On February 12, 2020 Anika Therapeutics, Inc. (NASDAQ: ANIK) reported that Dr. Cheryl Blanchard, a member of the Company’s Board of Directors since August 2018, has been named interim Chief Executive Officer, effective immediately, while the Board continues its search to identify a new CEO following the recent passing of Joseph Darling (Press release, Anika Therapeutics, FEB 12, 2020, View Source [SID1234554345]). In connection with Dr. Blanchard’s appointment, the previously announced interim Office of the President has been dissolved.

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"We are pleased that Dr. Blanchard, a seasoned executive who has previously served as a biotech President and CEO, has agreed to step in as our interim CEO as we search for Anika’s next leader," said Dr. Joseph Bower, Chairman of Anika’s Board of Directors. "With Dr. Blanchard’s experience, as well as her service on our Board since August 2018, we expect this to be a smooth transition. Given her experience in leading orthopaedic device, joint preservation and restoration businesses, we are fortunate to be able to call on her to help build on Anika’s strong foundation and continue its evolution into a leader in joint preservation and restoration. We are confident in Anika’s prospects and the strong and deep leadership team we have in place as we continue to enhance value for our shareholders, customers and other stakeholders."

"We are all saddened by Joe’s sudden passing, but we also share a commitment to carrying out the initiatives crafted by him and the rest of the executive team and Board. I step into this interim role confident in the team’s ability to continue the successful execution of Anika’s five-year strategic plan," said Dr. Blanchard. "Since joining the Board, I have developed a deep appreciation for the Company’s proprietary offerings and exceptional talent. Anika is well-positioned in its markets and has significant growth prospects. I look forward to leveraging my past experiences and collaborating with the team to maintain the high level of operating discipline while solidifying an expanding position and share in the $7 billion sports and regenerative medicine market."

The Company plans to issue its fourth-quarter and full-year 2019 financial results after the close of the market on February 20, 2020 and will hold its investor conference call on the same day, February 20, 2020, at 5:00 p.m. ET to discuss its financial results, business highlights, and outlook.

About Cheryl R. Blanchard, Ph.D.
Dr. Blanchard joined the Board of Directors of Anika Therapeutics in August 2018. She served as President and Chief Executive Officer of Microchips Biotech, Inc., a venture-backed biotechnology company developing regenerative medicine and drug delivery products, from 2014 until its sale to Daré Bioscience, Inc. in November 2019. From 2000 to 2012, she served in various officer positions of Zimmer, Inc. (now Zimmer Biomet), a medical device company focused on musculoskeletal products, including as the Senior Vice President, Corporate Chief Scientific Officer and General Manager of its Biologics Business. She was also a member of Zimmer’s executive committee and founded, built and led Zimmer’s Joint Preservation/Regenerative Medicine business. Prior to joining Zimmer, Dr. Blanchard built and led the medical device practice at Southwest Research Institute. Dr. Blanchard received her M.S. and Ph.D. in Materials Science and Engineering at the University of Texas at Austin and received her B.S. in Ceramic Engineering at Alfred University. Dr. Blanchard is a member of the National Academy of Engineering.

Until her appointment as Interim Chief Executive Officer, Dr. Blanchard was serving as a member of Anika’s Compensation Committee and Governance and Nominating Committee. She has served as a director of Neuronetics (NASDAQ: STIM) since February 2019 and a director of Daré Bioscience, Inc. (NASDAQ: DARE) since November 2019. Dr. Blanchard also serves on the Board of a privately held company in the life sciences industry.

On February 12, 2020 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, CRISPR Therapeutics, FEB 12, 2020, View Source [SID1234554299]).

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"In 2019, CRISPR Therapeutics achieved important milestones and momentum across key programs. We announced positive interim safety and efficacy data from the first two patients in our ongoing CTX001 clinical trials, one patient with beta thalassemia and one patient with sickle cell disease. These preliminary data support our belief in the potential of CTX001 to have meaningful benefit for patients following a one-time intervention," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "In addition, we advanced our first allogeneic CAR-T cell therapy, CTX110, targeting CD19+ malignancies and, building on this progress, reported that we have begun enrolling patients in a clinical trial for our second allogeneic CAR-T therapy, CTX120, targeting BCMA for the treatment of relapsed or refractory multiple myeloma."

Dr. Kulkarni added: "2020 has the potential to be a pivotal year in our company’s growth. We expect to conduct clinical trials in five indications, and we anticipate new data from our immuno-oncology and hemoglobinopathies programs. Our continued progress brings us closer to potentially providing transformative therapies to patients with serious diseases."

