On January 6, 2017 Atreca, Inc., a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported that Tito A. Serafini, Ph.D., Atreca’s President, Chief Executive Officer, and CoFounder, will participate in a panel discussion on immuno-oncology (IO) during the 9th Annual Biotech Showcase Conference on Wednesday, January 11, 2017, at 11:00a.m. Pacific Time (Press release, Atreca, JAN 6, 2017, View Source [SID1234522958]).

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The panel, titled "The Overflowing IO Buffet: Next Gen IO Antibodies, Oncolytic Viruses, Bispecifics, Adoptive Cell Therapies and Oral Immunometabolism / Immuno-Epigenetic Agents," will be held at the Hilton San Francisco Union Square in Continental Room 2.

On January 6, 2017 Acceleron Pharma Inc. (NASDAQ: XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported its major corporate research and development goals and priorities for 2017 (Press release, Acceleron Pharma, JAN 6, 2017, View Source [SID1234517348]).

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"With our Phase 3 luspatercept programs in MDS and beta-thalassemia advancing on plan, 2017 will be a transformational year for Acceleron. We are looking ahead to the clinical, regulatory and commercial milestones that will help us achieve our vision of becoming a fully integrated biopharmaceutical company," said Habib Dable, President and Chief Executive Officer of Acceleron. "Additionally, we continue to advance and expand our wholly-owned portfolio of innovative protein therapeutics for patients with serious diseases. With multiple Phase 3 and Phase 2 trials ongoing and new INDs expected in 2017 and 2018, we believe that our pipeline of therapeutic candidates positions us to create significant value for our shareholders while making a meaningful difference in the lives of patients who have limited treatment options."

The Company’s major research and development goals and priorities are highlighted below:

Luspatercept in Rare Blood Disorders
Luspatercept is being developed to treat patients who have anemia associated with rare blood disorders, including beta-thalassemia and malignant disorders such as myelodysplastic syndromes (MDS) and myelofibrosis.
Goals for luspatercept in myelodysplastic syndromes (MDS):
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Complete patient enrollment in the MEDALIST Phase 3 clinical trial in the second half of this year
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Release topline results for the MEDALIST Phase 3 trial by the end of next year
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Evaluate and design a clinical and regulatory strategy for luspatercept in first-line lower risk MDS patients

Goals for luspatercept in beta-thalassemia:

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Complete patient enrollment in the BELIEVE Phase 3 clinical trial in the second half of this year
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Release topline results for the BELIEVE Phase 3 trial by the end of next year
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Initiate a Phase 2 trial in patients with non-transfusion dependent beta-thalassemia by the end of this year

Goals for luspatercept in myelofibrosis:
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Initiate a Phase 2 trial in myelofibrosis by the end of this year

ACE-083 in Neuromuscular Disease
ACE-083 is being developed to increase muscle mass and strength in target muscle groups for diseases such as facioscapulohumeral muscular dystrophy (FSHD), where patients experience focal muscle loss. Acceleron plans to:
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Present initial topline results from the open label, dose-escalation stage of the Phase 2 study in FSHD in late 2017
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Initiate the randomized, double-blind, placebo-controlled stage of the Phase 2 study in 2018
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Initiate a Phase 2 clinical trial in a second neuromuscular disease

Pipeline Expansion
Acceleron continues its research on several preclinical protein therapeutics targeting fibrotic disorders, vascular disease, and musculoskeletal disease. Acceleron’s current goals for research and pipeline expansion include:
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Initiate a Phase 1 healthy volunteer study with ACE-2494 this year
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Conduct IND-enabling development work to advance a new protein therapeutic to the clinic in 2018
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Host an investor and analyst research day to discuss ongoing preclinical research and potential future disease areas in the second quarter of this year

Dalantercept in Advanced Renal Cell Carcinoma
Dalantercept is being developed in advanced renal cell carcinoma in combination with axitinib to further inhibit tumor angiogenesis. Acceleron expects to present topline results from the Phase 2 DART study in the second half of 2017. The primary endpoint of this trial, progression-free survival (PFS), is an event-driven assessment.

A slide presentation describing these research and development goals and other information will be available on the Investors page on the Company’s website at www.acceleronpharma.com on Monday, January 9, 2017.

On January 6, 2017 Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, reported it has expanded the fourth cohort from its Phase II SUMMIT clinical trial of its lead drug candidate PB272 (neratinib) as a single agent in patients with solid tumors who have an activating HER2 mutation (basket trial) (Press release, Puma Biotechnology, JAN 6, 2017, View Source [SID1234517291]). The cohort that has been expanded is the cohort that includes patients with metastatic cervical cancer and whose tumors have a HER2 mutation.

