On May 6, 2020 NANOBIOTIX (Paris:NANO) (Euronext: NANO – ISIN: FR0011341205 – the "Company"), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported that the protocol for the first trial from its clinical collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson) has been designated as "safe to proceed" by the US Food and Drug Administration (FDA) (Press release, Nanobiotix, MAY 6, 2020, View Source [SID1234557160]). The trial was co-developed with Nanobiotix and MD Anderson is the sponsor and executor of the trial.

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The investigational new drug application (IND), covers a phase I study evaluating the safety and feasibility of NBTXR3 activated by radiation therapy for patients with locally advanced (LAPC) or borderline resectable (BRPC) cases of pancreatic ductal adenocarcinoma (PDAC). This trial is the first to evaluate NBTXR3 activated by radiation therapy in pancreatic cancer.

Significant Unmet Needs and Opportunity in Pancreatic Cancer

Pancreatic cancer is a rare, deadly disease. Given that surgery with R0 resection (i.e. macroscopically complete tumor removal with negative microscopic surgical margins) remains the only hope for long-term survival, clinical trials have investigated various neoadjuvant strategies—wherein patients receive anti-cancer drugs or radiation prior to surgery—to increase the surgery-eligible population while also increasing the R0 resection rate.

In support of the rationale for neoadjuvant therapy, a retrospective analysis demonstrated a near doubling in overall survival (OS) in PDAC patients who underwent surgery, which was attributed, at least in part, to the increased proportion of BRPC patients who became eligible for surgery as a result of neoadjuvant intervention. Importantly, there are also select cases of LAPC patients being considered for surgical resection based on their response to therapy. Given the poor prognosis of PDAC, therapeutic regimens able to increase the proportion of BRPC and LAPC patients eligible for surgery could improve survival outcomes in this population with unmet need.

A Phase I Study Evaluating NBTXR3 Activated by Radiation Therapy in Patients with PADC

The MD Anderson trial is an open-label, single-arm, prospective phase I study consisting of two parts: (i) dose-escalation to determine the recommended phase 2 dose (RP2D); and (ii) expansion at RP2D.

The patient population will include adults (age ≥ 18 years) with BRPC or LAPC that are radiographically non-metastatic at screening, and that have not previously received radiation therapy or surgery for pancreatic cancer. The number of participants enrolled will be determined based on the maximum number required to establish the RP2D. Up to 24 subjects will be enrolled, including a maximum of 12 subjects with LAPC for the dose-finding part. Twelve additional subjects with either LAPC or BRPC will be enrolled for the RP2D expansion. The planned enrollment period is 18 months. The first patient should be injected the summer of 2020.

The objectives of the study are the determination of dose-limiting toxicity (DLT), the maximum tolerated dose (MTD), and the RP2D.

About NBTXR3

NBTXR3 is a first-in-class product designed to destroy tumors through physical cell death when activated by radiotherapy. NBTXR3 has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide radiation therapy standards of care. The physical mode of action of NBTXR3 makes it applicable across solid tumors such as lung, prostate, liver, glioblastoma, pancreas, and breast cancers.

NBTXR3 is actively being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) of the oral cavity or oropharynx in elderly and frail patients unable to receive chemotherapy or cetuximab with limited therapeutic options. Promising results have been observed in the phase I trial regarding local control. In the United States, the company has started the regulatory IND process to commence a phase III clinical trial in locally advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. Pursuant to an effective IND, the Company has launched a clinical trial of NBTXR3 activated by radiotherapy in combination with anti-PD-1 antibodies in locoregional recurrent (LRR) or recurrent and metastatic (R/M) HNSCC amenable to re-irradiation of the HN and lung or liver metastases (mets) from any primary cancer eligible for anti-PD-1.

The other ongoing NBTXR3 trials are treating patients with hepatocellular carcinoma (HCC) or liver metastases, locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma. The company has a large-scale, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center (9 new phase I/II clinical trials in the United States) to evaluate NBTXR3 across head and neck, pancreatic, lung, gastrointestinal and advanced cancers.

On May 6, 2020 PinCell, a biotechnology company developing novel therapies for rare dermatological diseases, reported the appointment of Gabriella Camboni, M.D., as Chief Executive Officer of PinCell and Member of the Board of Directors, and Luigi Costa as Chairman of the Board (Press release, PinCell, MAY 6, 2020, View Source [SID1234557159]). Paola Pozzi, Partner at Sofinnova Partners, also joins PinCell’s Board following a successful seed financing of €1.65 million led by Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The financing was part of a series of investments recently made through The Sofinnova Telethon Fund, the largest fund in Italy dedicated to early-stage biotech startups targeting cures for rare and genetic diseases.

