Rich Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Rich Pharmaceuticals, 2017, DEC 29, 2017, View Source [SID1234522790]).
On December 29, 2017 Progenics Pharmaceuticals, Inc. (NASDAQ:PGNX), an oncology company developing innovative medicines and imaging analytical tools for targeting and treating cancer, reported that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for AZEDRA in patients with malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors (Press release, Progenics Pharmaceuticals, DEC 29, 2017, View Source [SID1234522856]). The FDA granted Progenics’ request for Priority Review and has set an action date of April 30, 2018 under the Prescription Drug User Fee Act (PDUFA).
"With no FDA-approved therapies for these rare tumors, AZEDRA has the potential to address the high unmet need of patients with malignant pheochromocytoma and paraganglioma," said Mark Baker, Chief Executive Officer of Progenics. "We are pleased that the FDA has accepted our NDA with Priority Review, and look forward to working with the Agency during the review process. At the same time, we will continue to lay the groundwork for our commercial plan and prepare to launch quickly following a potential approval."
The NDA is supported by data from a pivotal phase 2b open-label, multi-center trial that was conducted under a Special Protocol Assessment (SPA) with the FDA. The trial met the primary endpoint evaluating the proportion of pheochromocytoma and paraganglioma patients who achieved a 50% or greater reduction of all antihypertensive medication for at least six months, and showed favorable results from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria In Solid Tumors (RECIST). AZEDRA was also shown to be safe and generally well tolerated.
AZEDRA (iobenguane I 131) is a high-specific-activity radiotherapeutic product candidate in development as a treatment for malignant, recurrent, or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors of neural crest origin. AZEDRA is a substrate for norepinephrine reuptake transporter which is highly expressed on the cell surface of neuroendocrine tumors. AZEDRA has been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy designation in the U.S. Under a SPA agreement with the FDA, a phase 2b pivotal study has been completed in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma. There are currently no FDA-approved therapies for the treatment of this ultra-rare disease.
About Pheochromocytoma and Paraganglioma
Pheochromocytoma and paraganglioma are rare neuroendocrine tumors that arise from cells of the autonomic nervous system. Pheochromocytoma forms in the adrenal medulla, whereas paragangliomas form outside the adrenal gland. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening hypertension, heart failure, and stroke in these patients. Malignant and recurrent pheochromocytoma and paraganglioma may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.
On December 29, 2017 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported an update from several clinical programs and provided a 2018 business overview (Press release, Bio-Path Holdings, 29 29, 2017, View Source [SID1234522814]).
"We are very excited about the potential for Bio-Path as we enter 2018. The year ahead is expected to be highlighted by a variety of value-creating milestones across a number of important clinical development programs aimed at further validating our DNAbilize platform as a potential treatment for a variety of oncology indications," stated Peter H. Nielsen, chief executive officer of Bio-Path Holdings. "We continue to advance our unique platform technology to address a number of cancers that remain unresponsive to current treatment paradigms."
"We are very encouraged about the potential for our DNAbilize technology, which is supported by compelling earlier data that show prexigebersen to be safe and efficacious against a wide range of cancer indications and are hopeful that these positive data will be replicated in our ongoing late-stage clinical trials," continued Mr. Nielsen.
Phase 2 Study of Prexigebersen in De Novo AML Patients
Bio-Path is conducting a Phase 2 clinical trial of its lead drug candidate, prexigebersen, in combination with frontline therapy low dose cytarabine (LDAC) in de novo acute myeloid leukemia (AML) patients who are ineligible or unwilling to undergo intensive induction therapy. The single arm trial is designed for up to 54 evaluable patients with an interim analysis performed after 19 patients.
To-date in this study, over 50 potential patients have been pre-screened, 26 patients have been screened, 23 patients have been enrolled and 17 patients have been deemed evaluable with 6 additional patients currently undergoing treatment. Bio-Path expects the 19 patient pre-specified analysis to be completed in early 2018, at which time the assessment of these patients will be addressed by Bio-Path.
Plans for a pivotal trial will be discussed with the FDA if these results exceed expectations for current standard of care therapy.
