On December 03, 2018 Oncobiologics, Inc. (NASDAQ:ONS) (the "Company") reported several key corporate events that enhance the Company’s ability to advance the development of its lead product candidate, ONS-5010, a proprietary ophthalmic bevacizumab product candidate for the treatment of wet age related macular degeneration (wet AMD) (Press release, Oncobiologics, DEC 3, 2018, View Source [SID1234616259]). These events are in conjunction with the recent disclosure of new details of the ONS-5010 program and the initiation of the first human clinical study, which the Company announced in November 2018.

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!

The Company has changed its name from Oncobiologics, Inc. to Outlook Therapeutics, Inc., effective immediately. The Company will continue to be listed on the Nasdaq Capital Market and its common stock and Series A warrants will begin trading under the ticker symbols "OTLK" and "OTLKW," respectively, beginning on Tuesday, December 4, 2018.

No action is required by stockholders with respect to the name change. The Company’s common stock has been assigned a new CUSIP number of 69012T 107 and its Series A warrants have been assigned a new CUSIP number of 69012T 115 in connection with the name change. Outstanding securities are not affected by the name change and will not need to be exchanged.

"We believe the timing of this corporate rebranding effectively signals the significance of the recent strategic shift in the business and the high value opportunity we are pursuing in the anti-VEGF ophthalmic market," said Lawrence A. Kenyon, President, Chief Executive Officer and Chief Financial Officer. "The progress we have made throughout 2018 in advancing the ONS-5010 program has brought us to this exciting new stage in the Company’s history. The path ahead is clear and we look forward to providing further updates as we execute upon our strategy."

Two Additions to Executive Leadership Team

The Company announced two additions to its executive leadership team with the appointments of Jeff Evanson as Chief Commercial Officer and Terry Dagnon as Chief Operating Officer.

"Jeff and Terry are welcome additions to the team as they are both highly respected ophthalmic industry veterans and bring extensive drug development expertise to our leadership team," said Mr. Kenyon. "Jeff has an exceptional commercial track record in the ophthalmic space and brings valuable experience and deep know-how in product launches and marketing. Terry is a highly skilled expert in the regulatory field and his extensive experience in strategic drug development will be valuable for moving ONS-5010 through advanced clinical development and into commercialization. In addition, they have been key advisors on the ONS-5010 program to date and we expect them to be integral to the success of the asset as we move forward," stated Mr. Kenyon.

"Joining Outlook Therapeutics is a tremendous opportunity," said Jeff Evanson, newly appointed Chief Commercial Officer. "I am enthusiastic about the commercial prospects for ONS-5010 and its potential to address a need for all patients suffering from wet AMD and other retinal diseases requiring anti-VEGF treatments. I look forward to working with Larry and the team at Outlook Therapeutics in leading the efforts to bring ONS-5010 to market and contributing to the Company’s success."

Jeff Evanson joins the Company with more than 25 years of commercial expertise, most notably with Novartis (Alcon) where he was the Vice President and Global Commercial Head of the Pharmaceutical Franchise between 2010 and 2014 where he was responsible for all aspects of strategy, portfolio (both internal and external opportunities), global brands, launches and campaigns. Prior to Novartis, Mr. Evanson spent 10 years at Medtronic in a variety of pre-commercialization and post-commercialization roles. Most recently, Mr. Evanson led Scott Three Consulting, LLC as Founder and President since April of 2018 and previously was a Managing Director in the Life Science Practice of Navigant. He received his MBA from the University of Minnesota (2001) and a BA in Chemistry from the University of St. Thomas in St. Paul Minnesota (1991). He serves on the Board of Directors of Children’s HeartLink and was formerly a two-term Board Member of Gillette Children’s Hospital in St. Paul, Minnesota, from 2008 to 2014.