2019 Highlights and Outlook

•Beta Thalassemia and Sickle Cell Disease

•In November 2019, CRISPR Therapeutics and its partner Vertex announced positive, interim data from the first two patients with severe hemoglobinopathies – one patient with transfusion-dependent beta thalassemia (TDT) and one patient with severe sickle cell disease (SCD) – treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in the ongoing Phase 1/2 CLIMB clinical trials. Enrollment is ongoing in both trials and the companies expect to provide additional data for these programs in 2020.

•CTX001 has been granted orphan drug designation (ODD) by the European Commission for the treatment of SCD. The European Commission previously granted CTX001 ODD for the treatment of TDT.

•Immuno-Oncology

•Patient enrollment continues in a clinical trial to assess the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting refractory CD19+ B-cell malignancies. The multi-center, open label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. If successful, CTX110 could enable off-the-shelf use of cell therapies and greatly expand their applicability and accessibility in immuno-oncology.

•CRISPR Therapeutics has begun enrolling patients in a clinical trial to assess the safety and efficacy of CTX120, its wholly-owned allogeneic CAR-T cell therapy targeting BCMA for the treatment of relapsed or refractory multiple myeloma. The multi-center, open label trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

•CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates toward clinical development, including CTX130, its wholly-owned allogeneic CAR-T cell therapy targeting CD70 for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. The Company continues to scale its capabilities to enable rapid advancement of these programs into and through the clinic.

•Other Corporate Matters

•In November 2019, CRISPR Therapeutics announced the pricing of an underwritten public offering of 4,250,000 common shares at a public offering price of $64.50 per share, plus the exercise in full of the underwriters’ option to purchase 637,500 additional common shares. Gross proceeds from the offering (including the exercise of the underwriters’ option), before deducting underwriting discounts and commissions and other offering expenses, were $315.2 million. The initial offering closed in November 2019 and the closing of the option to purchase additional shares occurred in December 2019.

•In 2019, CRISPR Therapeutics broadened its business development efforts through additional transactions, including an agreement with Bayer to place Casebia Therapeutics, previously a joint venture between Bayer and CRISPR Therapeutics, under CRISPR Therapeutics’ direct management; a license agreement with KSQ Therapeutics whereby CRISPR Therapeutics gains access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ gains access to CRISPR Therapeutics’ IP for editing novel gene targets for certain applications; a collaboration and license agreement with ProBioGen focused on the development of novel in vivo delivery modalities for CRISPR/Cas9 leveraging ProBioGen’s existing technology and expertise; and a collaboration with StrideBio, which expands upon an existing agreement to generate engineered adeno-associated viruses (AAV) capsids with improved properties for in vivo gene editing programs and includes additional undisclosed applications.

•In June 2019, CRISPR Therapeutics and Vertex expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1). In connection with this agreement, CRISPR Therapeutics received a $175.0 million up-front payment from Vertex, and is eligible to receive milestone payments from Vertex of up to $825.0 million in the aggregate. CRISPR Therapeutics continues to make advancements with programs utilizing an in vivo approach, which remains a key area of focus.

•In June 2019, CRISPR Therapeutics received notification that the United States Patent and Trademark Office (USPTO) has initiated an interference proceeding at the Patent Trial and Appeal Board between certain pending U.S. patent applications co-owned by the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively, the "CVC Group") and certain patents and a patent application currently owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology, all of which are related to the single guide format of CRISPR/Cas9 genome editing technology in eukaryotic cells. As of December 2019, the USPTO has granted twenty-one patents to the CVC group. None of these issued patents are involved in the interference.

•Fourth Quarter 2019 Financial Results

•Cash Position: Cash and cash equivalents as of December 31, 2019, were $943.8 million, compared to $456.6 million as of December 31, 2018, an increase of $487.2 million. The increase in cash was primarily driven by cash from financing activities of $430.9 million from our at-the market offering, completed in July 2019; from our November public offering; and from stock options exercised during 2019. In addition, the Company generated cash from operations in the current year of $56.7 million, driven by cash received from Vertex for milestone and option payments of $208.0 million as well as cash obtained from the Bayer transaction of $31.8 million, offset by operating expenses.

•Revenue: Total collaboration revenue was $77.0 million for the fourth quarter of 2019 compared to $0.1 million for fourth quarter of 2018, and $289.6 million for the year ended December 31, 2019, compared to $3.1 million for the year ended December 31, 2018. The increase in revenue was primarily attributable to revenue recognized in connection with the Company’s collaboration agreements with Vertex.

