On January 4, 2018 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported that the U.S. Food and Drug Administration (FDA), following the customary 30 day review period, has accepted its submission without comment, providing clearance for the OVATION II Study, the Company’s planned Phase I/II clinical trial of GEN-1, its DNA-based immunotherapy for the localized treatment of ovarian cancer (Press release, Celsion, JAN 4, 2018, View Source [SID1234522887]). The Phase I/II trial was developed with extensive input from the Company’s Medical Advisory Board. The OVATION II Study builds on the highly promising clinical and translational research data from the Phase IB dose-escalating OVATION Study where enrolled patients received escalating weekly doses of GEN-1, from levels beginning at 36mg/m², to 47mg/m², 61mg/m² and 79mg/m² weekly for 8 treatments in total, in combination with neoadjuvant chemotherapy, followed by interval debulking surgery.

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This next Phase I/II study is designed with a single dose escalation phase to 100 mg/m² to identify a safe and tolerable dose of GEN-1 while maximizing an immune response, followed by a continuation at the selected dose in Phase II in an open label, 1:1 randomized design up to 90 patients with Stage III/IV ovarian cancer at up to fifteen U.S. centers. The study is powered to show a 33% improvement in the primary endpoint, progression-free survival (PFS), when comparing GEN-1 with neoadjuvant chemotherapy versus neoadjuvant chemotherapy alone.

Progression-free survival for patients treated per protocol in the Phase IB OVATION Study continues to be followed. Of the thirteen patients who received GEN-1 treatment in all four dose escalating cohorts, only four patients’ cancer has progressed to-date. This compares favorably to the historical median progression-free survival of 12 months for newly-diagnosed patients with Stage III and IV ovarian cancer that undergo neoadjuvant chemotherapy followed by interval debulking surgery. Summarized below are the latest PFS results for all patients treated per protocol in the Phase IB OVATION Study:

Cohort 1 (36 mg/m²) – All patients have progressed; Average PFS was 19.25 months; Longest progression-free patient in 1st cohort was 24.8 months.

Cohort 2 (47 mg/m²) – No patients have progressed after 21 months.

Cohort 3 (61 mg/m²) – One patient has progressed after 14 months; Two other patients in 3rd cohort are progression free over 17 months.

Cohort 4 (79 mg/m²) – No patients have progressed; Average PFS for these five patients in 4th cohort is 14 months.
"In previous clinical studies performed to date, GEN-1 has demonstrated excellent safety and impressive clinical activity supported with dose dependent, pro-immune improvement in the tumor micro environment. A onetime dose escalation may prove to be even more impressive," stated Dr. Nicholas Borys, Celsion’s senior vice president and chief medical officer. "As we continue to follow patients, the latest PFS analysis from the OVATION Study is showing a median of at least 15.4 months in the as-treated group which compares favorably to a historical control of 12 months. Our highest dose cohort has not demonstrated any progressions at our current 14 month follow up. This same cohort also had a 100% R0 surgical resection rate. One of our patients in the OVATION Study even had a complete pathological response."

The Company expects to initiate enrollment of the Phase I portion of the OVATION II Study in the first half of 2018. The Company expects to have 25% of the study enrolled by the end of 2018. Due to the open label design, clinical data will be disclosed throughout the execution of the trial as it is released by the study’s investigators.

"GEN-1 holds the potential for tremendous promise as a cancer treatment in the rapidly emerging area of immunotherapy. This new trial will evaluate GEN-1’s value as an adjuvant to current standard of care in newly diagnosed Stage III/IV ovarian cancer patients with a relatively healthy immune system. We look forward to initiating the study in the first half of 2018," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "Designed in consultation with leading medical experts, this Phase I/II trial is expected to define an optimal dose, demonstrate GEN-1’s clinical benefit when compared with current standard of care, and provide insights on powering for a registration program as the candidate progresses through development."

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy in patients with peritoneally metastasized ovarian cancer, and a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer.

On January 4, 2018 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that Jonathan Lim, M.D., its Chairman and Chief Executive Officer, will make a presentation at the 36th Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2018, at 9:00 a.m. Pacific time (Press release, Ignyta, JAN 4, 2018, View Source [SID1234522878]). The conference will be held at the Westin St. Francis Hotel in San Francisco.

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A webcast of the presentation will be available during the presentation in the Investors section of the company’s website at View Source, and will be archived and available at that site for 14 days.

On January 4, 2018 Aduro Biotech, Inc. (NASDAQ:ADRO) reported that Stephen T. Isaacs, chairman, president and chief executive officer of Aduro, will present at the 36TH Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Thursday, January 11, 2018, at 9:00 am Pacific Time (Press release, Aduro Biotech, JAN 4, 2018, View Source;p=RssLanding&cat=news&id=2324984 [SID1234522902]).

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To access the live webcast and subsequent archived recording of this and other company presentations, please visit Aduro’s website at www.aduro.com.