Terry Dagnon has more than 20 years of regulatory experience with domestic and global investigational and marketing approvals in the pharmaceutical and medical device industries. He is also experienced in quality and compliance and working with R&D, marketing, sales, legal, and manufacturing, quality and supply chain organizations. Most recently, Mr. Dagnon was Senior Vice President of Operations at Dohmen Life Science Services (DLSS), where he worked with companies to mitigate their compliance risk, ensure quality, and achieve FDA approval for pharmaceutical, biologics, and medical device products. Mr. Dagnon began his career in the pharmaceutical industry as the Regulatory Affairs Manager for Physician Reliance Network Inc. (now known as U.S. Oncology). He continued his regulatory affairs career at Johnson & Johnson Medical Inc. with global regulatory responsibility for the Wound Care, Skin Care and Tissue Engineering franchises. He then served as the North America Head of Regulatory Affairs at Alcon a Novartis company prior to joining DLSS in March 2014. Prior to a career in the medical industry, Mr. Dagnon served 11 years on active duty with the United States Army and was a SFC/E-7 Special Forces Green Beret 18D Senior Non-Commissioned Officer. Mr. Dagnon serves on the Board of Directors of the Colorado Bioscience Association.

Private Placement Proceeds

The Company announced today that it closed on the second tranche (of four) of its $20.0 million private placement of common stock to BioLexis Pte. Limited (BioLexis), the Company’s strategic business partner and largest investor, receiving $4.0 million of cash proceeds in exchange for the issuance of 4,288,624 shares of common stock at $0.9327 per share. The Company has received $12.0 million to date from the sale of its common stock to BioLexis under this private placement, which was previously announced on November 6, 2018. The remaining $8.0 million will be funded in two equal tranches on each of January 3, 2019 and February 1, 2019, subject to achieving certain funding milestones as set forth in the purchase agreement. The Company intends to use the net proceeds from the private placement primarily for clinical trials for its lead product candidate, ONS-5010, and for working capital and general corporate purposes, including agreed repayments on its senior secured notes.

This news release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful, prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 3, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported the presentation of the efficacy and safety results of oral rigosertib in combination with azacitidine (Vidaza) in patients with HR-MDS reported at an oral presentation during the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego (Press release, Onconova, DEC 3, 2018, View Source [SID1234531818]). Rigosertib, the Company’s lead compound, is being evaluated in both intravenous and oral forms.

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!

ORAL PRESENTATION:

Phase 2 Expansion Study of Oral Rigosertib Combined with Azacitidine treatment in Patients with Higher-Risk (HR) Myelodysplastic Syndromes (MDS): Efficacy and Safety Results in HMA Treatment Naïve & Relapsed (Rel)/Refractory (Ref) Patients

Session Name: 637. Myelodysplastic Syndromes — Clinical Studies: Novel Therapeutics I

Date: Saturday, December 1, 2018

Presentation Time: 4:15 PM PST

Seventy-four (74) patients were treated with a median age of 69 years (range 42-90) at 9 clinical sites, and received either 840 mg or 1,120 mg of oral rigosertib daily divided into two doses, in combination with a standard dose of injectable azacitidine. Of the 55 evaluable patients, 29 patients were treated with a daily dose of 1,120 mg of oral rigosertib, either 560 mg twice daily (12 patients) or 840 mg in the a.m. and 280 mg in the afternoon (17 patients). Twenty-six patients were treated with 560 mg in the AM and 280 mg in the PM (daily dose of 840 mg) for the first three weeks of a four-week cycle. All patients also received 75 mg/m2/day SC or IV azacitidine during the second week of the four-week cycle. The median duration of treatment for the HMA naïve and HMA failed patients was 7.8 and 4.9 months respectively. The median duration of response in these groups was 12.2 and 10.8 months, respectively.

The overall response rate (ORR) using the IWG 2006 criteria, in 29 HMA naïve patients, was 90%; including 10 patients (34%) with Complete Remission (CR). Among the 26 evaluable HMA-

failed patients the ORR was 54% including 8% CR or PR. The median time to initial and best response were 1 and 4 cycles in the HMA naïve group and 2 and 5 cycles in the HMA failed group.

The safety population (n = 74) received at least 1 dose of oral rigosertib. The combination was well tolerated. Other than genitourinary adverse events (AEs), the AE profile was similar to those described for azacitidine alone in this patient population. Genitourinary AEs, including hematuria (45% incidence of all grades, including 9% grade 3, and dysuria (38% all grades and 9% grade 3) were observed. A Safety Optimization Strategy was implemented for the higher dose cohort of 1,120 mg of oral rigosertib. These strategies included earlier in the day administration of the PM dose, oral hydration, monitoring of urinary pH and mandatory bladder emptying at night. Collectively these strategies resulted in mitigation of the target genitourinary AEs, including reduction of genitourinary grade 3 AEs reported from an earlier cohort despite receiving a higher dose of oral rigosertib.