•R&D Expenses: R&D expenses were $48.8 million for the fourth quarter of 2019 compared to $28.8 million for the fourth quarter of 2018, and $179.4 million for the year ended December 31, 2019 compared to $113.8 million for the year ended December 31, 2018. The increase in expense for the year was driven by increased headcount and development activities supporting the advancement of the hemoglobinopathies program, the broadening of the Company’s wholly-owned immuno-oncology portfolio and $10.0 million of non-cash expense related to the Company’s collaboration with Vertex.

•G&A Expenses: General and administrative expenses were $17.3 million for the fourth quarter of 2019 compared to $16.5 million for the fourth quarter of 2018, and $63.5 million for the year ended December 31, 2019, compared to $48.3 million for the year ended December 31, 2018. The increase in general and administrative expenses for the year was driven by headcount-related expense and external professional and consulting service expense.

•Net Income/Loss: Net income was $30.5 million for the fourth quarter of 2019 compared to net loss of $47.6 million for the fourth quarter of 2018, and net income was $66.9 million for the year ended December 31, 2019, compared to a loss of $165.0 million for the year ended December 31, 2018.
About CTX001TM
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About CTX110TM
CTX110 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19, or CD19, for the treatment of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX110 is in a clinical trial designed to assess the safety and efficacy of CTX110 in relapsed or refractory B-cell malignancies. The multi-center, open-label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110.

About CTX120TM
CTX120 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting B-cell maturation antigen, or BCMA. A wholly-owned asset of CRISPR Therapeutics, CTX120 is in a clinical trial designed to assess the safety and efficacy of CTX120 in relapsed or refractory multiple myeloma. The multi-center, open-label clinical trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

About CTX130TM
CTX130 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting CD70, an antigen expressed on hematologic cancers. A wholly-owned asset of CRISPR Therapeutics, CTX130 is in development for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies.

On February 12, 2020 Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, reported that it will report financial results and provide an update on recent progress on its development programs post-market on February 19, 2020 (Press release, Reata Pharmaceuticals, FEB 12, 2020, View Source [SID1234554298]).

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Reata’s management will host a conference call on February 19, 2020, at 4:30 p.m. ET. The conference call will be accessible by dialing (844) 348-3946 (toll-free domestic) or (213) 358-0892 (international) using the access code: 1434037. The webcast link is View Source

Fourth-quarter and full-year 2019 financial results to be discussed during the call will be included in an earnings press release that will be available on the company’s website shortly before the call at View Source and will be available for 12 months after the call. The audio recording and webcast will be accessible for at least 90 days after the event at View Source.

On February 12, 2020 Sirtex Medical, a leading manufacturer of targeted liver cancer therapies, reported a collaboration with MIM Software Inc., a leading global provider of practical imaging solutions in the fields of radiation oncology, radiology, nuclear medicine, neuroimaging and cardiac imaging (Press release, Sirtex Medical, FEB 12, 2020, View Source [SID1234554247]). Under the terms of the agreement, Sirtex’s specialized sales teams in the United States, Europe and other key global markets will offer MIM SurePlanTM LiverY90 software alongside the company’s SIR-Spheres Y-90 resin microspheres to improve treatment precision for patients with hepatic tumors.

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MIM SurePlan LiverY90 provides timesaving tools and post-treatment dosimetry for interventional radiologists and nuclear medicine physicians treating hepatic tumors with SIR-Spheres. The software allows physicians to quickly calculate the dose delivered by the microspheres to improve treatment decisions and patient care. The technology also incorporates CT, PET, SPECT and MRI scans simultaneously to deliver results on spheres deposition, coverage and absorbed dose, allowing physicians to make informed decisions faster about next steps.

In addition to sharing a mission to provide physicians the tools they need to treat cancers, Sirtex and MIM also share a commitment to provide patients the best care throughout the treatment journey.

"Given the overlap of their technology and our SIR-Spheres product, Sirtex and MIM have a long history of working alongside clinical care teams," said Kevin Smith, Chief Executive Officer of Sirtex. "Now as partners, we will be able to combine our expertise and resources to provide an even higher level of support to treat liver tumors. We are excited to see how this collaboration will benefit physicians and ultimately improve treatment outcomes for patients."

"We are proud and excited to partner with Sirtex, mainly because it will provide access to personalized dosimetry for a large number of patients across the globe," said Andrew Nelson, Chief Executive Officer of MIM Software Inc. "The treatment of liver tumors requires a multi-disciplinary approach, so it’s a natural evolution for companies like ours to partner to provide an even faster, higher level of support and resources to treatment teams."

