On May 2, 2022 HAYA Therapeutics, SA, a company developing precision medicines that target tissue and cell-specific long non-coding RNAs (lncRNAs), reported that Innosuisse, the Swiss Innovation Agency, is supporting two research collaboration projects between HAYA and the University of Bern, University Hospital of Bern and Lausanne University Hospital (Press release, Haya Therapeutics, MAY 2, 2022, View Source [SID1234613326]). Innosuisse is funding 50 percent of the total project costs of approximately CHF 3.1 million (US$3.3 million).

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The first project will advance HAYA’s lead program, an antisense oligonucleotide targeting the lncRNA Wisper, a cardiac tissue-enriched driver of fibrosis in the heart. In collaboration with the Department for BioMedical Research at University of Bern and the Department of Cardiology at University Hospital of Bern (Inselspital), the two-year project will be focused on dosing studies, pharmacodynamics, and pharmacokinetics for this first-in-class therapeutic target for the potential treatment of non-obstructive hypertrophic cardiomyopathy. The study also includes state-of-the-art cardiac MRI read-outs to provide evidence for efficacy and translatability of the therapeutic approach and advancing it towards the clinic and in-need patients.

"The whole team at HAYA is extremely excited to see our lead Wisper-targeting antisense compound being evaluated in a translationally relevant preclinical model of heart failure," said Daniel Blessing, Ph.D., Co-founder and CTO of HAYA Therapeutics. "Support from Innosuisse has been instrumental to enable this study and collaboration with the University and Inselspital Bern."

"As a pioneer in the field of lncRNA, HAYA has made significant progress in developing a novel treatment targeting lncRNA for hard-to-treat cardiac diseases," said Dr. Robert Rieben, Professor at the University of Bern. "With experience in preclinical cardiovascular research, we are excited to work with HAYA and support their efforts of bringing this therapy to patients who desperately need them."

HAYA’s second project will aim to develop a next-generation oncology therapy targeting cancer-associated fibroblasts for the treatment of squamous cell carcinoma. Through a partnership with Lausanne University Hospital (Centre hospitalier universitaire vaudois, CHUV), the collaborators will use HAYA’s proprietary drug discovery engine, DiscoverHAYATM, to identify oncology-associated fibroblast-specific lncRNAs for the development of a precision RNA-targeted therapy.

"Since launch, HAYA has been diligently working on bringing our lead lncRNA-targeting antisense candidate for the treatment of heart failure closer to clinical testing. At the same time, we have been conducting studies using our innovative discovery engine to identify novel lncRNA targets outside of cardiomyopathy," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "With this project funding from Innosuisse, we can continue our efforts in heart disease and use our technology beyond cardiology into cancer. This will open up tremendous opportunities in the discovery of oncology-based lncRNA targets."

On May 2, 2022 Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, reported six poster presentations at PEGS (The Essential Protein Engineering & Cell Therapy Summit) Boston occurring May 2-6 virtually and in-person at the Hynes Convention Center in Boston, MA (Press release, Twist Bioscience, MAY 2, 2022, View Source [SID1234613325]). In addition, Aaron Sato, Ph.D., chief scientific officer of Twist Bioscience, will present ‘Writing the Future of Biologics’ on May 2 at 2:50 PM ET and Tracey Mullen, SVP, operations, will present ‘Rapid, Function-Forward mAb Discovery against a Cell Surface Target via Concurrent Use of Humanized and Hyperimmune Mice’ on May 4 at 12:30 PM ET. Twist will also host a networking event on May 4 from 7:00-10:00 PM ET. Visit https://bit.ly/TwistMixer to register.

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"These posters show the depth and breadth of the antibody discovery and library construction capabilities of Twist Biopharma against high-impact targets," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "Our next generation libraries enable antibody discovery for therapies such as CAR-T engineered cell therapies, which have the potential to change the treatment landscape for patients. In addition, the data presented on the discovery of an antibody targeting ADORA2A, a next generation checkpoint, demonstrate Twist Biopharma’s ability to generate antibodies independently."

Poster Number: P003

Advanced Antibody Discovery Workflow to Capture Maximum Repertoire Diversity

The complexity of antibody therapeutic targets continues to evolve, which in turn necessitates the evolution of integrated discovery methodologies. Incorporating state-of-the-art, high-resolution techniques enables reliable candidate triage more efficiently than traditional techniques. Major advancements in critical tools have improved antibody discovery by providing robust and thorough analysis of target specificity, function, and developability earlier in the drug development process. Effective integration of these technologies bolsters the antibody discovery process and facilitates lead candidate selection within as few as two months.