In conclusion, oral rigosertib in combination with azacitidine was well tolerated in HMA naïve and HMA failed HR-MDS patients. The combination produced an encouraging rate of overall response and complete remission in both groups. The safety optimization strategies and increased dose exploration of oral rigosertib in the combination is leading to the development of a pivotal Phase 3 trial in HMA and chemotherapy naïve patients.

Drs. Lewis Silverman and Guillermo Garcia Manero, the lead investigators of the study at Mount Sinai Medical Center and MD Anderson Cancer Center, respectively, commented, "This multi-institutional collaborative study based on earlier laboratory research showing synergistic activity of rigosertib in combination with azacitidine led to a clinical trial of this combination in higher-risk MDS patients for both HMA naive and failed patients. The high overall response rate reported today is impressive, as is the durability and rate of achieving complete remission. We are excited about progressing these studies to a randomized pivotal placebo-controlled Phase 3 trial. The overall tolerability of the combination and convenience of administration of oral rigosertib could be key advantages for these future studies."

Dr. Steve Fruchtman, President of Onconova Therapeutics, Inc, sponsor of this study and developer of rigosertib commented, "We are most grateful to the patients, their families and our dedicated collaborating investigators for their participation in this study. The impressive results presented here have led to our plan for a pivotal trial for these patients ultimately hoping to improve upon their current therapeutic options. Based on End of Phase 2 Meetings with the Health Authorities, we have developed a randomized controlled pivotal trial. We expect to start the regulatory process for the approval of this trial plan very shortly. We are hopeful that both intravenous and oral formulations of rigosertib will be useful in serving the needs of higher risk MDS patients".

This oral presentation was delivered by Shyamala Navada, MD, Mount Sinai Medical Center on Saturday, December 1, 2018.

A copy of the presentation is available by visiting the Scientific Presentations section of Onconova’s website.

Onconova plans to meet with the FDA to discuss the results of the Phase 2 trial and the planned Phase 3 trial, and to seek a Special Protocol Assessment. The Company has partnered rigosertib with SymBio Pharmaceuticals, for Japan and Korea, and with Pint Pharma for Latin American countries. Both partners have indicated their interest in participating in the proposed new pivotal Phase 3 trial by enrolling patients in their respective territories. SymBio is currently conducting Phase 1 studies with oral rigosertib in Japan and also participating in the Phase 3 global INSPIRE trial. The Company is also actively seeking additional collaborations for rigosertib in other geographies.

On December 3, 2018 Takeda Pharmaceutical Company Limited (TSE: 4502) reported the presentation of results from the randomized Phase 3 study TOURMALINE-MM3 to evaluate the efficacy of oral monotherapy with NINLARO (Ixazomib) as a maintenance treatment in adult multiple myeloma patients previously responding to high-dose therapy (HDT) and autologous stem cell transplantation (ASCT) at the 60th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), Sunday, December 2, 2018 in San Diego, California (Press release, Takeda, DEC 3, 2018, View Source [SID1234531834]).

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!

The study reached its primary endpoint with NINLARO leading to a statistically significant improvement in progression-free survival (PFS) compared to placebo in adult patients with multiple myeloma who responded to HDT and ASCT, with assessment by an independent review panel (Independent Review Panel) Review Committee, IRC) (HR 0.72, p-value = 0.002). This equates to a 28 percent reduction in the risk of disease progression or death and a 39 percent improvement in NINLARO PFS compared to placebo. The safety profile of NINLARO maintenance therapy is in line with the already published results on the use of NINLARO as monotherapy.

"The evidence that multiple myeloma maintenance therapy may extend the duration of disease control is increasing," said Meletios Dimopoulos, MD, professor and chair of the Department of Clinical Therapeutics at the Faculty of Medicine, University of Athens, Athens, Greece. "Because there are currently limited treatment options and no proteasome inhibitor among them, there is a need for additional maintenance therapies that can prolong remission and have an acceptable safety profile. Data from the TOURMALINE-MM3 clinical trial suggest monotherapy with NINLARO as a potential oral treatment option for maintenance therapy with an ASCT proteasome inhibitor. "

