On September 12, 2023 Ferring Pharmaceuticals reported the first patient in the United States with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) was dosed with the commercially available intravesical gene therapy ADSTILADRIN (nadofaragene firadenovec-vncg) as part of the ADSTILADRIN Early Experience Program announced earlier this year (Press release, Ferring Pharmaceuticals, SEP 12, 2023, View Source [SID1234635127]).

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ADSTILADRIN was approved by the U.S. Food & Drug Administration (FDA) in December 2022 for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. It is the first and only FDA-approved intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy.

"The first patient dosed with ADSTILADRIN marks an incredible milestone for Ferring, the bladder cancer community and the patients we aim to serve," said Shetal Vyas, Vice President, General Manager, Uro-Oncology at Ferring Pharmaceuticals. "Making this novel and efficacious intravesical gene therapy commercially available for patients underscores our commitment to changing the trajectory of NMIBC. We will continue bringing available doses of ADSTILADRIN forward responsibly as we scale up manufacturing."

The patient, a 78-year-old male treated at a clinic within the mid-west area also was enrolled in the non-interventional study, the "ADSTILADRIN in BLadder CancEr" (ABLE-41) U.S. Real World Evidence (RWE) Study. The research study, launched in tandem with participating Early Experience Program clinics, will explore early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting. Learn more at www.clinicaltrials.gov/study/NCT06026332.

"ADSTILADRIN represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder," said Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals. "The ADSTILADRIN Early Experience Program and ABLE-41 US RWE Study allow us to address pressing patient needs while collecting data on its use in a real-world setting, further expanding on what we’ve learned in our Phase 3 clinical program."

Earlier this month, Ferring made doses of ADSTILADRIN commercially available through an Early Experience Program to urologists at the clinical trial sites that participated in the Phase 3 study1 and a mix of community clinics with the highest number of eligible high-risk patients with NMIBC. The Early Experience Program is designed to ensure every patient who starts on therapy will continue to receive future doses of ADSTILADRIN for the duration of their treatment.

The ADSTILADRIN Early Experience Program is temporary, and more clinics will be offered the opportunity to participate as manufacturing volumes steadily increase over time.

About the ABLE-41 (Adstiladrin in BLadder CancEr) US RWE Study
The ABLE-41 US RWE Study is a real-world observational study of ADSTILADRIN focused on patient treatment outcomes and early use experiences in U.S. routine care settings. Up to 800 patients enrolled in the ADSTILADRIN Early Experience Program who previously have not received ADSTILADRIN will be followed for a minimum of 24 months. The effectiveness of ADSTILADRIN will be measured as complete response rates. Data analyzed will examine patterns of ADSTILADRIN use, and overall experience among patients, caregivers and healthcare providers.

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical gene therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene encoding interferon alfa-2b protein, administered by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high quantities of interferon alfa-2b protein, a naturally-occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).1

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.2 Bladder cancer is the sixth most common cancer in the U.S., and it is estimated that there were approximately 82,290 new cases of bladder cancer in the U.S. in 2022,3 75% of which present as NMIBC.4 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.2 Current treatment options for BCG-unresponsive patients are very limited, and often result in a highly invasive life-changing procedure of radical cystectomy (complete removal of the bladder).5

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with hypersensitivity to interferon alfa or any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

On September 12, 2023 Agilent Technologies, Inc. (NYSE: A) and Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported they have entered into a definitive agreement for the sale of Resolution Bioscience to Exact Sciences (Press release, Agilent, SEP 12, 2023, View Source [SID1234635126]). Financial terms of the agreement were not disclosed and are not material to either party.

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"We are pleased that Exact Sciences is acquiring Resolution Bioscience," said Sam Raha, senior vice president, Agilent, and president, Diagnostics and Genomics Group. "Exact is a leading centralized laboratory for advanced cancer diagnostic testing with significant capabilities and resources in this area, which makes Resolution Bioscience a strategic fit for them. This agreement will enable the talented Resolution Bioscience team to continue their work advancing diagnostic solutions for their customers and patients, a very positive outcome of this transaction."

"We are excited to work with the Resolution Bioscience team to integrate their innovative diagnostics into our Precision Oncology portfolio," said Brian Baranick, general manager of Precision Oncology at Exact Sciences. "Resolution Bioscience’s high-quality liquid therapy selection platform perfectly complements our OncoExTra test, allowing Exact Sciences to help more cancer patients determine their best treatment options."