Poster Number: P004

In Vivo VHH Discovery Workflow Based on Immunized Alpaca and Rapid Beacon-Based Single B Cell Screening

VHH antibodies have demonstrated tremendous promise as versatile building blocks for antibody-based therapeutics, multispecifics and cell-based biologics due to higher affinity and better access to hidden epitopes on cell surface targets as compared to conventional IgG antibodies. Effective integration of advanced tools for analysis of target specificity, function and developability can bolster the VHH discovery process and facilitate lead candidate selection for novel therapeutic modalities against traditionally challenging targets.

Poster Number: P143

Next Generation Synthetic Libraries for Enzyme Engineering, Cell Therapy and Gene Editing Technologies

This poster details Twist’s next generation libraries including CRISPR gRNA libraries, which are an efficient tool for high throughput gene editing and knockout of molecular targets; synonymous codon (SynCodon) libraries, which are optimized to improve protein yields, binding affinity, stability, and expression of proteins; and T cell receptor (TCR) and chimeric antigen receptor (CAR) T cell libraries, which can be used in combination to screen a large scale of module sets.

Poster Number: P144

Discovery of Pre-clinical ADORA2A Antibody with Twist High Throughput Antibody Discovery Platform

These data detail how Twist used its high-throughput DNA synthesis platform and a large-scale phage library built based on a mouse immunization platform from Abveris, a division of Twist Bioscience, to discover a high-affinity antagonistic ADORA2A antibody, which in preclinical studies restored T cell activity and showed anti-tumor activity.

Poster Number: P145

Engineering Synthetic Multivalent VHH Antibodies at Scale

These data describe Twist’s scalable process for engineering high affinity mono and bispecific multivalent VHH-Fc antibodies against the SARS-CoV-2 spike protein.

Poster Number: P146

Leveraging synthetic Library of Libraries to enable effective antibody discovery against high-impact targets

This poster demonstrates how Twist discovered high-affinity antibodies for six cytokine or immunomodulatory targets by constructing synthetic antibody libraries, using phage display to pan libraries against biotinylated protein targets, and screening for lead candidates through ELISA binding assays and NGS enrichment tracking.

About Twist Biopharma

By leveraging our unique ability to manufacture DNA at scale, we can construct proprietary antibody libraries precisely designed to match sequences that occur in the human body. The Library of Libraries gives our partners an integral and unbiased resource for antibody discovery and optimization. This precise and rational approach to library fabrication combined with sophisticated bioinformatics and software expertise expedites antibody discovery by decreasing risk, increasing speed, and lowering the failure rate for antibody development.

On May 2, 2022 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported the availability of Shield, the company’s first blood-based test for the detection of early-stage colorectal cancer (CRC) (Press release, Guardant Health, MAY 2, 2022, View Source [SID1234613324]). The test, which only requires patients to complete a simple blood draw, is intended for adults age 45 and older who are not up to date with recommended screening guidelines, show no symptoms, and are at average risk for CRC.

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Colorectal cancer is the second-leading cause of cancer-related deaths in the U.S.1 Today, one in three adults have not completed the recommended CRC screening even though colorectal cancer is curable if caught early. Barriers associated with currently available methods, such as a colonoscopy or a stool-based test, can make the process unpleasant, time-consuming and difficult to complete.2 With a simple blood draw, the Shield test overcomes these barriers because it requires no special preparation, no sedation, no dietary changes, no extra time away from family or work, and it can be completed as part of any patient office visit.3

The clinical performance of the Shield assay was validated using a set of 309 patient samples, including 92 with CRC, 51 with advanced adenomas and 166 normal cases. CRC patient samples were accrued across six unique cohorts collected in the U.S., Canada and the EU, and samples with advanced adenoma and normal cases were collected in the U.S. Subjects were balanced by age (a mean of 64 years old) and gender. The Shield assay demonstrated 91% sensitivity (detection rate) for CRC (95% confidence interval [CI]: 84% – 95%), including 90% for Stage I, 97% for Stage II, and 86% for Stage III CRC. The assay also demonstrated 20% sensitivity for advanced adenomas (95% CI; 11% – 32%) and 92% specificity (true negative rate) in normal cases.

"The availability of the Shield test represents a major milestone in our commitment to transform cancer screening. We have developed highly sensitive technology to detect early-stage cancers with a simple blood draw," said AmirAli Talasaz, Guardant Health co-CEO. "Colorectal cancer screening is the start of this journey. We will soon expand into multi-cancer screening, including lung, pancreas and others, where we believe cancer screening can save lives."