"The positive results of this pivotal trial, which is the first and only placebo-controlled Phase 3 trial to test a proteasome inhibitor in this setting, support the use of NINLARO as a potential maintenance therapy in patients undergoing stem cell transplantation," said Jesús Gómez Navarro, MD, Vice President, Head of Oncology Clinical Research and Development, Takeda. "It is imperative that we continue to assist patients with the development of treatment options that support and deepen remission and delay disease progression. NINLARO-treated patients showed an improvement in progression-free survival compared to the control arm,

"Continuing research efforts are constantly evolving treatment options for multiple myeloma. This message is encouraging for anyone dealing with multiple myeloma, but further efforts are needed to bring us closer to our goal of meeting patients’ unmet medical needs, "said Brian Durie, MD, Chairman of the Board Board, International Myeloma Foundation. "This requires the development of additional safe and effective maintenance therapies."

Maintenance therapy with oral proteasome inhibitor (PI) Ixazomib causes significant progression-free survival (PFS) after autologous stem cell transplantation (ASC) in patients with newly diagnosed multiple myeloma (NDMM): the TOURMALINE-MM3 phase 3 study Sunday, 2. December 2018, 7:30 am – 9:00 am, Marriott Marquis San Diego Marina, Grand Ballroom 7

Among the main results, by Dr. med. Meletios Dimopoulos are presented include:

The study reached its primary endpoint with NINLARO leading to a statistically significant improvement in PFS compared to placebo in adult patients with multiple myeloma who responded to HDT and ASCT, assessed by an independent review panel (IRC) (HR 0.72, 95% CI: 0.582, 0.890, p-value = 0.002). This equates to a 28 percent reduction in the risk of disease progression or death and 39 percent improvement in PFS under NINLARO.
According to the IRC assessment, median PFS for patients in the NINLARO arm was 26.5 months compared to 21.3 months for the placebo arm.
The conversion of documented positive finding for minimal residual disease (MRD) at study to MRD negativity was more common in NINLARO-treated patients compared to placebo (12 percent versus 7 percent).
Maintenance therapy with NINLARO resulted in higher rates of deep remission compared to placebo (relative risk 1.41, 95 percent CI: 1.10, 1.80, p = 0.0042).
PFS benefits were broadly distributed across subgroups, including ISS III (HR 0.661), PI pretreated (HR 0.750), PI naive (HR 0.497), and patients with high-risk cytogenetic signs (HR 0.625).
The secondary endpoints, including median PFS2 and OS, have not been reached in either arm. The median follow-up time was 31 months.
Overall quality of life scores (EORTC QLQ-C30) were similar in NINLARO-treated patients compared to the placebo group.
The safety profile of NINLARO maintenance therapy is in line with the already published results on the use of NINLARO as monotherapy.
The discontinuation of treatment due to adverse events (AEs) was low and was 7 percent in the NINLARO arm compared to 5 percent in the placebo arm.
A third or higher grade of AEs occurred in 42 percent of patients in the NINLARO arm compared with 26 percent in the placebo arm.
In the NINLARO arm, severe AEs were found in 27 percent of the cases compared to 20 percent in the placebo arm.
Common AEs 3 or higher included infections (15 vs. 8 percent) in both the NINLARO and placebo arm, including pneumonia (6 vs. 4 percent), gastrointestinal disorders (6 vs. 1 percent ), Neutropenia (5 vs. 3 percent), and thrombocytopenia (5 vs. <1 percent).
In the NINLARO arm, peripheral neuropathy was observed in 19 percent of patients compared to 15 percent in the placebo arm. In the NINLARO arm, less than 1 percent of the incidence of peripheral neuropathy was Grade 3 AEs compared to 0 percent in the placebo arm.
The second tumor rate was 3 percent in both arms.
One patient in the NINLARO arm died during the study, whereas no death occurred in the placebo arm. The only death in the study was classified as a treatment-related event and was the result of pneumonia.
About the study TOURMALINE-MM3

TOURMALINE-MM3 is a randomized, placebo-controlled, double-blind Phase 3 study in 656 patients to determine the effect of NINLARO (Ixazomib) maintenance therapy on progression-free survival (PFS) versus placebo in patients with multiple myeloma who have a history of disease Remission (complete remission [CR], very good partial remission [VGPR] or partial remission [PR]) on induction therapy followed by high-dose therapy (HDT) and autologous stem cell transplantation (ASCT) The primary endpoint is progression-free survival (PFS) An important secondary endpoint includes overall survival (OS). For more information, seeView Source .

