On January 16, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics reported its 2018 business strategy during a webcast presentation at the Biotech Showcase conference which brought together over 3,500 life science decision makers and investors from over 50 countries to collaborate and discuss industry trends (Press release, RXi Pharmaceuticals, JAN 16, 2018, View Source [SID1234523145]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"After a thorough review of our business operations, development programs and financial resources, a decision was made to focus our efforts on RXi’s expanding and promising immuno-oncology programs to accelerate growth and in turn support a return on investment for our shareholders," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He stated that: "The positive clinical results recently announced from our Phase 2 dermatology study validate the safety and efficacy of our sd-rxRNA therapeutics in humans. These outcomes coupled with robust preclinical results in our immuno-oncology program, further affirm the Company’s focused development path. With a much-improved regulatory landscape for the development of cancer therapeutics, we believe that there is great potential for our sd-rxRNA compounds to provide major advances that could change the paradigm in cancer treatments. Furthermore, the feedback that we have received, through relationships with leading cancer centers, potential business partners as well as institutional investors, in the context of last week’s Biotech Showcase meeting, endorses our sharpened strategy. It is our goal to develop novel immuno-oncology treatments to benefit patients as well as our long-term shareholders and business partners."

2018 Business Strategy

RXi will focus development initiatives on novel immuno-oncology therapeutics using its proprietary sd-rxRNA platform. Focusing the development portfolio on Immuno-oncology, with a near term focus on Adoptive Cell Transfer (ACT), will streamline and reduce our quarterly burn-rate for 2018. The Company is aiming to enter the clinic with at least one of our compounds in the next 18 months, targeting a large multi-billion dollar market. This will be achieved by:

Accelerating the development of RXI-762 and RXI-804, sd-rxRNA compounds targeting PD-1 and TIGIT respectively, for use in the treatment of solid tumors in the context of ACT. Finalize cGMP manufacturing of lead compound.
Expanding internal development efforts and external collaborations with various existing cell-based approaches, evaluating hematopoietic stem cells, NK cells, TILs, CAR T, TCR and engineered NK cells.
Developing immuno-oncology targets beyond checkpoint inhibition, i.e. cell differentiation.
Expanding our research efforts to further demonstrate that our technology is ideally suited to be combined with existing immune effector cell expansion/manufacturing.
Monetize Existing Dermatology and Ophthalmology Franchises:

Relating to the operational review, RXi intends to partner/out-license both its Dermatology and Ophthalmology Franchises. Successfully completing these transactions should provide non-dilutive funding for the Company’s focused development path in Immuno-oncology.

Each of these Franchises is comprised of preclinical and clinical-stage assets broadly covered by a robust intellectual property estate. These assets include:

Dermatology

RXI-109: Phase 2 asset for dermal hypertrophic scarring with positive and statistically significant results addressing a USD 1-3B market.
RXI-231: Topical cosmetic ingredient in a proprietary formulation with positive results reducing a change of skin tone (pigmentation) triggered by UV (p< 0.04) addressing an estimated USD multi-billion dollar market.
RXI-185: Cosmetic ingredient that reduces collagenase in in vitro models. May be developed as a cosmetic to improve the appearance of aging skin.
Samcyprone: Phase 2 small molecule topical immuno-therapy, with proven efficacy in cutaneous warts with an estimated market size USD 2-4B.
Access to the self-delivering platform for human therapeutic and diagnostic use in the field of dermatology, providing access to new compounds targeting proteins of relevance for the treatment of skin diseases.
The intellectual property estate for RXi’s Dermatology Franchise is comprised of 14 patent families covering: RXI-109, RXI-231, RXI-185 and Samcyprone. Importantly, this estate includes 18 granted patents broadly covering the sd-rxRNA platform, including RXI-109 in the US, Europe, Japan and China and Samcyprone in the US.