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Dr. Camboni is a clinical pharmacologist with 18 years of experience in oncology drug development. She is Chief Executive Officer of BiovelocITA, Italy’s first biotech accelerator, co-founded by Sofinnova Partners in 2015. Dr. Camboni is also co-founder and Chief Operating Officer at Ethical Oncology Science (EOS), an Italian startup dedicated to translational medicine in oncology. Previously, Dr. Camboni co-founded and acted as Head of Development at Novuspharma, a biopharma developing innovative treatments for cancer. Both EOS and Novuspharma are part of Sofinnova Partners’ portfolio alumni.

"I am happy to have the opportunity to help develop a game-changing treatment together with an outstanding scientific team and my long-term associates of Sofinnova Partners," commented Dr. Camboni.

PinCell also bolstered its Board of Directors with the appointment of Luigi Costa as Chairman of the Board. Mr. Costa has over 25 years of international pharmaceutical and biotech experience. He is an Entrepreneur in Residence at Sofinnova Partners and was the CEO of Nordic Nanovector, a biopharma developing novel targeted therapies for cancer. He also served as Board Member of Oncopeptides AS, a clinical stage biotech company developing a new treatment for Multiple Mieloma. Previously Mr. Costa was Regional Head International at Onyx Pharmaceuticals, acquired by Amgen in 2014, and held several leadership positions with Amgen including Head of International Oncology Franchise, General Manager of Italy and President of France, Amgen’s largest market outside the US. He also held various leadership positions of increasing responsibility with Eli Lilly both in Europe and in the US.

"I am very pleased to have the opportunity to support PinCell in the development of its innovative treatment for a severe autoimmune disease. I am confident that PinCell’s outstanding scientific team, together with my colleagues at Sofinnova Partners, will be able to bring a new therapeutic option to patients," said Mr. Costa.

PinCell’s lead program targets a rare autoimmune bullous disease called Pemphigus, which is characterized by blistering and erosions of the skin and mucous membranes. The condition is chronic, debilitating and potentially life-threatening. PinCell’s unique solution uses an innovative mechanism that inhibits the development of the typical skin blisters, without suppressing the immune system. This is a central factor that makes the treatment less prone to immune-related side effects, enabling patients to have a better quality of life.

Ms. Pozzi commented: "We are pleased to invest in this exceptional team of serial entrepreneurs who have significant clinical expertise in dermatological disorders. They have built strong relationships with US pharma-experienced consultants and international patients’ associations."

The financing secured last month will advance the company’s development of first-in-class anti-inflammatory therapies for the treatment of rare and severe skin diseases with limited therapeutic options.

Carlo Pincelli, M.D., Professor of Dermatology at the University of Modena and Reggio Emilia and Co-founder of PinCell, said: "We are delighted to have secured financing from Sofinnova Partners, an investor with unparalleled authority and expertise in the biotech domain, as well as the support of experienced biotech executives. Together we are confident we will progress towards clinical studies for the benefit of patients suffering from these debilitating skin conditions."

On May 6, 2020 Noxopharm, a clinical-stage Australian oncology drug development company, reported that it has announced the details of the radiographic review of its DARRT-1 clinical study and is pleased to report a 27% incidence of abscopal response in soft tissue lesions using a combination of Veyonda and low-dose radiotherapy (Press release, Noxopharm, MAY 6, 2020, View Source [SID1234557158]).

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An abscopal response, a highly prized clinical response, involves radiation directed at a single tumor which triggers resulting shrinkage of tumors well outside of the field of radiation. It is assumed to be an immune response in the irradiated tumor triggered by using a low, minimally-ablative dose of radiation. Once acknowledged as a very rare event, the growing use of immuno-oncology drugs in combination with radiotherapy has led to increasing reports of abscopal responses in a range of cancers, including lung, breast, and urogenital cancers and melanoma. No such reports have come from studies in prostate cancer with checkpoint inhibitors either alone or in combination with radiotherapy, leading to this cancer gaining a reputation as a "non-immunological" cancer.

The DARRT-1 study involved 15 men with mCRPC post-taxane and ADT therapy. They received experimental sphingosine-1-phosphate inhibitor, Veyonda, in combination with a palliative dose of external beam radiotherapy to a single tumor, typically a lymph node cluster. An abscopal response was determined on the basis of scans of other nonirradiated soft tissue lesions.

Dr. Graham Kelly, Noxopharm CEO, said, "To our knowledge, this is the first time that anyone has been able to obtain a meaningful abscopal response rate in prostate cancer. Prostate cancer has developed a reputation as a cancer with poor immune responsiveness, but this outcome suggests that this isn’t the case when a drug with the appropriate effect on the immune system is used. Today’s result positions Veyonda at the forefront of this emerging area of oncology and suggests that we have an exciting new prospective treatment for end-stage prostate cancer."

