On July 7, 2020 Senhwa Biosciences, Inc. (TPEx: 6492), a clinical-stage biopharmaceutical company focused on Next Generation DNA Damage Response (DDR) therapeutics for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) Designation for its drug Silmitasertib, a Casein Kinase 2 (CK2) inhibitor, being developed as a treatment for recurrent sonic hedgehog (SHH) medulloblastoma (Press release, Senhwa Biosciences, JUL 7, 2020, View Source [SID1234561730]). With the RPD designation, Senhwa Biosciences is eligible for a Priority Review Voucher (PRV) which can be used for a subsequent marketing application, and may be sold or transferred. In August 2015, AbbVie bought a PRV from United Therapeutics Corp for $350 million which allowed AbbVie to accelerate one of its drug’s FDA review process.

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Medulloblastoma is the most common cancerous brain tumor in children. Treatment for medulloblastoma usually includes surgery, followed by radiation or chemotherapy, or both. Currently there is no targeted drug available. Recurrent SHH medulloblastoma is recognized as one of the four major sub-groups of medulloblastoma, with about 80-100 new cases per year. The FDA grants RPD Designation for serious or life-threatening diseases that primarily affect people from birth to 18 years old and which affect fewer than 200,000 people in the U.S.

Senhwa’s clinical partner, the Pediatric Brain Tumor Consortium (PBTC, www.pbtc.org) is currently conducting a Phase I/II and Surgical study of Silmitasertib, in both children and adults with recurrent SHH medulloblastoma, at its participating member academic medical centers and children’s hospitals across the United States. This clinical trial is sponsored by the PBTC and funded through the Consortium grant awarded by the National Institute of Health (NIH) – Cancer Therapy Evaluation Program (CTEP).

Silmitasertib is safe and well-tolerated in humans. To date, three Phase I trials of Silmitasertib in cancer patients have been completed; currently, there are one ongoing Phase I and two ongoing Phase II studies of Silmitasertib.

About Silmitasertib
Silmitasertib is a first-in-class small molecule drug which targets CK2 and acts as a CK2-inhibitor. A Phase I/II study has shown that Silmitasertib achieved clinical benefit, resulting in stable disease and extending the duration of treatment in patients who are unresponsive to standard of care therapy. The combination of Silmitasertib with DNA-damaging agents such as gemcitabine (Gemzar) plus cisplatin (Platinol) has been shown to synergistically improve the efficacy of cholangiocarcinoma (CCA) treatments. In December 2016, Silmitasertib was granted Orphan Drug Designation by the US FDA for the treatment of CCA.

On July 7, 2020 HiberCell, a biotechnology company developing novel therapeutics for cancer relapse and metastasis, reported the acquisition of Biothera Pharmaceuticals’ (Biothera) Imprime PGG program (Press release, HiberCell, JUL 7, 2020, View Source [SID1234561714]). HiberCell will integrate Biothera’s Minnesota-based staff, operations and other assets into the company.

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Imprime PGG is a novel innate immune activator that binds to the dectin-1 receptor and activates innate immunity, reprogramming the immunosuppressive tumor microenvironment to enhance antigen presentation, trigger T cell activation and ultimately enhance the immune response against tumors. Phase II clinical studies of Imprime PGG in combination with checkpoint inhibition demonstrated compelling mechanistic proof-of-concept data including the activation of innate and adaptive immunity resulting in improved overall survival, overall response and disease control rates in metastatic triple negative breast cancer (mTNBC).

"Imprime PGG has provided meaningful responses in Phase 2 clinical trials for metastatic cancer patients. HiberCell will build on these data and their potential to transform the lives of people living with metastatic breast cancer," said Alan C. Rigby, Ph.D., co-founder and chief executive officer of HiberCell. "This acquisition signifies HiberCell’s transition into a clinical stage company with a broad portfolio of assets targeting cancer relapse and metastasis."

HiberCell’s scientific approach focuses on three mechanistic pillars of cancer dormancy: adaptive stress biology, disseminated tumor cells and the tumor microenvironment. By exploring each, the company continues to assemble a pipeline of differentiated therapeutic opportunities for relapsed and metastatic cancer. Importantly, the Imprime PGG program adds to the HiberCell pipeline a later-stage innate immune activator supported by a robust translational oncology platform that has demonstrated a ‘reprogramming’ and activation of the tumor microenvironment.

