Affibody Announces Option Exercise by Daewoong

On April 30, 2020 Affibody AB ("Affibody"), a clinical stage biopharmaceutical company developing a pipeline of innovative drug projects, reported that its partner Daewoong, a South Korea-based pharmaceutical company, has exercised an option under the companies’ ongoing collaboration (Press release, Affibody, APR 30, 2020, View Source [SID1234575710]).

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"We are pleased to announce that our longtime partner Daewoong has decided to exercise their option to develop and commercialize an innovative new half-life extended biotherapeutics product" said David Bejker, CEO of Affibody. "We believe that the preclinical results generated by Daewoong support the innovative strengths of our technology".

"I am delighted to have established this collaboration with Affibody to develop long-acting basal insulin that harness the benefits of high affinity of fused insulin and albumin binding domain to increase duration of action and peak-less pharmacokinetic profile: " says Jongsang Ryu, Head of C&D Center at Daewoong. "We are very excited about the potential of this collaboration to develop long-acting diabetes treatments and about its considerable commercial value to Daewoong in the future."

Affibody will receive a license conversion fee and milestone payments as well as royalties. Further financial details were not disclosed.

Sunesis Pharmaceuticals to Host Conference Call on May 7th to Discuss First Quarter 2020 Financial Results and Recent Highlights

On April 30, 2020 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported that it will host a conference call on Thursday, May 7th, 2020 at 4:30 p.m. Eastern Time to discuss corporate updates and financial results for the first quarter ended March 31, 2020 (Press release, Sunesis, APR 30, 2020, http://ir.sunesis.com/news-releases/news-release-details/sunesis-pharmaceuticals-host-conference-call-may-7th-discuss [SID1234561677]).

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The call can be accessed by dialing (844) 296-7720 (U.S. and Canada) or (574) 990-1148 (International) and entering passcode 6168259.

To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at View Source The webcast will be recorded and available for replay on the company’s website for two weeks.

Reblozyl® (luspatercept) Receives Positive CHMP Opinion for the Treatment of Adults with Anemia in Beta Thalassemia and Myelodysplastic Syndromes

On April 30, 2020 Bristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of (Press release, Acceleron Pharma, APR 30, 2020, View Source [SID1234561666]):

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Adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy.

Adult patients with transfusion-dependent anemia associated with beta thalassemia.
This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients. The safety and efficacy results provided in the application are from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.
"Patients with myelodysplastic syndromes who experience anemia have limited treatment options, and some have been shown to not respond to available erythropoietin-based therapies," said Uwe Platzbecker, M.D., Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital and lead investigator of the MEDALIST study. "If approved, the introduction of a new class of therapy in Reblozyl could provide a promising option to help relieve patients from the burden of regular transfusions to manage their disease."
"Today’s positive CHMP opinion of Reblozyl is an important milestone for adult beta thalassemia patients in the EU who have limited treatment options to address anemia, a serious consequence of the disease," said Maria Domenica Cappellini, M.D., Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda and lead investigator of the BELIEVE study. "Reblozyl has the potential to significantly decrease the number of red blood cell transfusions patients need."
"This decision by the CHMP is an important step towards making this first-in-class therapy an option for eligible patients with anemia due to beta thalassemia or myelodysplastic syndromes," said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. "We, and our partners at Acceleron, look forward to the opportunity to make this treatment option available in the EU and are extremely appreciative of the patients, families and individuals who continue to help us progress important research in a range of serious diseases."

About MEDALIST
MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA naïve and unlikely to respond due to endogenous serum erythropoietin levels of ≥ 200 U/L, and had no prior treatment with disease modifying agents. Results of the MEDALIST trial were first presented during the Plenary Session of the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and were selected for the Best of ASH (Free ASH Whitepaper). The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS
MDS are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections. People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions and infections. There are approximately 50,000 patients with MDS in the EU5 countries.

About BELIEVE
BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing luspatercept plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia. Results of the BELIEVE trial were first presented at the 2018 ASH (Free ASH Whitepaper) Annual Meeting and selected for the Best of ASH (Free ASH Whitepaper). The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia
Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemia – a condition that can be debilitating and can lead to more severe complications for patients – as well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl
Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and

anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Important Safety Information

WARNINGS AND PRECAUTIONS
Thrombosis/Thromboembolism
In adult patients with beta thalassemia, thromboembolic events (TEE) were reported in 8/223 (3.6%) REBLOZYL-treated patients. TEEs included deep vein thrombosis, pulmonary embolus, portal vein thrombosis, and ischemic stroke. Patients with known risk factors for thromboembolism (splenectomy or concomitant use of hormone replacement therapy) may be at further increased risk of thromboembolic conditions. Consider thromboprophylaxis in patients at increased risk of TEE. Monitor patients for signs and symptoms of thromboembolic events and institute treatment promptly.

Hypertension
Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated patients. Across clinical studies, the incidence of Grade 3 to 4 hypertension ranged from 1.8% to 8.6%. In patients with beta thalassemia with normal baseline blood pressure, 13 (6.2%) patients developed systolic blood pressure (SBP) ≥130 mm Hg and 33 (16.6%) patients developed diastolic blood pressure (DBP) ≥80 mm Hg. In adult patients with MDS with normal baseline blood pressure, 26 (29.9%) patients developed SBP ≥130 mm Hg and 23 (16.4%) patients developed DBP ≥80 mm Hg. Monitor blood pressure prior to each administration. Manage new or exacerbations of preexisting hypertension using anti-hypertensive agents.

Embryo-Fetal Toxicity
REBLOZYL may cause fetal harm when administered to a pregnant woman. REBLOZYL caused increased post-implantation loss, decreased litter size, and an increased incidence of skeletal variations in pregnant rat and rabbit studies. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 3 months after the final dose.