On May 6, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, reported financial results for the quarter that ended March 31, 2020, along with an update on the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 6, 2020, View Source [SID1234557156]).

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"During the first quarter, we continued to advance five programs across both the AAV and Lenti platforms," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "Notably this quarter, we completed patient treatment in the low dose cohort for RP-A501 in the Phase 1 Danon Disease trial with no dose-limiting toxicities, and are now pleased to report FDA and IDSMC clearance to advance to a higher dose cohort. This has been an important priority for us as Danon Disease affects so many young patients with devastating heart, muscle and CNS manifestations. We also continued to progress our clinical programs for FA, LAD-I, and PKD. Rocket is at an exciting stage of growth, with a rich pipeline of development-stage opportunities and the potential to transform the lives of patients afflicted with rare disease. In the coming months, we look forward to presenting updated FA ‘Process A’ and LAD-I ‘Process B’ data at ASGCT (Free ASGCT Whitepaper), treating our first patient in a higher dose cohort for RP-A501, and bringing our fifth program, IMO, to the clinic."

Dr. Shah continued, "With regard to the COVID-19 pandemic, we are well-positioned overall to continue our progress while we monitor and adjust to this challenging global crisis. We have experienced modest COVID-19-related pauses in patient enrollment and follow-up that are being managed on a patient-by-patient basis, along with some delays in data collection. Nonetheless, we remain on track for providing updates on our lentiviral pipeline. Assuming that COVID-19 associated delays have shorter-term impact, we remain committed to providing data for Danon by the end of the year. Lastly, we continue to actively monitor the pandemic and refine operations to support the complete safety and well-being of our patients, employees and community."

Key Pipelines and Operational Updates

First cohort of patients completed in Phase 1 trial of RP-A501 for Danon Disease, with U.S. Food and Drug Administration (FDA) and Independent Data Safety Monitoring Committee (IDSMC) clearance to initiate a higher dose cohort. Rocket has treated the first three patients at the low dose of 6.7×1013 vector genomes (vg)/kilogram (kg). No investigational product (IP)-related dose-limiting safety concerns have been observed in any of the three patients treated. The low dose cohort demonstrates a favorable safety profile, and the Company has received clearance from the IDSMC and the FDA to move to a higher dose cohort. Patient treatment in this cohort is anticipated in the third quarter, with preliminary Danon data readout in the fourth quarter.
The Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) clinical studies have continued to progress. Updated data from FANCOLEN-I (utilizing "Process A") will be presented at ASGCT (Free ASGCT Whitepaper), as will updates from the Phase 1 LAD-I study utilizing "Process B." Updates on FA "Process B" data will be presented in the fourth quarter.
The Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) facility in Cranbury, New Jersey resumes construction after COVID-19 associated delays. Build-out of the New Jersey facility experienced some construction delays associated with the COVID-19 pandemic, but resumed on May 4, 2020. Occupancy in the new facility is still anticipated in the first half of 2020. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. As previously guided, the first cGMP clinical product release is expected in 2021.
Recent publications for FA and Danon Disease provide greater insight into Rocket’s approach for ongoing clinical trials. In February, the peer-reviewed journal Annals of Hematology published Rocket’s comprehensive review of somatic mosaicism in Fanconi Anemia (FA), written in collaboration with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and other international research partners. The article, entitled, "Mosaicism in Fanconi Anemia: Concise review and evaluation of published cases with focus on clinical course of blood count normalization," highlights research describing mosaicism in FA and supports Rocket’s approach of treating patients with RP-L102 without any pre-treatment conditioning measures. In March, Science Translational Medicine published positive preclinical data on Rocket’s Danon program, demonstrating that vector-mediated transfer of LAMP2B to deficient mice improved heart function and survival. The article, "AAV9.LAMP2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease," underscores the promise of Rocket’s AAV-based gene therapy candidate for Danon, RP-A501.
Rocket recognizes Rare Disease Day with an event at New York City’s Carnegie Hall. On February 29, 2020, Rocket hosted its second annual Rare Disease Day event highlighting the theme "I Am Rare, Hear Me Roar." Over 200 members of the rare disease and broader New York City biotech communities heard from patients and their families, learning more about the impact of rare disease and how to become partners in the fight to find new treatment options. The event culminated with the lighting of the Empire State Building in Rare Disease Day colors, honoring all those affected by rare disease.
Anticipated Milestones

FA (RP-L102)
Additional "Process A" data update (2Q)
Preliminary "Process B" data (4Q)
Danon Disease (RP-A501)
First patient treatment in higher dose (3Q)
Preliminary Phase 1 data (4Q)
LAD-I (RP-L201)
Phase 1 data update on first patient (2Q)
Initiate Phase 2 study (4Q)
Phase 1 data update (4Q)
PKD (RP-L301)
First patient treatment (3Q, previously 2Q)
Preliminary Phase 1 data (4Q)
IMO (RP-L401)
Initiation of clinical study (4Q)
Upcoming Investor Conferences

