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Rubius Therapeutics Announces Appointment of Top Leadership

On January 4, 2017 Rubius Therapeutics, Inc., a Flagship Pioneering company, has reported the hiring of two key company leaders, David Epstein and Torben Straight Nissen, to accelerate development of new medicines from the company’s potentially revolutionary new product platform: Red-Cell Therapeutics (RCTs). Specifically, Epstein is joining as Chairman of the Board and Straight Nissen as President of the company. Flagship Pioneering developed Rubius in its innovation foundry, Flagship VentureLabs, throughout 2014 and launched the company in 2015 with an initial capital commitment of $25 million.

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Through the use of patented, advanced engineering techniques and know-how the company has successfully produced red blood cells that express therapeutic proteins that enable their use for the treatment of serious diseases. Rubius is now demonstrating that these newly equipped high performing, "off the shelf" Red-Cell Therapeutics have pre-clinical activity across a spectrum of medical applications. For example, in cancer a red blood cell can be transformed to express multiple synergistic proteins which can both inhibit cancer cell growth as well as activate the patient’s immune system. Alternatively, red blood cells have been engineered to replace missing enzymes for patients living with a variety of rare diseases, or even to remove unwanted metabolic products from the blood stream. Rubius has generated more than 200 prototypes to date. RCTs have demonstrated potential advantages compared to other cellular approaches to treating disease, such as greater durability, easier manufacturing scale-up and safety advantages due to the unique features of red blood cells.

"David and Torben bring a unique combination of scientific and business leadership experience which will accelerate Rubius’ development. As founders and primary investors, we are delighted to have the duo take the helm of this transformative company from the leadership provided by the Flagship VentureLabs team," stated Dr. Noubar Afeyan, Chairman of the Board of Rubius and CEO of Flagship Pioneering.

David Epstein, former CEO of Novartis Pharmaceuticals, has joined to serve as a Chairman of the Board. He will also will be serving in the newly created role of Executive Partner for Flagship Pioneering. At Novartis, Epstein was responsible for leading the development and commercialization of over 30 new medicines including breakthroughs such as Glivec, Gilenya, Cosentyx and Entresto.

As President of Rubius, Torben Straight Nissen, Ph.D, brings 20 years of deep scientific knowledge and business experience to expand the company’s platform and clinical programs. Torben joins Rubius from Pfizer, where he held multiple scientific and business leadership roles to advance the company’s therapeutic innovation, research and development.

"Flagship is breaking boundaries and exploring new frontiers that, I believe, could genuinely result in breakthroughs that directly improve survival and quality of life. I am tremendously excited to be part of the Rubius board and Flagship team," said David Epstein.

"Genetically-modified red blood cells hold incredible potential to provide superior treatment for many diseases, from rare to common conditions. Rubius is working on an exciting platform that could bring forward multiple therapies and modalities that improve and save lives," said Dr. Straight Nissen. "I am looking forward to growing the company and helping Rubius harness its platform to the fullest."

Commenting on the hires, Rubius Founding CEO and Flagship Partner Avak Kahvejian said, "The past two years of development at Rubius have shown the vast scope and applicability of Red-Cell Therapeutics. I look forward to continuing with this exciting venture in a new capacity as Chief Innovation Officer as Torben and David lead the transformation of Rubius into a development stage company in the near future."

About Rubius Therapeutics
Rubius Therapeutics is developing Red-Cell Therapeutics TM (RCTs) as a new treatment modality to address a wide array of indications including cancer, autoimmune disease, rare disease and metabolic disease. RCTs are enucleated cell-based therapies that can be engineered to have diverse activities. The company has established a platform for the rapid design, generation, testing and development of RCT products. The company was founded and launched by Flagship VentureLabs, the innovation foundry of Flagship Pioneering.