ADVERSE REACTIONS
Beta Thalassemia

Serious adverse reactions occurred in 3.6% of patients on REBLOZYL. Serious adverse reactions occurring in 1% of patients included cerebrovascular accident and deep vein thrombosis. A fatal adverse reaction occurred in 1 patient treated with REBLOZYL who died due to an unconfirmed case of acute myeloid leukemia (AML)

Most common adverse reactions (at least 10% for REBLOZYL and 1% more than placebo) were headache (26% vs 24%), bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and dizziness (11% vs 5%)

Myelodysplastic Syndromes

Grade >3 (≥2%) adverse reactions included fatigue, hypertension, syncope and musculoskeletal pain. A fatal adverse reaction occurred in 5 (2.1%) patients

The most common (≥10%) adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis, and urinary tract infection
LACTATION
It is not known whether REBLOZYL is excreted into human milk or absorbed systemically after ingestion by a nursing infant. REBLOZYL was detected in milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because many drugs are excreted in human milk, and because of the unknown effects of REBLOZYL in infants, a decision should be made whether to discontinue nursing or to discontinue treatment. Because of the potential for serious adverse reactions in the breastfed child, breastfeeding is not recommended during treatment and for 3 months after the last dose.

Please see full Prescribing Information for REBLOZYL

Bristol Myers Squibb: Advancing Cancer Research
At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients’ quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.
Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

Intellia Therapeutics Names David Lebwohl, M.D., Chief Medical Officer

On April 30, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA) reported that it has named David Lebwohl, M.D., as its new executive vice president and chief medical officer (Press release, Intellia Therapeutics, APR 30, 2020, View Source [SID1234556934]). Dr. Lebwohl brings decades of biopharmaceutial leadership and drug development experience, and joins Intellia to lead its clinical development and regulatory organizations.

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"Dr. Lebwohl joins Intellia during an exciting time for the company, as we continue to rapidly progess our first systemic CRISPR/Cas9-based therapy to the clinic. David’s vast experience in rare diseases, engineered cell therapy and clinical development complement our R&D capabilities and leadership team strength," said Intellia President and Chief Executive Officer John Leonard, M.D. "He has been at the helm of premier development organizations that launched breakthrough oncology therapies to patients, most notably the recent CAR-T therapy, Kymriah, and the multi-indication blockbuster, Afinitor. Under his leadership, we look forward to the impact he can make in delivering Intellia’s CRISPR/Cas9-based treatments to patients."

Dr. Lebwohl’s Professional and Academic Credentials

Dr. Lebwohl’s career spans three decades in the biopharmaceutical industry, successfully bringing novel medicines through all phases of clinical trials and global regulatory approvals. During his career, he has overseen multiple full-scale development programs with more than 200 clinical studies across myriad indications. He joins Intellia from Semma Therapeutics, Inc., where he was chief medical officer and led the company’s regenerative medicine efforts using stem-cell-derived pancreatic islets to cure type I diabetes starting in November 2018. Semma was acquired by Vertex Pharmaceuticals Inc., in October 2019. Prior to his role at Semma, Dr. Lebwohl held numerous senior-level drug development leadership positions at the global healthcare company, Novartis Pharmaceuticals Inc. (Novartis), where most recently he was senior vice president and franchise global program head, CAR-T, Promacta and SEG101 Global Program Teams, responsible for the development of the breakthrough therapy Kymriah (tisagenlecleucel), approved for the treatment of B-cell acute lymphoblastic leukemia. Dr. Lebwohl also was responsible for numerous other Novartis oncology drug development programs, and led the company’s Cell and Gene Therapies Unit. Under his leadership, the blockbuster drug, Afinitor (everolimus), was approved for five indications including metastatic breast, kidney, brain and lung cancers. Prior to working at Novartis, Dr. Lebwohl spent five years at Bristol Myers Squibb, Inc., where he worked in the Oncology Clinical Development group at the company’s Pharmaceutical Research Institute. He is a well-recognized medical oncologist, with certifications in hematology and internal medicine.

Dr. Lebwohl received an undergraduate degree in Biochemical Sciences from Harvard College, and an M.D. from the Yale University School of Medicine. He completed his fellowship training at Memorial Sloan Kettering Cancer Center, and his residency in Internal Medicine at Brigham and Women’s Hospital in Boston. He has authored more than 50 peer-reviewed publications.

"I am thrilled to join Intellia’s leadership team as we propel the first systemic CRISPR/Cas9 genome editing treatment to the clinic this year," said Dr. Lebwohl. "I am passionate about working on the next generation of treatments, like genome editing therapies, that are both game-changing for medical practice and can address the unmet needs of patients. I look forward to leading the Development organization and building on the company’s clinical capabilities."

Savara to Report First Quarter 2020 Financial Results and Provide Business Update

On April 30, 2020 Savara Inc. (NASDAQ: SVRA), an orphan lung disease company, reported that the Company will report first quarter 2020 financial results and provide a business update on Thursday, May 7, 2020 (Press release, Savara, APR 30, 2020, View Source [SID1234556917]). Savara management will host a conference call/webcast at 4:30 p.m. Eastern Time (ET)/1:30 p.m. Pacific Time (PT).

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Shareholders and other interested parties may access the call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and requesting the "Savara Inc." call. A live webcast of the call can be accessed on the Investors page of Savara’s website at View Source

Approximately one hour after the call, a telephone replay will be available and will remain available through May 14, 2020 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10143130. A webcast replay will be available on the Investors page of Savara’s website and will remain available for 30 days.