In Phase II clinical studies, Imprime PGG paired with the checkpoint inhibitor Keytruda resulted in encouraging benefits across all clinical measures in patients with metastatic breast cancer, including TNBC. HiberCell will initiate additional clinical studies in TNBC and other indications to further demonstrate the safety and efficacy of various Imprime PGG combination therapies.

"We are very proud of the translational platform and science behind Imprime PGG," said Jeremy R. Graff, Ph.D., chief development officer of HiberCell. "We look forward to building a robust clinical pipeline in support of the meaningful data Imprime PGG has generated to date and are thrilled to continue this work at HiberCell."

On July 7, 2020 Quantum Leap Healthcare Collaborative (Quantum Leap) reorted the initiation of an investigational treatment arm with Byondis’ [vic-]trastuzumab duocarmazine (SYD985) in the ongoing I-SPY 2 TRIAL for neoadjuvant treatment of locally advanced breast cancer (Press release, QuantumLeap, JUL 7, 2020, View Source [SID1234561732]). This treatment arm will focus on treatment for HER2 low early-stage breast cancer.

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This study arm will evaluate if SYD985 will limit tumor growth in a population of patients whose tumors are not normally treated with HER2-targeted therapies. Specifically, these tumors have low expression of HER2, and require better HER2-targeted drugs in order to be as effectively treated as HER2+ patients.

SYD985 is being developed for the treatment of HER2 expressing or HER2 mutant tumors. In nonclinical and clinical studies, SYD985 has demonstrated potent antitumor activity. Based on the preliminary clinical observations in a Phase 1 study, SYD985 demonstrated antitumor activity in HER2-expressing cancers including breast cancer. Researchers and clinicians believe SYD985 inhibits tumor growth for the following reasons: SYD985 is a next generation ADC, comprised of the mAb (monoclonal antibody) trastuzumab and a cleavable linker-drug called valine-citrulline-seco-DUocarmycin-hydroxyBenzamide-Azaindole (vc-seco-DUBA). The antibody part of SYD895 binds to HER2 on the surface of the cancer cell and is internalized by the cell. After proteolytic cleavage of the linker, the inactive cytotoxin is activated and DNA damage is induced, resulting in death of dividing and non-dividing tumor cells. An earlier phase I study (SYD985.001) in patients with histologically-confirmed, locally advanced or metastatic tumors, concluded that SYD985 is effective in patients with high HER2 levels in their tumor, as well as in a subset of patients with lower levels of expression of the HER2 protein (HER2- low).

The SYD985 arm of the I-SPY 2 clinical trial will be open for randomization to the HER2-low subset of the HER2- I-SPY 2 population, further defined as HER2-low by HER2 immuno histo-chemical staining (i.e. IHC 1+ and 2+) and in-situ hybridization. Tumors will be further characterized by genetic testing employing MammaPrint (MP1 or MP2) and by hormone receptor status (HR+ or HR-). Within the HER2 negative population, patients will be excluded for HER2 IHC 0 and ISH negative.

"This I-SPY 2 TRIAL arm is designed to test a less toxic and a potentially highly effective approach where we need better options for patients." stated Dr. Laura Esserman, Lead PI for the I SPY 2 TRIALs. "Low levels of HER2 protein expression are not sufficient to generate a response to traditional HER 2 treatment currently on the market. This particular agent works differently and is a promising way to deliver a targeted toxin. We need to continue to push to find better therapies. The I-SPY investigators are excited to partner with Byondis to test this agent in I-SPY 2."

The I-SPY 2 TRIAL, sponsored by Quantum Leap, is a standing phase 2 randomized, controlled, multicenter study with an innovative Bayesian adaptive design aimed to rapidly screen and identify promising new treatments in specific subgroups of women with newly-diagnosed, high-risk (high likelihood of recurrence), locally-advanced breast cancer (Stage II/III).

Byondis will supply the investigational drug and provide financial and regulatory support. Quantum Leap, as sponsor, will provide the clinical sites and clinical expertise.

On July 7, 2020 Nucleai (www.Nucleaimd.com), an Israeli start-up providing an artificial intelligence-powered precision oncology platform for research and treatment decisions and Debiopharm (www.debiopharm.com), a Swiss-based biopharmaceutical company specializing in drug development, manufacturing and digital health investment, reported the Series-A initial closing of $6.5M (Press release, Nucleai, JUL 7, 2020, View Source [SID1234561733]).