Ophthalmology

RXI-109: Phase 1/2 asset with proven safety, using intra-ocular injections, for wet AMD with estimated market size USD 3-5B. Readouts expected Q1 2018.
Preclinical data package supporting development of RXI-109 by intraocular injection or topical application to the eye for retinal or corneal scarring indications.
Patented set of sequences against a variety of relevant proteins for targeting ocular disorders. Also, sd-rxRNA compounds against targets which may be involved in retinoblastoma or retinal or corneal scarring.
Preclinical intra-ocular asset (sd-rxRNA targeting VEGF) with dose-dependent effects and tolerability demonstrated in a rodent model.
Access to the self-delivering platform for human therapeutic and diagnostic use in the field of ophthalmology, providing ready access to new compounds targeting proteins of relevance for the treatment of ophthalmic diseases.
RXi’s Ophthalmology intellectual property estate includes 21 patent families. This estate includes 73 patents covering the composition and methods of use the sd-rxRNA platform and targets and sequences from the OPKO assets, including the use of RXI-109 for the treatment of ocular scarring in the US, Japan and China.

The webcast presentation took place on January 8, 2018, and the relating slides and audio may be found on 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 significant 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.

On January 16, 2018 MannKind Corporation (Nasdaq:MNKD), focused on the development and commercialization of inhaled therapeutic products for patients with diseases such as diabetes and pulmonary arterial hypertension, reported that its Chief Executive Officer, Michael Castagna, will present at NobleCon14 — Noble Capital Markets’ Fourteenth Annual Investor Conference on Monday, January 29, 2018 at 10:00 Eastern Standard Time at the W Hotel in Fort Lauderdale, Florida (Press release, Mannkind, JAN 16, 2018, View Source [SID1234523147]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A high-definition, video webcast of the presentation will be available the following day on the Company’s web site www.mannkindcorp.com, and as part of a complete catalog of presentations available at Noble Capital Markets’ websites: www.noblecapitalmarkets.com, and www.nobleconference.com. You will require a Microsoft SilverLight viewer (a free download from the presentation link) to participate. The webcast and presentation will be archived on the Company’s website and on the Noble websites for 90 days following the event.

On January 16, 2018 Gamida Cell, a leading cellular and immune therapeutics company, reported the initiation of a phase I study evaluating its proprietary NAM-expanded natural killer cells (NAM-NK Cells) in patients with relapsed or refractory CD20+ non-Hodgkin lymphoma (NHL) and multiple myeloma (Press release, Gamida Cell, JAN 16, 2018, View Source [SID1234523184]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"There is significant need for novel therapeutic approaches for refractory non-Hodgkin lymphoma and multiple myeloma, which are aggressive malignancies with limited treatment options," said Veronika Bachanova, M.D., Ph.D., hematologist/oncologist at University of Minnesota Health and lead investigator of the clinical study at the Masonic Cancer Center, University of Minnesota. "We initiated this phase I study based on encouraging preclinical data to bring a potential immunotherapeutic treatment approach to patients with these life-threatening cancers."

The phase I study is designed to determine the maximum tolerated dose of NAM-NK Cells; secondary endpoints include overall antitumor response and toxicity. The study is currently recruiting and will enroll approximately 24 patients aged 18 to 70 years old. Participants will undergo a lymphodepleting preparative regimen of cyclophosphamide and fludarabine, and then receive two doses of NAM-NK Cells followed by a short course of interleukin-2 (IL-2). Monoclonal antibodies, rituximab for NHL or elotuzumab for multiple myeloma, are administered prior to and after the NAM-NK Cells infusion to facilitate tumor targeting and antibody dependent cellular cytotoxicity.

"NAM-expanded NK cells have demonstrated increased killing potential and increased in vivo persistence and proliferation in preclinical studies," said Tony Peled, Ph.D., chief scientific officer at Gamida Cell. "We are pleased to see the NAM-NK Cell program enter clinical testing and are committed to collaborating with Dr. Bachanova and her team at the Masonic Cancer Center."

About NAM-NK Cells
Gamida Cell applied the capabilities of its NAM technology to the expansion of highly functional NK cells. In addition to a higher killing potential, NAM-NK Cells secretes higher levels of inflammatory cytokines that facilitate recruitment and activation of other endogenous immune cells to attack tumors. NAM-NK Cells is in phase I development (NCT03019666) in patients with relapsed or refractory CD20+ non-Hodgkin lymphoma and multiple myeloma.