Phase 2a Study of Prexigebersen in Accelerated and Blast Phase CML Patients
Bio-Path today announces the initiation of its Phase 2a clinical study of prexigebersen for the treatment of chronic myeloid leukemia (CML) in accelerated and blast phase patients. The trial is being conducted at The University of Texas MD Anderson Cancer Center as a potential salvage therapy for accelerated and blast phase CML patients.
Two cohorts of three evaluable patients each will be enrolled to evaluate two doses (60 mg/m2 and 90 mg/m2) of prexigebersen in combination with the front-line treatment dasatinib.
Phase 1 Study of BP1002 in Refractory or Relapsed Lymphoma Patients
In 2018, Bio-Path intends to initiate a Phase 1 clinical trial of BP1002, an antisense RNAi nanoparticle targeting the Bcl-2 protein, in refractory or relapsed lymphoma patients. The clinical trial would evaluate the safety of BP1002 in several dose escalating cohorts to determine a maximum tolerated dose and/or optimal biologically active dose.
On December 29, 2017 SELLAS Life Sciences Group, Inc. (Nasdaq:SLS) (SELLAS Inc.) reported that the proposed merger of the businesses of SELLAS Life Sciences Group Ltd. (SELLAS Ltd.) and Galena Biopharma, Inc. (Galena) has closed effective December 29, 2017, following approval by Galena’s stockholders (Press release, Sellas Life Sciences, DEC 29, 2017, View Source [SID1234526857]). Upon completion of the merger, Galena was renamed "SELLAS Life Sciences Group, Inc." and now features a late-stage pipeline led by novel immunotherapies targeting a broad range of indications in hematologic and solid malignancies. SELLAS Inc. is expected to commence trading on the Nasdaq Capital Market on January 2, 2018, under the ticker symbol "SLS".
"This public listing provides us with the opportunity to further develop our novel immunotherapies for a wide range of cancers with unmet medical needs as a public company," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS Inc. "Over the past year, we have advanced the development of galinpepimut-S (GPS), our Wilms Tumor 1-targeting immunotherapy, through collaborations with Merck & Co., among others, as well as our ongoing Phase 2 trials of GPS in patients with multiple myeloma, as a monotherapy, and with ovarian cancer, in combination with Bristol-Myers Squibb’s nivolumab. We also had successful End-of-Phase 2 meetings with the FDA pertaining to our planned Phase 3 studies in acute myeloid leukemia and malignant pleural mesothelioma. We are excited by the potential of GPS, both as a monotherapy and in combination with other agents to serve as a promising therapy for patients with a broad array of cancers."
"I am delighted to be working with Angelos and the rest of the SELLAS Inc. team, who have been responsible for building the company and progressing its novel, high potential pipeline to address the unmet needs of patients battling various types of cancer. The transition to being a publicly traded company marks a significant milestone, providing the opportunity for greater financial resources and enhanced corporate structure to better advance the company’s clinical programs," noted Jane Wasman, newly-appointed Chair of SELLAS Inc.’s Board of Directors.
Following completion of the merger, the combined company will relocate its headquarters to New York City, NY, and operate under the management team of SELLAS Ltd.: Dr. Angelos M. Stergiou (Chief Executive Officer), Aleksey N. Krylov (Interim Chief Financial Officer), Dr. Nicolas J. Sarlis (Chief Medical Officer), and Dr. Gregory M. Torre (Chief Regulatory Officer). The Board of Directors now is led by Jane Wasman (Chair), along with Stephen F. Ghiglieri, Fabio Lopez, Dr. David A. Scheinberg, Dr. Angelos M. Stergiou, Robert L. Van Nostrand and John Varian.
Guggenheim Securities acted as SELLAS Ltd.’s financial advisor for the transaction, and Cooley LLP and Conyers Dill & Pearman acted as SELLAS Ltd.’s legal counsel. Canaccord Genuity Inc. acted as Galena’s financial advisor for the transaction and Paul Hastings LLP and BeesMont Law Limited acted as Galena’s legal counsel.