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In recent years, cancer immunotherapies have revolutionized cancer treatment, providing long term benefits to certain patient populations. However, the efficacy of these treatments is often observed in only a small subset of patients. Without an adequate way to predict drug response, patients risk receiving ineffective treatment accompanied by unnecessary payor coverage. In light of the high percentage of clinical trial failure, this lack of predictive precision can also have a heavy impact on pharmaceutical companies conducting costly clinical research. Nucleai’s core technology analyzes large and unique datasets of tissue images using computer vision and machine learning methods to model the spatial characteristics of both the tumor and the patient’s immune system, creating unique signatures that are predictive of patient response. In a recent work performed in collaboration with leading researchers and published at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 conference, Nucleai has demonstrated the predictive power of tumor microenvironment AI-based spatial analysis for breast cancer.

Through this investment, Debiopharm and Nucleai will collaborate in order to leverage Debiopharm’s 40 years of pharmaceutical expertise towards an acceleration of predictive biomarker discovery for drug response and the improvement of data from clinical trials involving cancer patients. The company intends to use the funds to further progress the development of its advanced platform for use in Immuno-oncology and other diseases. The funds will be used also to further progress its commercial reach to pharmaceutical and biotech companies.

"We are excited to have Debiopharm’s corporate VC as our lead investor along with our longterm partner investors Vertex Ventures and Grove Ventures. Debiopharm is bringing decades of pharmaceutical expertise which will assist in accelerating our development and market reach," explained Avi Veidman, Nucleai’s CEO. "Nucleai has multiple revenue-generating, commercial partnerships, with leading Immunotherapies pharma companies and US-based payors. We plan to use these funds to expand our offering to additional indications and diseases as well as to increase our commercial footprint substantially."

Founded in Israel, a growing hotspot for computational biology innovation, Nucleai was founded by former members of an elite technological unit of Israeli intelligence corps specializing in artificial intelligence and big data: Avi Veidman (CEO), Eliron Amir (COO), and Lotan Chorev (VP R&D). This Tel Aviv hub positions the start-up to leverage a wealth of valuable resources including a centralized medical system, 25 years of EMR data, and top AI talents.

"Our team is thrilled to embark on this adventure to develop and further understand the extent to which AI can help pathologists and oncologists become more precise in both diagnosis and prediction," explained Tanja Dowe, CEO of Debiopharm Innovation Fund. "We recognize the huge impact that Nucleai’s AI-powered platform, could have on clinical research for pharmaceutical treatments."

Avi Veidman, Nucleai’s CEO added, "The battle between the tumor and the immune cells is clearly visible by inspecting the pathology slide, just like a satellite image of a battlefield. Our AI platform analyzes the hundreds of thousands of cells in a slide, examines the interplay between the tumor and the immune system and matches the right patient to the right drug based on these characteristics".

"Our solution is purely based on software which is embedded into the biomarker researcher workflow. This allows us to scale our solution rapidly, to provide service to a large number of pharmaceutical and biotech partners as well as to patients," expressed Eliron Amir, COO of Nucleai. "It is an elegant solution to a complex problem. There is no need for an expensive wet-lab biology operation. Our cloud-based solution allows us to gather huge amounts of immuno-oncology data from different sources, creating complex insights that a single pharma or institute cannot generate by itself."

On July 7, 2020 Crescendo Biologics Ltd (Crescendo), the drug developer of novel, targeted T cell enhancing therapeutics reported that it has signed a pre-clinical collaboration agreement with Orano Med, a nuclear biotech company developing innovative targeted therapies in oncology (Press release, Crescendo Biologics, JUL 7, 2020, View Source [SID1234561698]).

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Orano Med is developing a new generation of Targeted Alpha Therapies (TAT) using the unique properties of lead-212 (²¹²Pb), a rare radioisotope. With this innovative approach, Orano Med is generating novel therapeutics capable of specifically recognising and destroying cancer cells while limiting the impact on nearby healthy tissues, a cutting-edge treatment for cancers with high unmet need.

Crescendo and Orano Med are working to exemplify the benefit of using Humabody VH for precise targeting of Orano Med’s TAT.

Theodora Harold, CEO of Crescendo Biologics, commented:
"Orano Med is one of the leaders in the field of nuclear medicine. Collaborating on its Targeted Alpha Therapy is an exciting opportunity for Crescendo to demonstrate the advantages of Humabody VHs for exquisitely targeted delivery of a potent alpha-emitting payload. We welcome the opportunity to work with the team at Orano Med."