and no new safety signals were identified.

Imfinzi and tremelimumab were granted Orphan Drug Designation in the US for the treatment of HCC in January 2020.

As part of its extensive clinical development programme in gastrointestinal (GI) cancers, AstraZeneca is further assessing Imfinzi across multiple liver cancer settings, including locoregional HCC (EMERALD-1, EMERALD-3) and adjuvant HCC (EMERALD-2).

Notes

Liver Cancer
About 75% of all primary liver cancers are HCC.1 Between 80-90% of all patients with HCC also have cirrhosis.5 Chronic liver diseases are associated with inflammation that over time can lead to the development of HCC.5

More than half of patients are diagnosed at advanced stages of the disease, often when symptoms first appear.6 A critical unmet need exists for patients with HCC who face limited treatment options.6 The unique immune environment of liver cancer provides clear rationale for investigating medications that harness the power of the immune system to treat HCC.6

HIMALAYA
HIMALAYA was a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy and the STRIDE regimen, comprising a single priming dose of tremelimumab 300mg added to Imfinzi 1500mg followed by Imfinzi every four weeks versus sorafenib, a standard-of-care multi-kinase inhibitor.

The trial included a total of 1,324 patients with unresectable, advanced HCC who had not been treated with prior systemic therapy and were not eligible for locoregional therapy (treatment localised to the liver and surrounding tissue).

The trial was conducted in 181 centres across 16 countries, including in the US, Canada, Europe, South America and Asia. The primary endpoint is overall survival (OS) for STRIDE versus sorafenib and key secondary endpoints included OS for Imfinzi versus sorafenib, objective response rate and progression-free survival (PFS) for STRIDE and for Imfinzi alone.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy, and is the global standard of care in this setting based on the PACIFIC Phase III trial.

Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.

In the past year, Imfinzi has demonstrated clinical benefit in multiple additional cancer settings with positive Phase III trials in advanced biliary tract cancer (TOPAZ-1), unresectable advanced liver cancer (HIMALAYA) and metastatic NSCLC (POSEIDON).

As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with small cell lung cancer, NSCLC, bladder cancer, several gastrointestinal cancers, ovarian cancer, endometrial cancer, and other solid tumours.

Tremelimumab
Tremelimumab is a human monoclonal antibody and potential new medicine that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Tremelimumab blocks the activity of CTLA-4, contributing to T-cell activation, priming the immune response to cancer and fostering cancer cell death.

Beyond HIMALAYA, tremelimumab is being tested in combination with Imfinzi across multiple tumour types including locoregional HCC (EMERALD-3), SCLC (ADRIATIC) and bladder cancer (VOLGA and NILE).

Tremelimumab is also under review by global regulatory authorities in combination with Imfinzi and chemotherapy in 1st-line metastatic NSCLC based on the results of the POSEIDON Phase III trial, which showed the addition of a short course of tremelimumab to Imfinzi plus chemotherapy improved both overall and progression-free survival compared to chemotherapy alone.

AstraZeneca in GI cancers
AstraZeneca has a broad development programme for the treatment of GI cancers across several medicines spanning a variety of tumour types and stages of disease. In 2020, GI cancers collectively represented approximately 5.1 million new diagnoses leading to approximately 3.6 million deaths.7

Within this programme, the Company is committed to improving outcomes in gastric, liver, biliary tract, oesophageal, pancreatic, and colorectal cancers.

Imfinzi (durvalumab) is being assessed in combinations including with tremelimumab in HCC, biliary tract, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease across settings. In January 2022, results of the TOPAZ-1 Phase III trial in 1st-line advanced biliary tract cancer demonstrated a significant improvement in OS with Imfinzi plus standard-of-care chemotherapy versus chemotherapy alone.

The Company aims to understand the potential of Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate, in the two most common GI cancers, colorectal and gastric cancers. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.

Lynparza (olaparib) is a first-in-class PARP inhibitor with a broad and advanced clinical trial programme across multiple GI tumour types including pancreatic and colorectal cancers. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. inside the US and Canada).

AstraZeneca in immunotherapy
Immunotherapy is a therapeutic approach designed to stimulate the body’s immune system to attack tumours. The Company’s Immuno-Oncology (IO) portfolio is anchored in immunotherapies that have been designed to overcome evasion of the anti-tumour immune response. AstraZeneca is invested in using IO approaches that deliver long-term survival for new groups of patients across tumour types.

The Company is pursuing a comprehensive clinical-trial programme that includes Imfinzi as a single treatment and in combination with tremelimumab and other novel antibodies in multiple tumour types, stages of disease, and lines of treatment, and where relevant using the PD-L1 biomarker as a decision-making tool to define the best potential treatment path for a patient.