About the Masonic Cancer Center
Masonic Cancer Center, University of Minnesota is a comprehensive cancer center designated by the National Cancer Institute. For more than 25 years, researchers, educators, and care providers have worked to discover the causes, prevention, detection and treatment of cancer and cancer-related disease. Learn more about the Masonic Cancer Center and the phase I clinical trial at cancer.umn.edu.

On January 16, 2018 Aspyrian Therapeutics, Inc., a biotechnology company developing precision-targeted cancer therapies based on a proprietary Photoimmunotherapy (PIT) platform, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RM-1929, a first-in-class precision targeted therapy, for treatment of patients with locoregional recurrent Head and Neck Squamous Cell Carcinoma (HNSCC) that have failed 2 lines of therapies (Press release, Rakuten Aspyrian, JAN 16, 2018, View Source [SID1234535086]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"I am very excited by the progress that Aspyrian has made building on game-changing innovation," said Hiroshi "Mickey" Mikitani, Chairman of Aspyrian. "Aspyrian is aiming to conquer cancer, for life – some might see this as wildly ambitious but we are humbly committed to this as the end goal."

RM-1929 is currently being tested in an ongoing Phase 2 study in recurrent Head and Neck cancer (Study RM-1929/101, NCT02422979). Enrollment has been completed and Aspyrian expects to report top-line data in the second half of 2018. Aspyrian also recently completed a successful end-of-phase 2 meeting with the FDA to discuss interim clinical data from this Phase 2 study. The design of pivotal studies was also discussed, including clinical strategies to potentially support accelerated approval under Subpart E, Accelerated Approval of Biological Products for Serious or Life-Threatening Illnesses, Code of Federal Regulations, Title 21, Volume 7. Based on the FDA feedback, Aspyrian plans to initiate the first global pivotal study for RM-1929, to treat recurrent Head and Neck cancer, within the first quarter of 2018.

"We are extremely pleased that the FDA has granted Fast Track designation for RM-1929, a first-in-class precision targeted therapy, for treatment of patients with locoregional recurrent Head and Neck Squamous Cell Carcinoma (HNSCC) that have failed 2 lines of therapies. The Fast Track designation recognizes the potential of RM-1929 to address the large unmet medical need in this clinical setting," said Dr. Merrill Biel, MD, PhD, Chief Medical Officer of Aspyrian. "The interim results of the Phase 2 study showed improvement in the clinically meaningful endpoints of Overall Response Rate, Progression Free Survival and Overall Survival compared to the historical data for the standard of care treatments currently available to these patients. These data support the potential for RM-1929 to control this disease while preserving normal healthy tissues in the head and neck area that are so critical to maintaining the patient’s quality of life and we look forward to initiating the pivotal clinical trials."

Pivotal studies will include evaluation of RM-1929 as a single agent and in combination with immunomodulators to treat both locoregional and metastatic head and neck cancer lesions. Studies conducted by Aspyrian have shown that treatment with RM-1929 can induce not only rapid tumor destruction, but also drive innate and adaptive anti-cancer immune responses.

"I congratulate the Aspyrian team for successfully driving the progression of RM-1929 towards pivotal studies in recurrent Head and Neck cancer, to start in early 2018. We are delighted that the FDA granted Fast Track designation to RM-1929 for the treatment of patients with recurrent Head and Neck cancer, illustrating their recognition of the large unmet medical need for this severe disease. Based on the interim Phase 2 data, we believe that RM-1929 as a single agent has the potential to offer effective treatment options for patients that have failed all existing therapies, providing the opportunity to treat with curative intent" said Miguel Garcia-Guzman, President and CEO of Aspyrian. "In addition, based on our preclinical and clinical data, we expect that RM-1929-mediated tumor destruction may serve to trigger innate and adaptive anti-cancer immune responses that have the potential to enhance the destruction of the patient’s cancer. Aligned with the visionary leadership of our Chairman Hiroshi Mikitani and our commitment to develop the best possible treatment options for patients, we plan to initiate additional trials evaluating combination treatments of RM-1929 with immunomodulators in early 2018. The combination trials are designed to explore synergistic effects of RM-1929 together with activation of T-cell mediated anti-cancer responses."