Abbott Completes the Acquisition of St. Jude Medical

On January 4, 2017 Abbott (NYSE: ABT) reported it has completed the acquisition of St. Jude Medical, Inc., establishing the company as a leader in the medical device arena (Press release, Abbott, JAN 4, 2017, View Source [SID1234517289]). The transaction provides Abbott with expanded opportunities for future growth and is an important part of the company’s ongoing effort to develop a strong, diverse portfolio of devices, diagnostics, nutritionals and branded generic pharmaceuticals.

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"Abbott has a strong track record of successfully integrating dozens of businesses on a global scale and accelerating growth," said Miles D. White, chairman and chief executive officer, Abbott. "The addition of St. Jude Medical strengthens our global medical device leadership while offering innovative products to address more areas of care, in more physicians’ offices and hospitals around the world."

Pursuant to the terms of the Merger Agreement, upon completion of the acquisition, St. Jude Medical became a wholly-owned subsidiary of Abbott. As a result of the completion of the acquisition, Jan. 4, 2017, was the last day of trading of St. Jude Medical shares on the New York Stock Exchange.

Strategic Fit
St. Jude Medical’s strong positions in fast-growing areas such as atrial fibrillation, heart failure, structural heart and chronic pain complement Abbott’s leading positions in coronary interventions and mitral valve disease. Together, the company will compete in nearly every area of the $30 billion cardiovascular market and hold the No. 1 or 2 positions across large and high-growth cardiovascular device markets. This leading combined portfolio will have the depth, breadth, scale and innovation to help patients restore their health, improve outcomes and deliver greater value to customers and payors. Furthermore, the acquisition balances and strengthens the Abbott portfolio, which includes leading positions across all of its four core businesses.

Breakthrough Invention
Abbott will have a powerful pipeline across cardiovascular and neuromodulation patient care ready to deliver next-generation medical technologies and offer improved efficiencies for health care systems around the world. In fact, Abbott will continue to bring numerous new products to key markets during the coming years, including:

EnSite Precision (which received U.S. FDA approval last month) next-generation cardiac mapping system to visualize and navigate catheters in the heart during ablation procedures

ConfirmRx Implantable Cardiac Monitor to help physicians remotely diagnose and treat the most difficult to detect cardiac arrhythmias

HeartMate 3, which offers physicians more options for patients with advanced stage heart failure
Portico Transcatheter Aortic Heart Valves for patients with severe aortic stenosis – the narrowing of the aortic valve that obstructs blood flow from the heart

Proclaim DRG system and other stimulation waveform technologies to provide more options for patients with chronic pain
Absorb, the world’s first bioresorbable coronary stent and MitraClip, the world’s first transcatheter mitral-valve repair device in additional countries

"We continue to deliberately shape our business for long-term success by securing leadership positions in attractive markets and focusing on customer needs," said Mr. White. "This philosophy has served as the foundation for significant and sustainable value creation for our shareholders. The addition of St. Jude Medical creates one of the broadest medical device portfolios in the world and provides a steady stream of new technologies and therapies for many years to come."

Tarveda Therapeutics Provides Year-End Update and Outlines Milestones for 2017

On January 4, 2017 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company discovering and developing Pentarins as a new class of potent and selective anti-cancer medicines, reported highlights from 2016 and outlined expected milestones for 2017 (Press release, Tarveda Therapeutics, JAN 4, 2017, View Source [SID1234517285]).

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"2016 was a year of transformative progress for Tarveda’s Pentarin platform and acceleration in the development of our novel treatments to improve the effectiveness of cancer therapies for patients with solid tumors. The company raised substantial capital and strengthened its premier venture capital syndicate, completed the in-licensing of PEN-866, which will be in a Phase I clinical trial in 2017, and embarked on our first-in-human trial for our lead drug candidate PEN-221," said Drew Fromkin, President and Chief Executive Officer of Tarveda. "We have constructed an attractive pipeline of novel, anti-cancer medicines, built a solid foundation on which to create true value for patients and our Company’s stakeholders, and look forward to achieving additional, validating milestones in 2017."