BofA Securities 2020 Virtual Health Care Conference, May 13, 2020
Jefferies Virtual Healthcare Conference, June 2-4, 2020
Third Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of March 31, 2020, were $275.9 million.
Debt. Our balance sheet includes $52.0 million of fully convertible notes.
R&D expenses. Research and development expenses were $17.0 million for the three months ended March 31, 2020, compared to $15.1 million for the three months ended March 31, 2019. The increase was primarily driven by an increase in clinical trial expenses of $1.5 million.
G&A expenses. General and administrative expenses were $7.2 million for the three months ended March 31, 2020, compared to $3.8 million for the three months ended March 31, 2019. The increase was primarily driven by fees incurred for the convertible notes exchange of $1.6 million, and an increase in compensation expense of $1.3 million due to increased headcount.
Net loss. Net loss was $24.7 million or $0.45 per share (basic and diluted) for the three months ended March 31, 2020, compared to $19.5 million or $0.43 per share (basic and diluted) for the three months ended March 31, 2019.
Shares outstanding. 55,126,474 shares of common stock were outstanding as of March 31, 2020.
Financial Guidance

Cash position. As of March 31, 2020, we had cash, cash equivalents and investments of $275.9 million. Rocket expects such resources will be sufficient to fund its operations into 2022.

On May 6, 2020 InteRNA Technologies reported regulatory approvals in the Netherlands and Belgium for the initiation of the first-in-human (Phase I) clinical trial testing its lead microRNA candidate, INT-1B3, in patients with advanced solid tumors (Press release, InteRNA Technologies, MAY 6, 2020, View Source [SID1234557155]). INT-1B3 is a lipid nanoparticle (LNP) formulated, chemically modified miR-193a-3p mimic that can be delivered by systemic administration to cancer cells. Due to the ongoing COVID-19 pandemic, clinical trial initiation has been delayed and is now anticipated to start during H2 2020. The Company will provide an update once the trial has officially launched.

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The study is a multicentric, open-label and multiple ascending dose clinical trial that will investigate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of INT-1B3 in patients with advanced solid tumors. The study is expected to enroll a total of up to 80 patients at 12 clinical centers in the United States and Europe. The first part of the trial is a Phase Ia dose escalation study, conducted in the Netherlands and Belgium, which will enroll approximately 30 patients with advanced solid tumors. These patients will receive INT-1B3 via infusions twice weekly in 21-day cycles. Subsequently, approximately 50 patients with either hepatocellular carcinoma or triple negative breast cancer will be enrolled in the Phase Ib dose expansion part of the trial. Initial data readout from the Phase Ia study is expected in the end of 2021.

"The multi-targeted nature of microRNAs represents a novel approach to treating a broad range of cancer indications by effectively targeting several biochemical pathways simultaneously," said Dr. Roel Schaapveld, CEO of InteRNA Technologies. "Driven by the promise of microRNAs as a treatment modality in cancer, our understanding of the underlying mechanism of action of INT-1B3 and the identification of a delivery vehicle for optimized performance, our primary focus has been on preparing our lead candidate for first-in-human clinical trials. As such, we are truly excited to bring INT-1B3 into the clinic and to gain deeper insights on how it reacts against solid tumors."

"INT-1B3 is a promising and novel anti-cancer approach with anti-tumor potential involving direct effects on tumor cells and modulation of the immunosuppressive tumor microenvironment leading to immune system activation," added Prof. Dr. Emile Voest, Chairman of InteRNA’s Scientific Advisory Board and Medical Director of the Netherlands Cancer Institute in Amsterdam. "The safety profile and significant anti-tumor efficacy demonstrated in preclinical studies provide a strong basis for first-in-human studies with INT-1B3."

About INT-1B3

INT-1B3’s unique mechanism of action addresses multiple hallmarks of cancer simultaneously. It directly targets tumor cells and the tumor microenvironment by specific modulation of multiple signaling pathway components across the PTEN tumor suppressor pathway and the oncogenic PI3K/Akt and Ras/MAPK pathways resulting in cell cycle arrest, induction of apoptosis and immunogenic tumor cell death (ICD); as well as downregulation of the adenosine-A2A receptor pathway and immunosuppressive FoxP3/Lag3 Tregs and monocytic myeloid-derived suppressor cells (mMDSCs). As a result, the immune system is activated, and long-term immunity is triggered by recruitment of CD8+ Teffs leading to decreased metastasis development and improved animal survival compared to anti-PD1 treatment. The created T cell-mediated immune response activity is also transferrable to naive mice via adoptive T cell transfer.