In the United States, SIR-Spheres Y-90 resin microspheres received Premarket Approval (PMA) from the FDA and are indicated for the treatment of non-resectable metastatic liver tumors from primary colorectal cancer in combination with intrahepatic artery chemotherapy using floxuridine. SIR-Spheres Y-90 resin microspheres are approved for the treatment of inoperable liver tumors in Australia, the European Union, Argentina, Brazil, Canada and several countries in Asia, such as India and Singapore.

About Selective Internal Radiation Therapy (SIRT) using SIR-Spheres Y-90 resin microspheres
SIR-Spheres Y-90 resin microspheres is a prescription device for the treatment of inoperable liver tumors. SIRT is a minimally invasive treatment that delivers high doses of high-energy beta radiation directly to the tumors. SIRT is administered to patients by interventional radiologists and nuclear medicine physicians, who infuse millions of radioactive resin microspheres via a catheter into the liver arteries that supply blood to the tumors. By using the tumors’ blood supply, the microspheres selectively target liver tumors with a dose of radiation that is up to 40 times higher than conventional radiotherapy, while sparing healthy tissue.

On February 12, 2020 miR Scientific LLC, reported that it will present the results of the recently completed Case-Control validation study of 1,436 patients consisting of 836 patients in a fully cross-validated training group and independent testing group of 600 patients (Press release, miR Scientific, FEB 12, 2020, View Source [SID1234554246]). The podium presentation will be in Oral Abstract Session A: Prostate Cancer at 1:10 p.m. on Thursday, February 13, 2020 and is titled Abstract 277: Analysis of small non-coding RNAs in urinary exosomes to classify prostate cancer into low-grade (GG1) and higher-grade (GG2-5).

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The study describes the miR Scientific Sentinel Disease Management Platform for Prostate Cancer which currently consists of three tests: The Sentinel PCa Test, that determines the presence or absence of prostate cancer, the Sentinel CS Test which identifies patients with low grade (GG1) or intermediate and high grade (GG2-GG5) prostate cancer and the Sentinel HG Test that identifies patients with high grade, high-risk cancer. Each test examines the expression levels of small non-coding RNAs (sncRNAs) extracted from urinary exosomes to diagnose, classify and monitor prostate cancer. The company’s classification algorithms identify and interrogate between 200-280 small non-coding RNAs using a customized, high throughput qPCR OpenArray platform for each test.

The results of the validation study demonstrate that the Sentinel PCa Test has a sensitivity of 94% and a specificity of 92%; the Sentinel CS Test has a sensitivity of 93% and a specificity of 90%; and the Sentinel HG Test has a sensitivity of 94% and a specificity of 96%. The performance characteristics of these tests support the company’s efforts to introduce the miR Scientific Disease Management Platform to mitigate poor outcomes and waste for patients and all key stakeholders, including healthcare providers, employers and payors. This validation now enables stakeholders to engage as early adopters worldwide. The combination of the three tests, which are standalone, do not require nor rely on any other testing nor risk analysis and can be performed on a single urine sample, providing a very precise platform for disease management, with high utility for diagnosing, classifying and monitoring prostate cancer.

"The results presented at ASCO (Free ASCO Whitepaper) GU demonstrate that the miR Scientific Sentinel Tests can transform clinical practice with broadly new and powerful capabilities to directly classify patients into actionable pathways: those with no evidence of prostate cancer, patients in need of definitive treatment, and patients eligible for active surveillance, which the Sentinel Tests can monitor," said Sam Salman, Chairman & CEO of miR Scientific. "The accuracy demonstrated by the miR Scientific Sentinel tests is significantly better than that of other current technologies and could prove to be even more accurate as we are currently ascertaining what proportion of this discordance represents misattribution of core needle biopsy histopathology or genuine misclassification errors of the Sentinel Tests. miR Scientific believes that the results suggest that the non-invasive Sentinel tests can be used as part of the Sentinel Prostate Cancer Disease Management Platform to provide patients and health care providers with an unprecedented level of information, allowing for more accurate and effective treatment of these cancers."

Renowned practitioners have expressed their confidence in these results. "The burden of over diagnosis and unnecessary treatments in prostate cancer worldwide is well known. What has been lacking is an effective, non-invasive tool to identify which patients may harbor aggressive cancers that are life threatening. The miR Scientific platform of liquid biopsy represents a highly sensitive and accurate way to identify these patients with less need for invasive procedures," said James M. McKiernan, M.D., the John K. Lattimer Professor and Chairman of the Department of Urology at Columbia University College of Physicians and Surgeons, Urologist in Chief, New York Presbyterian Hospital.