Key 2016 Accomplishments

Secured $38 million in Series C financing co-led by Novo A/S and New Enterprise Associates (NEA) with Flagship Pioneering, NanoDimension and Eminent Venture Capital also participating

Concentrated the Company’s development and discovery resources on Tarveda’s Pentarin Platform through the spin-off of BTP-114 to Placon Therapeutics. BTP-114 is a next-generation, platinum-based chemotherapy that is now in a Phase I clinical trial in collaboration with a domestic pharmaceutical partner

Expanded clinical program and R&D expertise with the appointment of Leila Alland, M.D. to Chief Medical Officer. Dr. Alland brings more than 15 years of pharmaceutical drug development experience including her most recent leadership roles in oncology clinical development at AstraZeneca and Bristol-Myers Squibb

In-licensed the broad Heat Shock Protein 90 (HSP90) targeting conjugate platform, including lead drug PEN-866, from Madrigal Pharmaceuticals

Miniaturized HSP90 targeting drug conjugates are designed to have high affinity for the well-characterized intracellular target, HSP90, and increase the killing of cancer cells while reducing collateral damage to normal cells

PEN-866 accumulates and is retained in xenograft tumors, releasing over time the potent tumor-killing payload SN-38, the active component of the approved anticancer medicine, irinotecan

PEN-866 has shown efficacy and durability of response in multiple preclinical tumor models including small cell lung, pancreatic, and ovarian cancers, sarcoma and patient derived tumor models

Presented preclinical data for PEN-221 demonstrating complete and sustained tumor regressions in hard-to-treat, somatostatin receptor 2 (SSTR2) positive cancer models

Miniaturized size and unique design of Pentarins such as PEN-221 allow for rapid penetration deep into solid tumors to drive efficacy
PEN-221 binds with high affinity to the cell surface target, SSTR2, internalizes into the tumor cell and releases its DM1 payload, which is retained in the tumor cell long after PEN-221 is cleared from circulation

Initiated a first-in-human Phase 1/2a trial of PEN-221 at multiple, premier clinical sites in the United States

The Phase 1/2a study of PEN-221 selects patients most likely to respond to PEN-221 by using FDA approved imaging diagnostics to identify those with tumors expressing SSTR2

Expected Key 2017 Milestones
Complete PEN-221 Phase 1 dose escalation and identify the recommended Phase 2 dose of PEN-221 in patients with SSTR2 expressing neuroendocrine tumors and small cell lung cancer in 2017

Initiate the first-in-human trial of PEN-866 in 2017, a Phase 1/2a dose escalation/expansion study evaluating safety and efficacy of PEN-866 in advanced, topoisomerase-1 sensitive cancer patients

Report preclinical data in 2017 on new Pentarins that bind to intracellular and cell-surface targets and drive anti-tumor activity
Announce the Tarveda Scientific Advisory Board in the first quarter of 2017

About Pentarins
Tarveda is developing Pentarins, potent and selective miniaturized drug conjugates with high affinity for specific cell surface and intracellular targets. Pentarins are engineered to bind to their tumor cell targets and provide sustained release of their potent therapeutic payloads deep into solid tumor tissue. Comprised of a targeting ligand conjugated to a potent cell killing agent through a chemical linker, Pentarins are designed to overcome the deficits of both larger antibody drug conjugates and small molecules that limit their therapeutic effectiveness against solid tumors. Together, the components of Tarveda’s Pentarins have distinct, yet synergistic, anticancer attributes: the small size of Pentarins allows for effective penetration and distribution into the tumor tissue, the ligand’s targeting ability allows for specific binding and retention in tumor cells, and the chemical linker is tuned to optimize the release of the potent, cell killing payload inside the cancer cells for efficacy.

The USF Tampa Bay Technology Incubator Welcomes Venn Therapeutics

On January 4, 2017  Venn Therapeutics, a biotechnology company focused on developing immuno-oncology therapies, has recently joined the University of South Florida’s Tampa Bay Technology Incubator (TBTI) (Press release, Venn Therapeutics, JAN 4, 2017, View Source [SID1234519736]). The company’s focus is on immune system priming and advances therapies that trigger the body’s innate immune response to target cancer.