Resolution Bioscience develops and commercializes next-generation sequencing-based precision oncology solutions. Agilent acquired the company in 2021.

On September 12, 2023 Innocare reported that American Journal of Hematology recently published the study result of BTK (Bruton Tyrosine Kinase) inhibitor orelabrutinib in patients with relapsed or refractory (r/r) Marginal Zone Lymphoma (MZL), which investigated the efficacy and safety of orelabrutinib in r/r MZL (Press release, InnoCare Pharma, SEP 12, 2023, View Source [SID1234635125]). The journal concluded that orelabrutinib demonstrated high response rates with durable disease remission and was well tolerated in patients with relapsed or refractory MZL.

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The majority of the enrolled patients had late-stage disease. After a median follow-up duration of 24.3 months, the overall response rate (ORR) assessed by an Independent Review Committee (IRC) ORR was 58.9%. Tumor reduction was observed in 92.2% of patients. The IRC-assessed median duration of response (DOR) was 34.3 months and median progression-free survival (PFS) was not reached. The 12-month PFS rate and overall survival (OS) rate was 82.8% and 91.0% respectively.

Due to the high target selectivity and fewer off-target effects, orelabrutinib demonstrated a good safety profile in the treatment of patients with r/r MZL.

In China, orelabrutinib was the first and only approved BTK inhibitor for the treatment of r/r MZL. Orelabrutinib has been also approved in China for the treatment of r/r chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and r/r mantle cell lymphoma (MCL).

Marginal zone lymphoma (MZL) is a type of non-Hodgkin lymphoma (NHL) of indolent nature originating from B cells in the marginal zones of the spleen, lymphatic tissue, and lymph nodes. MZL accounts for 7%-8% of NHL cases and is the second most prevalent lymphoma among elderly adults. The annual incidence of MZL has increased globally.

The journal concluded that orelabrutinib produced a robust response and was well tolerated in r/r MZL patients. High response rates were consistent among patients with different MZL subtypes and those with negative disease prognostic factors at baseline. The results of this study support the use of orelabrutinib as an effective and tolerable oral treatment option for r/r MZL patients.

The American Journal of Hematology is an academic journal focusing on hematology, which was founded in 1976 and published monthly by WILEY publisher. The journal has been included in SCIE and SCI databases, with an impact factor of 13.268 in 2022.

Note: In addition to background information, the content of this press release is derived from this published article. Full text can be found in View Source

About Orelabrutinib

Orelabrutinib is a highly selective BTK inhibitor developed by InnoCare for the treatment of cancers and autoimmune diseases.

On Dec. 25, 2020, orelabrutinib received conditional approval from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). At the end of 2021, orelabrutinib was included into National Reimbursement Drug list to benefit more lymphoma patients. On Nov. 22, 2022, orelabrutinib was approved for the treatment of R/R MCL in Singapore. On April 20, 2023, orelabrutinib was approved for the treatment r/r MZL in China.

In addition to the approved indications, multi-center, multi-indication clinical trials are underway in the US and China with orelabrutinib as monotherapy or in combination therapies, such as first line treatment of MCD subtype of diffuse large B-cell lymphoma (DLBCL).

Orelabrutinib was granted as Breakthrough Therapy Designation for the treatment of r/r MCL by U.S. Food and Drug Administration (FDA). Patient enrollment of Phase II registrational trial for R/R MCL was completed in the U.S. The Company expects to submit the NDA to the U.S. Food and Drug Administration (US FDA) in the middle of 2024.

In addition, the Company has achieved proof of concept (PoC) of orelabrutinib for the treatment of primary immune thrombocytopenia purpura (ITP) and the Phase III registrational trial is ongoing in China. Orelabrutinib’s global phase II studies for the treatment of Multiple Sclerosis (MS), and clinical trials for the treatment of SLE achieved proof of concept (PoC), and orelabrutinib’s phase II study for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) is ongoing in China.

On September 12, 2023 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, reported that it intends to offer and sell shares of its common stock, and to certain investors, pre-funded warrants to purchase shares of its common stock, in an underwritten public offering (Press release, Rocket Pharmaceuticals, SEP 12, 2023, View Source [SID1234635124]). The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $150 million. The purchase price of each pre-funded warrant will equal the price per share at which shares of common stock are being sold to the public in this offering, minus $0.01, which will be the per share exercise price of each pre-funded warrant. All shares and pre-funded warrants in the offering are to be sold by Rocket. In addition, Rocket intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of shares of its common stock offered in the public offering. The offering is subject to market 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.