Why CRC screening compliance matters

More than 75% of people who die from CRC today are not up to date with recommended screening.4 Cancer underscreening is an important factor contributing to the high cancer mortality rate in underserved populations. For example, in the area of CRC, only 59% of individuals age 50 and older who are Hispanic and 65% of individuals who are Black/African American are up to date with recommended screenings, compared to 68% of individuals who are white.5

"The value of colorectal cancer screening is well-established for individuals at average risk, age 45 and older," said J. Leonard Lichtenfeld, MD, MACP, Chief Medical Officer, Jasper Health. "But the benefits of screening have been limited due to the suboptimal completion rates of stool tests and colonoscopy. A high-sensitivity test that can be completed with a convenient blood draw during any healthcare provider visit can help patients overcome the barriers to adherence and help healthcare professionals identify more cancers at their earliest stages, when they are most treatable."

"Colorectal cancer is largely preventable if caught early, yet barriers to screening tests remain. It can be especially challenging for underserved populations to get screened. These barriers include lack of healthcare access, limited capacity in healthcare systems, transportation challenges, childcare, and lack of paid leave from work," said Anjee Davis, MPPA, president of Fight Colorectal Cancer. "As the research continues to progress for blood-based tests, the availability of a test like this is a welcomed addition to the tools we can use to prevent cancer. This has the potential to improve access to screening for underserved populations and dramatically improve overall screening rates."

How the Shield assay was developed

The Shield test detects early signs of CRC signals in the bloodstream. The assay was developed using multiple cohorts comprising 2,089 subjects with CRC, 357 with advanced adenoma and 3,757 normal subjects. The samples were collected within both prospective screening collections designed to capture the intended use population and retrospective case-control cohorts designed to enrich for subjects with CRC.

Where the Shield test is available currently

Shield is now available for eligible individuals by prescription only through healthcare professionals. This LDT (Laboratory Developed Test) is intended to be complementary to, and not a replacement for, current recommended CRC screening methods. A negative result does not rule out the presence of cancer. Patients with an abnormal blood-based screening result should be referred for a diagnostic colonoscopic evaluation.

What’s next for the Shield test

This year Guardant Health plans to share results of the ECLIPSE clinical study (NCT04136002) to demonstrate the performance of the Shield test to detect early signs of CRC in individuals ages 45-84 who are at average risk. The prospective, multi-site registrational study is one of the largest cancer screening studies of its kind, with enrollment of more than 12,750 patients from across the U.S. Subject to positive results, the study will support a premarket approval (PMA) submission to the U.S. Food and Drug Administration.

On May 2, 2022 Fresenius Kabi reported it has introduced Bortezomib for Injection, a new generic equivalent to Velcade in the U.S. and the newest addition to the most comprehensive injectable oncology portfolio in the industry (Press release, Fresenius Kabi Oncology, MAY 2, 2022, View Source [SID1234613323]).

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Fresenius Kabi Bortezomib for Injection is available in a 3.5 mg per 10 mL single-dose vial presentation for subcutaneous (SQ) or intravenous (IV) use.

Fresenius Kabi Bortezomib for Injection is an affordable treatment option for adult patients with multiple myeloma and mantle cell lymphoma. Multiple myeloma represents nearly 2 percent of all new cancer cases in the U.S. and is expected to double in 20 years.1 Mantle cell lymphoma represents approximately 5 percent of all non-Hodgkin’s lymphoma diagnoses annually, and there are approximately 4,000 new cases each year.2,3

"Adding a generic equivalent Bortezomib for Injection to our expansive oncology portfolio reflects our continued plan of expanding access to affordable cancer therapies," said John Ducker, president and CEO of Fresenius Kabi USA. "We’re pleased to be able to provide an affordable option to patients and we are especially pleased that Fresenius Kabi Bortezomib for Injection is produced at one of our U.S. facilities."

Fresenius Kabi offers more than 30 different oncology drugs in the U.S., and nearly 90 percent are formulated, filled and finished in the U.S. Bortezomib for Injection is the newest example of the company’s commitment to investing "More in America." This effort is focused on providing more supply, more science, more support and more care to its customers and the patients they serve in the U.S. Fresenius Kabi has invested nearly $1 billion to modernize and expand advanced U.S. pharmaceutical production and distribution facilities.