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Current immuno-oncology therapies help between 10-30 percent of the patient population. Venn is designed to meet the needs of the large group of patients who are unresponsive to current therapies. Their novel therapeutics will be used both alone and in combination with other therapies that target the adaptive immune system.

Most current cancer immunotherapies focus primarily on the adaptive system. Venn Therapeutics, which licensed their first asset in February 2016, believes that triggering the innate immune system in combination with harnessing adaptive immunity is essential to produce durable, long-term survival in patients.

"Our focus is developing cancer therapies designed to power the immune system to kill cancer cells," said Sam Shrivastava, Venn Therapeutics’ chairman and chief executive officer. "By turning the immune system ‘on’ and harnessing the natural ability of the body to fight aberrant cells, we are hoping to provide better patient outcomes." Venn Therapeutics is currently developing a pipeline of novel, first-in-class agents that overcome immunosuppression by modulating the innate immune system. The company’s pipeline includes a novel therapeutic for p53 engineered personalized cancer vaccine, which is in Phase II clinical trials, as well as a STING based platform technologies and a novel Oncolytic virus therapy.

"We have multiple shots to develop a successful cancer therapy and we can do that at a major cost advantage to competitors. We intend to also strike deals with major pharmaceutical companies in the very near future to advance our lead asset into clinic," Shrivastava said. "We are excited to see our drug pipeline mature." The TBTI supports technology research as a catalyst for economic development and advocates the creation and development of facilities for high-technology companies and related support functions. As an Incubator company, Venn Therapeutics will have access to dedicated wet lab and office space as well as essential, costly research equipment.

"We are very pleased to be part of the Tampa Bay Technology Incubator," Shrivastava said. "Being able to utilize the TBTI laboratories and facilities is helping our company perform essential R&D functions and is providing fundamental support as we move towards our goal of expanding the population of patients who received clinical benefit from immunotherapy."

Venn Therapeutics was recently awarded funding from the University of South Florida Research Foundation’s Seed Capital Accelerator program.

"The Seed Accelerator program was created to help TBTI affiliated companies like Venn Therapeutics reach specific goals in a year or less, allowing startups to reach critical development milestones and advance to the next step of commercialization more rapidly," said Valerie McDevitt, USF associate vice president for technology transfer & business partnerships.

CTI BioPharma Announces Removal Of Full Clinical Hold On Pacritinib

On January 5, 2017 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) reported that the full clinical hold (February 2016) implemented by the U.S. Food and Drug Administration (FDA) on all clinical trials conducted under the Investigational New Drug (IND) application for pacritinib has now been removed (Press release, CTI BioPharma, JAN 5, 2017, View Source [SID1234517273]).

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The Company’s complete response submission included, among other items, final Clinical Study Reports for both PERSIST-1 and 2 trials and a dose-exploration clinical trial protocol that the FDA requested. The new trial, PAC203 plans to enroll up to approximately 105 patients with primary myelofibrosis who have failed prior ruxolitinib therapy to evaluate the safety and the dose response relationship for efficacy (spleen volume reduction at 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID. The 200 mg BID dose regimen was used in PERSIST-2. The Company expects to start the trial in the second quarter of 2017.

"We are pleased to resolve the full clinical hold through working diligently with the FDA to provide a comprehensive response to their requests," said Richard Love, Interim President and CEO of CTI BioPharma. "We look forward to discussing with the FDA the future development of pacritinib. We believe pacritinib can ultimately address the unmet need of patients with myelofibrosis who are ineligible to receive or are not benefitting from the approved JAK1/JAK2 inhibitor, ruxolitinib, as these patients have limited treatment options."