In addition, the ability to combine the IO portfolio with radiation, chemotherapy, and targeted small molecules from across AstraZeneca’s oncology pipeline, and from research partners, may provide new treatment options across a broad range of tumours.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

On April 25, 2022 MacroGenics, Inc. (Nasdaq: MGNX), a biopharmaceutical company focused on developing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported that the Company will release its financial results for the first quarter 2022 after the market closes on Tuesday, May 3, 2022 (Press release, MacroGenics, APR 25, 2022, View Source [SID1234612907]). MacroGenics will host a conference call to discuss the financial results and recent corporate progress on Tuesday, May 3, 2022, at 4:30 pm ET. The conference call can be accessed by dialing (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and providing the Conference ID# 6791448.

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The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On April 25, 2022 Hologic, Inc. (Nasdaq: HOLX) reported that the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) has published an update which expands the utility of Breast Cancer Index (BCI) within its Clinical Practice Guideline: "Biomarkers for Adjuvant Endocrine and Chemotherapy in Early-Stage Breast Cancer (Press release, Hologic, APR 25, 2022, View Source [SID1234612924])." Specifically, ASCO (Free ASCO Whitepaper) now recognizes BCI as the only genomic test to help guide extended endocrine therapy decisions in early-stage, HR+ breast cancer patients with node negative or node positive (one-three positive nodes) disease when treated with five years of primary endocrine therapy without evidence of recurrence. A special article highlighting the new ASCO (Free ASCO Whitepaper) guidelines was recently published in the Journal of Clinical Oncology on this subject.1

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"The clinical decision to either extend or end adjuvant endocrine therapy after five years is a challenging decision for healthcare providers and their patients. I am pleased to see updated guidelines from ASCO (Free ASCO Whitepaper) affirming the use of a data-driven biomarker like Breast Cancer Index to predict likelihood of benefit from extended endocrine therapy, helping to better inform decision-making processes around treatment plans," said Mark Pegram, MD, Chief Medical Consultant for Breast Oncology at Biotheranostics, a subsidiary of Hologic. "There is an extensive body of clinical evidence consistently proving the utility of BCI, and its addition to major oncology clinical guidelines like those from ASCO (Free ASCO Whitepaper) further underscores the test’s potential in clinical decision-making regarding extended adjuvant endocrine therapy."

BCI is a proprietary molecular gene expression-based test that is uniquely positioned to provide information to help physicians individualize treatment plans beyond five years. It is also the only test to be recognized by other major clinical practice guidelines for prediction of which early-stage, HR+ breast cancer patients are likely to benefit from extended endocrine therapy.2 Extended endocrine therapy has been demonstrated to help reduce the risk of recurrence in some women with early-stage, HR+ breast cancer. However, for breast cancer patients on extended endocrine therapy, the potential side effects and toxicities of treatment often have significant negative effects on health and quality of life, such as osteoporosis, bone fractures and joint pain.3-6 As a result, it’s important to know if a patient is unlikely to benefit from extended endocrine therapy to help reduce these challenging side effects and health consequences.

"We are pleased to see that ASCO (Free ASCO Whitepaper) updated its clinical practice guidelines to include BCI as the only genomic test to predict the value of extended endocrine therapy, reaffirming other guidelines within clinical oncology," said Kevin Thornal, Hologic’s president, Diagnostic Solutions Division. "We look forward to continuing to improve women’s health by giving healthcare providers the information they need to make the best treatment decisions for their patients."

According to the ASCO (Free ASCO Whitepaper) Guideline Update, the purpose was to "update recommendations on appropriate use of breast cancer biomarker assay results to guide adjuvant endocrine and chemotherapy decisions in early-stage breast cancer.1" An updated literature search identified 24 randomized clinical trials and prospective-retrospective studies published from January 2016 to October 2021, which were evaluated by an Expert Panel to develop evidence-based recommendations.

About Breast Cancer Index
Breast Cancer Index is a molecular, gene expression-based test uniquely positioned to provide information to help physicians individualize treatment decisions for patients with early-stage, HR+ breast cancer. This breakthrough test helps oncologists and patients navigate the difficult trade-offs between taking steps to prevent recurrence of their disease and facing significant side effects and safety challenges related to unnecessary treatment. Breast Cancer Index has guideline designation from the American Joint Committee on Cancer for cancer staging based on molecular profile. ASCO (Free ASCO Whitepaper), the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), the European Group on Tumor Markers (EGTM) and St. Gallen acknowledge Breast Cancer Index as a biomarker to inform the chemotherapy decision; and ASCO (Free ASCO Whitepaper) and EGTM acknowledge BCI as a biomarker to inform the extended endocrine treatment decision. It is the only validated, commercially available test that predicts benefit from extended endocrine therapy. Breast Cancer Index is intended for routine clinical use, and treatment decisions based on results are the responsibility of the physician. It is a laboratory developed test (LDT) performed in a single CLIA-certified and CAP-accredited diagnostic laboratory and is not required to be cleared or approved by the US Food and Drug administration. For more information, visit www.breastcancerindex.com.