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The Phase II SUMMIT basket trial is an open-label, multicenter, multi-histology, international study to evaluate the safety and efficacy of PB272 administered daily to patients who have solid tumors with activating ERBB mutations including EGFR, HER2 and HER3. The cohorts included in the basket study receiving neratinib monotherapy are hormone receptor-negative breast cancer; biliary tract cancers; endometrial cancer; gastric/esophageal cancer; ovarian cancer; and all other solid tumors with a HER2 mutation. The cohorts receiving combination treatment are hormone receptor-positive breast cancer (neratinib plus fulvestrant) and bladder cancer (neratinib plus paclitaxel). The cervical cancer patients initially entered the study in the "other solid tumors with a HER2 mutation" cohort, and due to the preliminary activity seen in the trial, the Company has expanded a separate cervical cancer cohort pursuant to the protocol for the trial. The expanded HER2-mutant cervical cancer cohort will now enroll a total of 18 patients.

Dr. David Hyman, Director, Developmental Therapeutics at Memorial Sloan Kettering Cancer Center and principal investigator of the trial, stated, "We are pleased to expand our evaluation of neratinib in metastatic cervical cancer, an orphan and deadly disease with few treatment options. We believe this once again demonstrates the value of the basket study approach, in particular for developing targeted therapy for rare diseases with clinically-actionable mutations. We look forward to presenting the full results of the SUMMIT trial at a medical meeting in 2017."

"We are pleased to expand the fourth cohort in the basket trial," said Alan H. Auerbach, Chief Executive Officer and President of Puma. "Although it is early, we are pleased with the initial activity that we are seeing in the patients with HER2 mutated cervical cancer in the trial. We look forward to continuing enrollment into this expanded cohort and look forward to presenting the full results from the SUMMIT study in 2017."

On January 5, 2017 Avidity Biosciences reported the completion of a $16 million Series B financing round to support the development of its Antibody-siRNA Conjugate (ASC) platform (Press release, Avidity Biosciences, JAN 5, 2017, View Source [SID1234521096]). The Series B round includes investment of $10 million in new capital and conversion of $6 million in convertible debt. Takeda Pharmaceuticals, through its venture group, led the Series B round, and both new and existing investors participated, including Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners and Tavistock Life Sciences.

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"Our ASC platform unites two of the most impactful innovations over the past twenty years – monoclonal antibodies and oligonucleotides – to create a new class of precision medicines," said Troy Wilson, Ph.D., J.D., president and chief executive officer of Avidity Biosciences. "Although siRNA-based therapeutics have demonstrated significant clinical and commercial promise, conventional approaches are limited to targeting diseases of the liver. Because ASCs use antibodies to overcome barriers of delivering siRNA, they have the potential to impact a broader range of therapeutic areas. Our goal is to partner with leading pharmaceutical and biotechnology companies to deliver a pipeline of ASCs targeting genetic drivers of disease."

ASCs link a monoclonal antibody—designed against a specific molecular target—with a siRNA therapeutic payload, allowing the conjugate to have unmatched specificity and selectivity. In preclinical models, ASCs have shown potential to knockdown messenger RNA levels in multiple important cell types and tissues, including tumor, muscle, heart, lung, liver and B cells. In addition, ASCs have drug-like properties comparable to antibodies and antibody-drug conjugates.

In connection with the financing, Avidity Biosciences annouced that Michael Martin, Ph.D., global head of Takeda Ventures Inc., and Todd Brady, director of finance and investments of Brace Pharma Capital, will join its board of directors. Tony Hsu, founder and chief investment officer of Alethea Capital, will also join the board as a non-voting member.

"We believe Avidity’s ASC platform offers a compelling approach that builds on successes with antibodies, ADCs and oligonucleotide-based therapeutics," said Dr. Martin. "Avidity has recruited a top-notch team and made significant progress against its scientific and business goals. We look forward to working with the company to realize the promise of ASCs as a new class of precision medicines."

On January 5, 2017 Neon Therapeutics, an immuno-oncology company developing neoantigen-based therapeutic vaccines and T cell therapies to treat cancer, reported the successful completion of a $70 million Series B financing (Press release, Neon Therapeutics, JAN 5, 2017, View Source [SID1234517277]). Proceeds from the financing will be used to advance Neon Therapeutics’ lead program NEO-PV-01, a fully personalized neoantigen vaccine, through an ongoing Phase 1b clinical trial. In addition, this investment will support preclinical development of NEOPTC-01, a personalized adoptive T cell program, and the Shared Neoantigen Program.

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The Series B financing was led by Partner Fund Management. Joined by existing investors Third Rock Ventures and Access Industries, additional new investors included Fidelity Management & Research Company, Wellington Management Company, Inbio Ventures and Nextech Invest.

"The investor quality in this financing reflects the promise of the neoantigen biology platform, as well as our leading position," said Hugh O’Dowd, chief executive officer of Neon Therapeutics. "This capital is a testament to our strong team and our approach to neoantigen-based therapeutics, and will provide important resources to bring potentially life-changing medicines to cancer patients in need."