About the Phase 3 Development Program of Pacritinib

Pacritinib was evaluated in two Phase 3 clinical trials, known as the PERSIST program, for patients with myelofibrosis, with one trial in a broad set of patients without limitations on platelet counts, the PERSIST-1 trial; and the other in patients with low platelet counts, the PERSIST-2 trial. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy; or patients who are intolerant of, or whose symptoms are not well controlled (sub-optimally managed) on other JAK2 therapy.

Clinical studies under the investigational new drug (IND) for pacritinib were subject to a full clinical hold issued by the FDA in February 2016. The FDA noted interim overall survival results from the PERSIST-2 showing a detrimental effect on survival were consistent with the results from PERSIST-1 and that deaths in PERSIST-2 in pacritinib-treated patients include intracranial hemorrhage, cardiac failure and cardiac arrest.

PERSIST-1 was a randomized (2:1), controlled, open-label, multinational Phase 3 trial evaluating the efficacy and safety of pacritinib compared to BAT, excluding JAK2 inhibitors, which included a broad range of currently utilized treatments – in 327 patients with myelofibrosis, regardless of the patients’ platelet counts. The study included patients with severe or life-threatening thrombocytopenia. Patients were randomized to receive 400 mg pacritinib once daily or BAT, excluding JAK2 inhibitors. The trial met its primary endpoint of spleen volume reduction (35 percent or greater from baseline to Week 24 by MRI/CT scan).

PERSIST-2 was a randomized (1:1:1), controlled, open-label, multinational Phase 3 clinical trial evaluating pacritinib compared to best available therapy (BAT), including the approved JAK1/JAK2 inhibitor ruxolitinib, for patients with myelofibrosis whose platelet counts were less than or equal to 100,000 per microliter (≤100,000/μL). Patients were randomized to receive 200 mg pacritinib twice daily (BID), 400 mg pacritinib once daily (QD) or BAT. Results of the trial were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual meeting in Dececember 2016. The trial met one of its co-primary endpoints, that of spleen volume reduction (35 percent or greater from baseline to Week 24 by MRI/CT scan). The co-primary endpoint of reduction of Total Symptom Score (TSS) was not achieved but trended toward improvement in TSS. Irrespective of prior ruxolitinib treatment, pacritinib therapy resulted in a statistically significant higher proportion of patients with SVR than patients on BAT. Although secondary objectives could not be evaluated formally due to the study not achieving one of the primary objectives, when the two pacritinib dosing arms were evaluated separately versus BAT, pacritinib BID showed a higher percent of SVR and TSS responses compared to BAT; whereas, pacritinib given QD showed only a higher percent SVR responses compared to BAT. There was no significant difference in overall survival (OS) across treatment arms, censored at the time of clinical hold. The most common treatment-emergent adverse events (AEs), occurring in 20 percent or more of patients treated with pacritinib within 24 weeks, of any grade, were gastrointestinal (generally manageable diarrhea, nausea and vomiting) and hematologic (anemia and thrombocytopenia) and were generally less frequent for BID versus QD administration. The most common serious treatment-emergent AEs (incidence of ≥5 percent reported in any treatment arm irrespective of grade) were anemia, thrombocytopenia, pneumonia and acute renal failure none of which exceeded 8 percent individually in any arm.

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia, or AML, myelodysplastic syndrome, or MDS, chronic myelomonocytic leukemia, or CMML, and chronic lymphocytic leukemia, or CLL, due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

About Myelofibrosis and Myeloproliferative Neoplasms

Myelofibrosis is one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of progressive blood cancers. The three main types of MPNs are primary myelofibrosis (PMF), polycethemia vera (PV) and essential thrombocythemia (ET).1

Myelofibrosis is a serious and life-threatening bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue and pain.

The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S., of which myelofibrosis accounts for approximately 18,000 patients.2 In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis3 and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia (AML).4 The median survival for high-risk myelofibrosis patients is less than 1.5 years, while the median survival for patients with myelofibrosis overall is approximately 6 years.4