About NINLARO (ixazomib) capsules

NINLARO (Ixazomib) is an oral proteasome inhibitor that is also being studied in the multiple myeloma therapy continuum and in systemic light chain amyloidosis (AL), the first oral proteasome inhibitor tested in Phase 3 clinical trials and approved. NINLARO was approved by the US Food and Drug Administration (FDA) in November 2015 after a Priority Review and by the European Commission in November 2016. In the US and Europe, NINLARO is used in combination with lenalidomide and dexamethasone in the treatment of patients with multiple myeloma NINLARO has received marketing approval from regulators in more than 60 countries.

In 2011, Ixazomib was granted orphan drug status in multiple myeloma in the US and Europe, and AL amyloidosis in the US and Europe in 2012. In 2014, Ixazomib received breakthrough therapy from the FDA for relapsed or refractory systemic light chain amyloidosis (AL), a related extremely rare disease. The Japanese Ministry of Health, Labor and Social Affairs granted Ixazomib orphan drug status in 2016.

TOURMALINE, the comprehensive Ixazomib clinical development program, includes a total of six ongoing regulatory trials – five investigating all major groups of multiple myeloma patients and one dealing with light chain amyloidosis:

TOURMALINE-MM1 for testing ixazomib over placebo in combination with lenalidomide and dexamethasone in relapsed and / or refractory multiple myeloma
TOURMALINE-MM2 for the testing of ixazomib versus placebo in combination with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma
TOURMALINE-MM3 for testing ixazomib versus placebo as a maintenance treatment in patients with newly diagnosed multiple myeloma after induction therapy and autologous stem cell transplantation (ASCT)
TOURMALINE-MM4 for the testing of ixazomib versus placebo as a maintenance treatment in patients with newly diagnosed multiple myeloma who have not undergone autologous stem cell transplantation (ASCT)
TOURMALINE-AL1 for the testing of ixazomib plus dexamethasone for a physician on the basis of a selection of therapies in patients with relapsed or refractory AL amyloidosis. Patients are currently enrolled in this study.
TOURMALINE-MM5 for the testing of ixazomib plus dexamethasone versus pomalidomide plus dexamethasone in patients with relapsed and / or refractory multiple myeloma who have become resistant to lenalidomide. Patients are currently enrolled in this study.
For more information on actively recruiting Phase 3 studies, visit View Source

In addition to the TOURMALINE study program, Ixazomib is currently being evaluated in several therapeutic combinations in various patient populations worldwide through investigator-initiated studies (IIT).

NINLARO Ixazomib) Capsules: Important Safety Information Worldwide

SPECIAL WARNINGS AND PRECAUTIONS

Thrombocytopenia has been reported with the use of NINLARO (28% vs. 14% with the NINLARO or placebo regimen). Platelets reached their lowest point between the 14th and 21st day of the 28-day treatment cycle and recovered to baseline by the start of the next cycle. This did not lead to increased bleeding events or platelet transfusions. During treatment with NINLARO, the platelet count should be monitored at least monthly and more frequent monitoring should be considered during the first three cycles. Thrombocytopenia should be treated with dose adjustment and platelet transfusions according to the recommendations of the standard guidelines.

Gastrointestinal toxicities have been reported in the NINLARO and placebo regimens, for example, diarrhea (42% vs. 36%), constipation (34% vs. 25%), nausea (26% vs. 21%), and vomiting (22% vs 11%). These occasionally required the use of medication for vomiting and diarrhea, as well as supportive therapy.

Peripheral neuropathy has been reported in NINLARO (28% vs. 21% with the NINLARO or placebo regimen). The most commonly reported adverse reaction was peripheral sensory neuropathy (19% and 14% in the NINLARO or placebo regimen). Peripheral motor neuropathy has not been reported in any of the two regimens (<1%). Patients should be monitored for signs of peripheral neuropathy and dosing adjusted if necessary.

Peripheral edema has been reported in NINLARO (25% vs. 18% with the NINLARO or placebo regimen). The underlying causes should be clarified. If necessary, patients should receive supportive care. Dose adjustment should be made with dexamethasone according to the SPC or with NINLARO if severe symptoms occur.

Skin reactions occurred in 19 percent of patients with the NINLARO regimen compared to 11 percent of patients on the placebo regimen. The most common form of rash on both schemes was a maculopapular and macular rash. Rashes should be treated with supportive therapy, dose adjustment or discontinuation of the drug.