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J.P. Morgan, Morgan Stanley, Leerink Partners and TD Cowen are acting as joint book-running managers, and LifeSci Capital is acting as lead manager for the offering.

The shares and pre-funded warrants are being offered by Rocket pursuant to an effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (the "SEC"). The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source

When available, copies of the final prospectus supplement relating to the offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, by telephone at (866) 803-9204, or by email at [email protected]; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, by telephone at (866) 718-1649 or by email at [email protected]; Leerink Partners LLC, Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525 ext. 6105 or by email at [email protected]; or Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by telephone at (833) 297-2926 or by email at [email protected]. You may also obtain a copy of this document free of charge by visiting the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any 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.

On September 12, 2023 Mission Bio, a leader in single-cell multi-omic solutions for precision medicine, reported to have launched the Tapestri Genome Editing Solution (Press release, Mission Bio, SEP 12, 2023, View Source [SID1234635123]). Designed to meet the pressing need for high-resolution analysis of genome editing, this first-of-its-kind solution addresses a long-standing gap in advanced therapy development, disease modeling, and functional genomics. Already drawing substantial interest from key opinion leaders in pharmaceuticals, academia, and government labs, this much-anticipated solution was initially previewed at The American Society of Gene and Cell Therapy annual conference.

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The Tapestri Genome Editing Solution has numerous applications for genome engineers, advanced therapy developers, and disease modelers. The solution allows for the rapid characterization of gene-edited drug products, identifying on- and off-target editing, the zygosity of edits, and the co-editing of multiple targets in single cells. The solution also supports multi-omic analysis — co-measuring editing and cell-surface protein expression in the same cells. This paints a clearer picture of cell engineering, as one can determine which cell types are edited, or perhaps validate a knockout through the loss of a protein.

"The Tapestri Genome Editing Solution equips researchers with the capability to scrutinize gene therapies at the single-cell level, a crucial step for ensuring patient safety," said Brittany Enzmann, PhD, Product Manager for Mission Bio’s Tapestri Genome Editing Solution. "By providing this unparalleled resolution, we’re not just offering a new tool; we’re contributing to the evolution of safer and more effective gene therapies. The result is a faster transition from the lab to the patient."

Gene editing technologies, such as CRISPR, have offered immense opportunities for addressing incurable and untreatable diseases like sickle cell disease, where the first CRISPR-edited cell therapy may be approved by the U.S. Food and Drug Administration later this year. Gene editing has also proven to be a powerful strategy in developing disease models and determining genomic drivers of disease progression. However, they also bring intricate challenges such as evaluating the fidelity of both intended and unintended edits. Conventionally, researchers have to perform single-cell cloning in order to fully understand what types of edits were occurring in individual cells, a labor-intensive process that involves cell culture for several weeks.

Mission Bio’s Tapestri Genome Editing Solution was developed to provide a high-throughput workflow that combines genotypic and immunophenotypic assessments in single cells for samples of thousands of cells, capturing crucial details often overlooked by bulk analysis methods. The platform features automated data reporting, eliminating the need for devoted bioinformatics resources and saving days of computational work. As a result, a wider range of professionals, from genome engineers to clinicians to therapy developers, can access and interpret this critical information more readily.

Saar Gill MD, PhD, Associate Professor of Medicine at the University of Pennsylvania, whose laboratory focuses on developing genetically engineered immune cells for the treatment of cancer states, "This multi-omic genome-editing solution will enable critical insights for gene-edited cell therapies beyond what conventional bulk analysis can offer. The automated report gives an immediate and intuitive first look at our data, something that would usually require hours of bioinformatic labor. All in all, we are better equipped to understand our editing results with this technology."

The technology will be showcased at the upcoming Cell Therapy Analytical Development Summit in Amsterdam from September 19-21. As gene editing continues to take on an increasingly critical role in the medical and scientific communities, the spotlight will be on this new technology’s potential to reshape our understanding of cell therapy characterization. Matthew Cato, Vice President, Business and Strategic Market Development at Mission Bio, is scheduled to deliver a presentation aimed at unpacking the technology’s promise and utility.

For more information on the Single-cell Tapestri Genome Editing Solution, visit View Source