On January 4, 2018 ChemoCentryx, Inc., (Nasdaq:CCXI) reported that it will receive a $50 million milestone payment from Vifor Fresenius Medical Care Renal Pharma (VFMCRP), a company of the Vifor Pharma Group and Fresenius Medical Care (Press release, ChemoCentryx, JAN 4, 2018, View Source [SID1234522888]). The milestone was triggered by the European Medicines Agency (EMA)’s validation of the Company’s Conditional Marketing Authorization (CMA) application for avacopan in the treatment of patients with anti-neutrophil cytoplasmic auto-antibody-associated vasculitis (ANCA-associated vasculitis), announced earlier today. In addition to the $50 million milestone from VFMCRP, the Company also entered into a $50 million growth capital financing agreement with Hercules Capital, Inc. (NYSE:HTGC), bringing total new capital commitments of up to $100 million to ChemoCentryx. Such additional capital is expected to provide funding to advance avacopan through topline data from the Phase III ADVOCATE trial as well as potential registration filings in the U.S. and EU.

"The validation of our CMA application by the EMA is a pivotal milestone in our Kidney Health Alliance with Vifor. It is also a major advance in heightening the awareness of the plight of ANCA vasculitis patients," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "The status quo is simply not good enough for ANCA patients and new therapies are desperately needed. The decision of the EMA to validate our conditional marketing application will enable a thorough examination of how we at CCXI and our partners at Vifor may create just such a valuable new paradigm for ANCA treatment."

ChemoCentryx, which is responsible for the discovery and development of avacopan, owns and retains the commercial rights to the drug in the United States and China, and VFMCRP has licensed the rights to commercialize the drug in all other countries. Under the terms of the Kidney Health Alliance with Vifor Pharma, which comprises both avacopan and CCX140, ChemoCentryx has received a total of $155 million in upfront cash and cash commitments in addition to the $50 million milestone announced today. ChemoCentryx is eligible to receive additional payments upon the achievement of certain development, regulatory and sales-based milestones, as well as tiered double-digit royalties on potential net sales of avacopan and CCX140 in the Vifor licensed territories.

The $50 million credit facility from Hercules Capital comprises three tranches. The first tranche of $15 million, of which $5.0 million was funded upon closing of the agreement, is available through June 2018. The remaining $35 million is available in two additional tranches, subject to certain conditions. The term loan has a 24-month interest-only period from initial funding, which is extendable to 30 months upon the achievement of certain milestones and matures in 48 months. Further information with respect to the growth capital financing agreement with Hercules is contained on a Form 8-K to be filed by ChemoCentryx with the Securities and Exchange Commission.

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On January 4, 2018 PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, reported the appointment of Sanuj K. Ravindran, M.D., to the position of president and chief executive officer (Press release, PellePharm, JAN 4, 2018, View Source [SID1234576282]). In parallel, Dr. Ravindran will join BridgeBio Pharma, PellePharm’s lead investor, as CEO-in-Residence, to advance its broader orphan dermatology portfolio.

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"Dr. Ravindran’s biopharma industry experience and rare disease expertise will offer enormous strategic benefit to PellePharm as we move into our next stage of development," said Ervin Epstein, M.D., chief medical officer and co-founder of PellePharm. "Having been successful with the management and growth of multiple biopharma companies, Dr. Ravindran brings the right experience to PellePharm so that we may offer topical patidegib to patients as swiftly as possible."

Dr. Ravindran brings more than 15 years of strategic and operational biopharma experience to PellePharm. Most recently, he was chief business officer at aTyr Pharma ("LIFE"), a clinical stage rare disease-focused biotechnology company, where he led corporate and financial strategy, business development, and investor relations. Prior to that, Dr. Ravindran was senior vice president of corporate development for The Medicines Company ("MDCO"), where he worked to execute multiple transactions totaling more than $2 billion in potential aggregate value. Previously a practicing physician, Dr. Ravindran began his industry career as a venture capitalist for 10 years with Burrill & Company, Radius Ventures, and Asian Healthcare Fund. Dr. Ravindran is trained in Internal Medicine and completed his residency training at Thomas Jefferson University Hospital. Dr. Ravindran received his B.A. from Northwestern University, his M.D. from Jefferson Medical College and his MBA from the Kellogg School of Management.

"I am thrilled to join PellePharm at such an important juncture, as the Company prepares to advance topical patidegib one step closer to patients with Gorlin Syndrome. With PellePharm’s scientific premise, clinical progress, and recently strengthened leadership team, the company is well on its way to meeting its mission of delivering therapies for rare genetic dermatological conditions," said Dr. Ravindran.

PellePharm today also announced that it has expanded its executive team to enhance regulatory and operational capabilities. Alix Alderman is now vice president of regulatory affairs at PellePharm, and Gerd Kochendoerfer, Ph.D., is vice president of technical operations and program management. Both Ms. Alderman and Dr. Kochendoerfer bring many years of experience in drug development, quality management and global regulatory affairs.

"We are pleased to welcome Dr. Ravindran, Dr. Kochendoerfer and Ms. Alderman," said Neil Kumar, CEO and co-founder of BridgeBio Pharma. "At this inflection point, having the right team in place, with the collective experience this group brings, enables PellePharm to more ably achieve its goal of helping patients with serious unmet dermatologic conditions."

About Patidegib

Topical patidegib gel has shown early promise in a Phase 2 clinical study in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials, but without the adverse systemic side effects of oral hedgehog inhibitors. Patidegib’s gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome. Topical patidegib currently is being studied in a United States-based Phase 2 clinical trial for the treatment of sporadic basal cell carcinomas (BCCs).

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by mutations in the tumor suppressor gene encoding Patched1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to hundreds of basal cell carcinomas, especially on the face and sun-exposed areas.

With no FDA-approved drugs available for Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive and scarring. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including BCCNS, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, or Nevoid Basal Cell Carcinoma Syndrome.