Hepatotoxicity, drug-induced liver damage, hepatocellular damage, fatty liver, and cholestatic hepatitis have not been reported commonly in NINLARO-treated patients. Liver values ​​should be monitored regularly and dose adjustments should be made for symptoms of Grade 3 and 4.

Pregnancy – NINLARO can lead to harm to unborn life. For fertile men and women of childbearing potential, contraceptive methods should be used during treatment and for a further 90 days after the last dose of NINLARO. Due to the possible risk to the unborn child, women of childbearing age should be prevented from taking pregnancy while being treated with NINLARO. Women who use hormonal contraceptives should also use a barrier method for contraception.

Breastfeeding – It is not known whether NINLARO or its breakdown products are excreted in breast milk. Due to possible adverse events in breast-fed infants, NINLARO-treated patients should abstain.

SPECIAL PATIENT POPULATIONS

Hepatic impairment: The starting dose of NINLARO should be reduced to 3 mg in patients with moderate or severe hepatic impairment.

Renal impairment: In patients with severe renal impairment or dialysis-dependent patients with end stage renal disease (ESRD), the starting dose of NINLARO should be reduced to 3 mg. NINLARO is not dialyzable and can therefore be administered independently of the dialysis schedule.

INTERACTIONS WITH OTHER MEDICINAL PRODUCTS

Simultaneous use of NINLARO and strong CYP3A inducers is foreseeable.

SIDE EFFECTS

The most common adverse reactions reported in at least 20% of NINLARO regimens or more frequently than placebo were diarrhea (42% vs. 36%), constipation (34% vs. 25%), thrombocytopenia (28% vs. 14%), peripheral neuropathy (28% vs. 21%), nausea (26% vs. 21%), peripheral edema (25% vs. 18%), vomiting (22% vs. 11%) and back pain (21% vs. 16%). Serious side effects that occurred in at least 2 percent of patients included thrombocytopenia (2%) and diarrhea (2%). For each adverse reaction, one or more of the three drugs was discontinued in ≤ 1 percent of patients on NINLARO regimen.

On December 3, 2018 Tiziana Life Sciences plc (Nasdaq: TLSA / AIM: TILS), a biotechnology company focusing on the discovery and development of innovative therapeutics for inflammation and oncology indications, reported that patient enrollment in the ongoing Phase 2a clinical trial (CDKO-125A-010) is completed (Press release, Tiziana Life Sciences, DEC 3, 2018, View Source [SID1234531836]). This is a single-arm, repeated-dose, 6-month duration study to evaluate safety, tolerability and anti-tumor activity of Milciclib in Sorafenib-refractory or -intolerant patients with unresectable or metastatic HCC. Topline data from this multi-center trial, being conducted in Italy, Greece and Israel, will be available in the second quarter of 2019.

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!

Previously we reported interim analysis data from the first 10 patients, following 6 months of treatment, showing that Milciclib (100 mg once daily; 4 days on/3 days off every 4 weeks defining each cycle) was well-tolerated in this HCC patient population. It was concluded by an Independent Data Monitoring Committee (IDMC) that there were no major signals of tolerability concerns, and the IDMC allowed continuation of patient enrollment in the trial. Following completion of 6 months of treatment, three patients opted to continue treatment under the compassionate use program. Notably, one patient is still continuing treatment in the 14th month and the other two patients received treatment until 9th month and 13th month, respectively.

About HCC

HCC is the 5th most common cancer and the 3rd cause of cancer mortality worldwide. In 2007 the approval by the European Medical Agency (EMA) and Food and Drug Administration (FDA) of Sorafenib (Nexavar), an inhibitor of several receptor tyrosine kinases, in HCC represented the first systemic therapy for improving outcome in patients unsuitable for loco-regional and surgical therapies and created a new standard of treatment for the disease. However, although significant in respect to placebo, the benefits of Sorafenib are modest; the response rate is less than 3%, the improvement in median survival is 2-3 months and the drug-related symptoms are not ordinary. The complex multi-factorial etiology of HCC warrants a need for systemic therapies that target different signaling cascades to provide improved efficacy and safety for both naive patients presenting with unresectable, advanced stage and those who suffer recurrence after curative treatments (resection, ablation and transplantation).