About the Transaction
In connection with and prior to the closing of the merger, Galena effected a 1-for-30 reverse stock split of its common stock. As a result of the reverse stock split, every 30 shares of Galena common stock issued and outstanding or held by Galena in treasury immediately prior to the effective time of the reverse stock split was automatically reclassified into one fully paid and nonassessable share of Galena common stock. Pursuant to the merger, the holders of SELLAS Ltd. shares outstanding immediately prior to the merger received 43.9972 shares of Galena common stock in exchange for each SELLAS Ltd. share in the merger. Post-merger and post-reverse split, SELLAS Inc. now has approximately 5,766,891 shares of common stock issued and outstanding, with prior SELLAS Ltd. securityholders collectively owning approximately 67.5% of the combined company, and prior Galena securityholders collectively owning approximately 32.5% of the combined company, in each case on a fully diluted basis, without taking into account certain out-of-the-money Galena warrants.
About SELLAS Inc.’s Pipeline
The combined company features a late-stage pipeline led by novel immunotherapies targeting a broad range of indications in hematologic and solid tumor malignancies. SELLAS Inc. plans to initiate a Phase 3 trial of GPS for the treatment of acute myeloid leukemia (AML) in 2018, pending funding availability, and has also successfully completed a Phase 2 study of GPS in malignant pleural mesothelioma (MPM). End-of-Phase 2 meetings have been completed with the U.S. Food and Drug Administration (FDA) for GPS in both indications.
In addition, SELLAS Inc. is currently conducting two Phase 2 trials of GPS, in multiple myeloma as a monotherapy, as well as in a combination trial in ovarian cancer with nivolumab (OPDIVO; Bristol-Myers Squibb). SELLAS Inc. is currently preparing for an open label, 5-arm ‘basket’ type combination trial of GPS in combination with the anti-PD-1 therapy pembrolizumab (KEYTRUDA; Merck & Co.). SELLAS Inc. also assumes Galena’s Phase 2 investigator-sponsored nelipepimut-S clinical trials in breast cancer and a controlled release version of anagrelide for potential internal development or strategic partnership.
On December 29, 2017 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics reported that the Company’s President and CEO, Dr. Geert Cauwenbergh, will present at the 10th Annual Biotech Showcase (Press release, RXi Pharmaceuticals, DEC 29, 2017, View Source [SID1234522789]). Taking place during one of the industry’s largest annual healthcare investor conferences, this investor and partnering conference attracts pharmaceutical executives from around the world focused on investment and business development opportunities in the life sciences industry. The conference will be held January 8–10, 2018 at the Hilton San Francisco Union Square, California.
Date: Monday, January 8, 2018
Time: 9:30am Pacific
Track:Yosemite – C (Ballroom Level)
Dr. Cauwenbergh will present an overview of the Company’s novel self-delivering RNAi (sd-rxRNA) technology and the multiple business development and commercial opportunities available based on this proprietary platform. The presentation will be webcast and available on the "Investors – Events and Presentations" section of the Company’s website, www.rxipharma.com.
About RXi’s self-delivering RNAi (sd-rxRNA) technology platform
sd-rxRNA, RXi’s proprietary self-delivering RNAi platform, is a single chemically modified compound with delivery and therapeutic properties built directly into the compound itself. The compound is asymmetrical with a phosphorothioate backbone and contains chemical modifications that provide for efficient cellular uptake and gene silencing. These compounds are potent, stable and specific, and demonstrated to be safe and active in a clinical setting.
RXi’s novel sd-rxRNA technology differs from natural and most synthetic RNA interference (RNAi) molecules in that they are chemically modified to allow for efficient internalization of the compounds by cells and silencing of the targeted genes. Importantly, unlike other naked siRNA compounds, delivery of sd-rxRNAs are not limited to a specific cell type. For local delivery and ex vivo cell-based therapeutic applications, our compounds do not require delivery vehicles. This is a major advantage, since delivery vehicles can have related toxicity that affects cell viability. sd-rxRNA has demonstrated nearly 100 percent transfection efficiency with high cell viability in numerous cell types.