In order to advance RM-1929 into clinical studies in Japan, Aspyrian successfully submitted a Clinical Trial Notification (CTN) application for RM-1929 to the PMDA and has initiated a Phase 1, single center, open label clinical study in Japanese patients with recurrent Squamous Cell Head and Neck Cancer.

In addition to RM-1929, Aspyrian is progressing preclinical development of other new proprietary immune oncology approaches that have the potential to eliminate the immunosuppressive behavior associated with tumor microenvironments, and to elicit adaptive systemic immune responses. This approach is anticipated to destroy tumors that are resistant to other classes of immune modulators, such as anti-PD1 antibodies. Preclinical data have shown that Aspyrian’s immune oncology treatment modality has the potential to achieve robust tumor destruction mediated by tumor specific CD8+ T-cells and to elicit long-term cancer‑specific immune memory resulting in robust cancer vaccination. Aspyrian intends to expedite the development of these new immune oncology approaches into clinical studies.

On January 16, 2018 Innovation Pharmaceuticals, (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported shareholders of a productive appearance by Innovation last week at leading health care meetings held in San Francisco, with an audio file of the Biotech Showcase presentation now available on the Company website (Press release, Innovation Pharmaceuticals, JAN 16, 2018, View Source [SID1234523142]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Interest in the Brilacidin Franchise—based on the breadth of its treatment potential—was notable. Strong momentum gained as a result of Brilacidin’s recent positive topline findings seen in both Inflammatory Bowel Disease (IBD) and Oral Mucositis (OM), coupled with previously successful results achieved in Acute Bacterial Skin and Skin Structure Infection (ABSSSI), has delivered a complementary cross-indication scientific anchoring for each Brilacidin indication in the Company’s pipeline.

In particular, Brilacidin-OM received an exceptional degree of attention at the San Francisco shows, further adding to an already robust partnering matrix. Innovation is developing Brilacidin-OM under a FDA Fast Track designation as a novel therapeutic and recently met the primary endpoint in a Phase 2 trial for preventing severe OM as well as a key secondary endpoint by delaying the onset of severe OM. Brilacidin has effectively earned a leadership position as an easily-delivered oral rinse drug candidate in OM—what many analysts predict will become a $1 billion market opportunity within the next few years.

"What must be stressed is that Brilacidin is a mature, later-stage drug candidate with platform potential. It took the recent completion of two Phase 2 trials, in IBD and OM, to further validate the exceptional results achieved from our Phase 2b study in ABSSSI. These recent data, taken in aggregate, are what various actively engaged pharmaceutical companies have desired from us for some time—and they are what triggered a newfound flurry of inbound partnership discussions at the San Francisco conferences," commented Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "Datasets now in hand, reflecting three successfully completed Phase 2 studies across which multiple endpoints were met, have brought the Company to an important inflection point regarding Brilacidin. In coming weeks, we look forward to advancing these discussions with attractive partnership / licensing scenarios, towards determining the best path forward for the Company and its loyal shareholders."

Biotech Showcase Event Recording

An audio file of the Corporate Overview given at 2018 Biotech Showcase, on January 8, in San Francisco, is now available and accessible on the Events and Presentations section of the Company’s website.

Brilacidin-OM (Phase 2 Trial Completed)

As announced earlier, the Company has completed a Phase 2 randomized, double-blind, placebo-controlled trial (see NCT02324335) evaluating Brilacidin’s ability, administered as an oral rinse, to prevent and attenuate OM in patients with Head and Neck Cancer (HNC) receiving chemoradiation with at least 55 Gy across 7 weeks. The study’s primary endpoint was met, with Brilacidin, as a preventative treatment, showing a clear reduction in the incidence of severe OM (SOM) (WHO Grade ≥ 3) compared to placebo. A key secondary endpoint in the trial, delaying the onset of SOM, also was met. The Company has begun exploring patient-friendly improvements to drug delivery (the use of sachets) and will be meeting with the FDA to determine next steps in the clinical development of Brilacidin-OM. Patents for Brilacidin-OM have also been granted globally, most recently in Europe. Worldwide, the potential market for OM, largely untapped, is estimated to be in excess of $1 billion, with the Company considered a top contender in this space.