Bortezomib for Injection, along with other oncology medicines, is part of the company’s KabiConnect program, a recent expansion of its KabiCare patient support program that offers copay assistance to eligible U.S. patients. The program can lower out-of-pocket costs to as little as $0 per month for eligible patients. To determine eligibility, patients should speak to their physician. Enrollment is a simple online process. Details can be found on the KabiCare website at kabicare.us.

Important Safety Information
INDICATIONS AND USAGE

Bortezomib for Injection is a proteasome inhibitor indicated for:

treatment of adult patients with multiple myeloma
treatment of adult patients with mantle cell lymphoma
IMPORTANT SAFETY INFORMATION

Bortezomib for Injection is contraindicated in patients with hypersensitivity (not including local reactions) to bortezomib, boron, or mannitol, including anaphylactic reactions. Bortezomib for Injection is contraindicated for intrathecal administration.

For subcutaneous or intravenous use only. Each route of administration has a different reconstituted concentration. Exercise caution when calculating the volume to be administered.

Peripheral neuropathy: Manage with dose modification or discontinuation. Patients with pre-existing severe neuropathy should be treated with Bortezomib for Injection only after careful risk-benefit assessment.

Hypotension: Use caution when treating patients taking antihypertensives, with a history of syncope, or with dehydration.

Cardiac Toxicity: Worsening of and development of cardiac failure has occurred. Closely monitor patients with existing heart disease or risk factors for heart disease.

Pulmonary Toxicity: Acute respiratory syndromes have occurred. Monitor closely for new or worsening symptoms and consider interrupting Bortezomib for Injection therapy.

Posterior Reversible Encephalopathy Syndrome: Consider MRI imaging for onset of visual or neurological symptoms; discontinue Bortezomib for Injection if suspected.

Gastrointestinal Toxicity: Nausea, diarrhea, constipation, and vomiting may require use of antiemetic and antidiarrheal medications or fluid replacement.

Thrombocytopenia or Neutropenia: Monitor complete blood counts regularly throughout treatment.

Tumor Lysis Syndrome: Closely monitor patients with high tumor burden.

Hepatic Toxicity: Monitor hepatic enzymes during treatment. Interrupt Bortezomib for Injection therapy to assess reversibility.

Thrombotic Microangiopathy: Monitor for signs and symptoms. Discontinue Bortezomib for Injection if suspected.

Embryo-Fetal Toxicity: Bortezomib for Injection can cause fetal harm. Advise females of reproductive potential and males with female partners of reproductive potential of the potential risk to a fetus and to use effective contraception. Most commonly reported adverse reactions (incidence ≥ 20%) in clinical studies include nausea, diarrhea, thrombocytopenia, neutropenia, peripheral neuropathy, fatigue, neuralgia, anemia, leukopenia, constipation, vomiting, lymphopenia, rash, pyrexia, and anorexia.

On May 2, 2022 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported the finalized data from the Phase 1 clinical trial of SACT-1, a repurposed small molecule drug targeting Neuroblastoma and potentially other cancer types (Press release, Aptorum, MAY 2, 2022, View Source [SID1234613322]).

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Following the announcement of the Phase 1 clinical trial of SACT-1 in January 2022, Aptorum is pleased to announce further data updates from the trial conducted by an independent clinical contract research organization. The Phase 1 clinical trial of SACT-1 was an open-label, randomized, 3-period, 3-sequence, single-dose crossover bioavailability and food effect study of SACT-1 (oral suspension) in healthy adult volunteers. The primary objective of this study was to assess the relative bioavailability of 150 mg of SACT-1 (oral suspension) under fasted and fed conditions. The secondary objectives were to evaluate the safety, tolerability and any potential QT prolongation after a single oral administration of 150mg of the studied drug under fasted and fed conditions in healthy adult subjects. The study treatments were well tolerated and no subjects were discontinued from study participation because of adverse events. No serious adverse events were reported during the study. The phase 1 clinical data also suggested that any QT interval after oral administration of SACT-1 at 150mg was well within clinically acceptable limits. Regarding the relative bioavailability under the Fed vs Fasted condition, the AUC0-tlast, AUC0-∝ and Cmax ratio of SACT-1 were determined to be 189.87%, 189.43%, and 205.25% respectively.

Dr. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented: "Further to our previous announcements, we are very encouraged by the impressive safety data even at a relatively high dosage. The relative bioavailability data also enabled us to estimate the starting dose for pediatric neuroblastoma patients via PK modeling. We are planning to meet with the US FDA for an end of Phase 1 meeting as soon as possible and are targeting for submission for a Phase 1b/2a clinical trial in neuroblastoma patients."

About SACT-1

SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in our preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed). SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.