On April 25, 2022 Catalent, the global leader in enabling biopharma, cell, gene, and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, reported that it has acquired from Erytech Pharma its state-of-the-art, commercial-scale cell therapy manufacturing facility in Princeton, New Jersey, for $44.5 million (Press release, Catalent, APR 25, 2022, https://www.catalent.com/catalent-news/catalent-acquires-commercial-scale-cell-therapy-development-and-manufacturing-facility-in-princeton-new-jersey-from-erytech/ [SID1234612892]). The deal includes an exclusive long-term supply agreement for Catalent to support Erytech’s lead product candidate eryaspase (GRASPA), a red blood cell-derived product, which is currently in late-stage development for the treatment of acute lymphoblastic leukemia.

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The 30,900-square-foot facility is located within the University Square Campus Park and houses 16 suites positioned for production of products in a cGMP-compliant manner, as well as laboratories for analytical, quality control and microbiology testing. The site will become a strategic campus for development as well as clinical- and commercial-scale manufacturing of cell therapies, and is located within easy reach of Catalent’s facilities around Baltimore, Maryland, where the company develops and manufactures viral vectors and plasmid DNA. It will also work in collaboration with Catalent’s existing U.S. clinical-scale cell therapy facility in Houston, Texas.

Concurrent with the acquisition, Catalent has agreed to terms to lease an adjacent 23,000-square-foot building that could be used for additional laboratory or cGMP capabilities, and another building on the campus for potential future expansions. All staff at the site will transfer to Catalent’s employment, and Catalent is developing its plans for its future recruitment needs.

Under the supply agreement, Catalent will continue to manufacture eryaspase (GRASPA) at the site. It will also support the development of Erytech’s pipeline of encapsulated red blood cell-based therapeutics for severe forms of cancer and orphan diseases.

"This acquisition is strategically important to Catalent’s commitment to support the development and clinical and commercial supply of cell therapies to meet rapidly growing demand," said Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy. "The talented and experienced staff already employed at the facility, the capabilities it has in place, and the opportunity to quickly add further capacity on the same site, allow Catalent to expand rapidly to create a U.S. campus and center of excellence for cell therapy development and manufacturing that will serve customers around the world."

"In Catalent we have found a great partner for the manufacturing of our innovative red blood cell-derived products," said Gil Beyen, Chief Executive Officer at Erytech. "Catalent Cell & Gene Therapy is an industry-leading technology, development, and manufacturing partner for advanced therapeutics, and we believe this win-win strategic partnership will meet our long-term manufacturing needs in the United States. Erytech will now further focus its resources on seeking regulatory approval of GRASPA and on the development of potentially transformative therapeutics for serious diseases."

Catalent’s cell and gene therapy network includes six U.S. facilities across Maryland and Texas, a European center of excellence in Belgium, and its cell innovation facility in Germany. These sites offer a range of small- and large-scale clinical and commercial manufacturing, as well as fill/finish capabilities.

ABOUT CELL & GENE THERAPY
Catalent Cell & Gene Therapy is an industry-leading technology, development, and manufacturing partner for advanced therapeutics. Its comprehensive cell therapy portfolio includes a wide range of expertise across a variety of cell types including CAR-T, TCR, TILs, NKs, iPSCs, and MSCs. With deep expertise in viral vector development, scale-up and manufacturing for gene therapies, Catalent is a full-service partner for plasmid DNA, adeno-associated viral (AAV), lentiviral and other viral vectors, oncolytic viruses, and live virus vaccines. An experienced and innovative partner, Catalent Cell & Gene Therapy has a global network of dedicated, small- and large-scale clinical and commercial manufacturing facilities, including an FDA-licensed viral vector facility, and fill/finish capabilities located in both the U.S. and Europe.

On April 25, 2022 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer cell therapies to treat cancer, reported that it has commenced a proposed underwritten public offering to issue and sell $150 million of shares of its common stock (Press release, Nkarta, APR 25, 2022, View Source [SID1234612908]). The Company also intends to grant the underwriters a 30-day option to purchase up to an additional $22.5 million of shares of its common stock on the same terms and conditions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering. All shares in the offering are to be issued and sold by Nkarta.

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Nkarta intends to use the net proceeds from the offering to fund the continued clinical development of NKX101 and NKX019, preclinical studies for research stage programs and the continued buildout of internal manufacturing capabilities, and for working capital and for general corporate purposes.

Cowen, SVB Securities and Evercore ISI are acting as joint book-running managers for the offering.

The offering is being conducted pursuant to a shelf registration statement (File No. 333-258766), which was filed with the Securities and Exchange Commission ("SEC") on August 12, 2021 and declared effective by the SEC on September 2, 2021. The proposed offering will be made only by means of a prospectus supplement and accompanying prospectus describing the terms of the offering. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained by contacting Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at (833) 297-2926, or by email at [email protected]; SVB Securities LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected]; and Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at [email protected]. These documents may also be obtained for free on the SEC’s website located at View Source The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities described herein, nor shall there be any offer, solicitation or sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.