About Milciclib

Milciclib (PHA-848125AC) is a small molecule inhibitor of several cyclin dependent kinases such as CDK1, CDK4, CDK5 and CDK7. CDKs are serine threonine kinases that play crucial roles in progression of the cell cycle from G1 to S phase. Overexpression of CDKs and other downstream signaling pathways that regulate cell cycles have been frequently associated with development of resistance towards chemotherapies. In a Phase 1 study, oral treatment with Milciclib was well-tolerated and the drug showed promising clinical responses in patients with advanced solid malignancies such as in NSCLC, pancreatic and colon cancer, thymic carcinoma and thymoma. Additionally, milciclib met its primary endpoint in two separate Phase 2 multi-center clinical trials (CDKO-125A-006: 72 patients and CDKO-125A-007: 30 patients) in thymic carcinoma and thymoma patients.

About Sorafenib

Sorafenib (co-developed and co-marketed by Bayer and Onyx Pharmaceuticals as Nexavar) is a small molecular multi-tyrosine kinase inhibitor drug approved for the treatment of primary kidney cancer (advanced renal cell carcinoma), HCC, and radioactive iodine resistant advanced thyroid carcinoma. Treatment with Sorafenib induces autophagy, which may suppress tumor growth. However, autophagy can also cause drug resistance.

On December 3, 2018 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on using the body’s immune system to fight cancer, presented the latest data from its ongoing study focusing on early detection of breast cancer, utilizing Cchek, its artificial intelligence (AI) driven cancer detection technology, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy (Press release, Anixa Biosciences, DEC 3, 2018, View Source [SID1234531852]). The conference is designed to integrate multidisciplinary facets of basic cancer immunology and immunotherapy to broaden the understanding of ways to harness the immune system to address cancer. The conference was held November 27–30, 2018 in Miami Beach, Florida.

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!

Anixa presented data demonstrating the ability of Cchek to identify the presence of early stage breast cancer in a subject by using its AI technology to analyze a simple blood draw. The Cchek technology demonstrated a sensitivity of 89.3% when detecting early stage breast cancer (Stage I or II) and a specificity (the ability to correctly identify healthy subjects) of 94.7% when used to test blinded samples. Furthermore, Cchek was also able to detect the early stages of breast cancer (Stage 0) in subjects with biopsy-confirmed ductal carcinoma in situ (DCIS), a type of pre-cancerous/non-invasive breast lesion that often leads to invasive breast cancer, with 72% sensitivity. The presentation is available on Anixa’s website, or it may be requested by sending an email to AACR (Free AACR Whitepaper)[email protected] and including your name, title, and contact information.

"We are pleased to have made our presentation titled, Combining the immunophenotyping of MDSCs and lymphocytes with artificial intelligence (AI) to predict early stage breast cancer, at the AACR (Free AACR Whitepaper) Tumor Immunology and Immunotherapy conference. This data was focused on using our Cchek technology to detect breast cancer in its early stages, primarily stage I and II when the cancer is more easily treated. The majority of screening technologies currently used for breast cancer detection, such as mammography, have the ability to detect later stage breast malignancies rather successfully but have shown difficulty with earlier stages. A major challenge with mammography is the large number of false positives resulting in overtreatment and many unnecessary biopsies," stated Dr. Amit Kumar, President and CEO of Anixa Biosciences. "As our study continues, we hope to enable a better approach for identifying breast cancer as early as possible including even when a non-invasive breast cancer lesion is present, such as in the case of DCIS. We have previously announced that our initial commercial focus is on a prostate cancer test for which we will be meeting with the USFDA on December 17, 2018. We recently presented our latest prostate cancer data at the 33rd Annual Meeting of The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)," added Dr. Kumar.

American Association of Cancer Research (AACR) (Free AACR Whitepaper)
The American Association of Cancer Research (AACR) (Free AACR Whitepaper) (www.aacr.org) is a 501(c)(3) public charity headquartered in Philadelphia, PA. The mission of the AACR (Free AACR Whitepaper) is to prevent and cure cancer through research, education, communication and collaboration. Through its programs and services, the AACR (Free AACR Whitepaper) fosters cancer research and related biomedical science; accelerates the dissemination of new research findings among scientists and others dedicated to the conquest of cancer; promotes science education and training; and advances the understanding of cancer causes, prevention, diagnosis and treatment throughout the world.