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.

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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 2, 2018 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported the presentation of results from the Company’s Phase 1b/2 clinical trial of its non-covalent BTK inhibitor vecabrutinib in adults with relapsed/refractory chronic lymphocytic leukemia (CLL) and other B-cell malignancies (Press release, Sunesis, DEC 3, 2018, View Source [SID1234531815]). The results will be presented today, December 2, from 6:00-8:00 p.m. PT in a poster session titled "CLL: Therapy, excluding Transplantation: Poster II" at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, California. The poster, titled "Preliminary Safety, Pharmacokinetic, and Pharmacodynamic Results from a Phase 1b/2 Dose-Escalation and Cohort-Expansion Study of the Noncovalent, Reversible Bruton’s Tyrosine Kinase Inhibitor, Vecabrutinib, in B-Lymphoid Malignancy Patients with Prior BTKi Therapy," Abstract No. 3141, is available at www.sunesis.com.

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"To date, vecabrutinib has demonstrated both an encouraging safety profile and evidence of pharmacodynamic activity in CLL and other B cell cancer patients both with and without the BTK C481 mutation," said Dayton Misfeldt, Sunesis interim Chief Executive Officer. "We believe vecabrutinib has significant potential to be an important new treatment for ibrutinib-resistant B-cell malignancy patients, and its additional activity as an ITK inhibitor suggests further directions for clinical investigation. We look forward to continuing the dose escalation, as we believe that the target dose level is likely to be between 100 mg and 300 mg BID. We are excited to be working with such thoughtful and diligent investigators at eight premier sites across the U.S., and we thank our investigators for their continued support."

Data reported today were available from 11 of 13 treated patients. These included 7 with relapsed/refractory CLL, two with mantle cell lymphoma (MCL), and two with Waldenstrom macroglobulinemia (WM). Patients had received an average of 5 lines of prior therapy, and all had progressed on prior covalent BTK inhibitor treatment. Four of the 7 CLL patients had BTK C481 mutations. Currently, 4 patients are on study: one in Cycle 2, one in Cycle 3, and two new subjects who are in Cycle 1 and are anticipated to complete the 50 mg cohort.

The poster builds vecabrutinib’s profile in three key areas:

Safety: data on treatment-emergent adverse events (TEAEs) were available for 10 patients. The most common TEAEs of any grade were anemia (70%) and neutropenia and night sweats (50% each). Grade 3 drug-related AEs were anemia, neutropenia,

leukocytosis, and ALT increase (10% each). In the second cohort, one patient experienced a dose-limiting toxicity of an inadequate number of Cycle 1 doses administered due to a drug-related grade 3 ALT elevation, resulting in expansion of the cohort to 6 patients.

Pharmacokinetics: the pharmacokinetic profile of the 50mg dose is approximately dose proportional to the 25 mg dose. The next dose levels are expected to produce plasma concentrations associated with consistently high inhibition of BTK.

Pharmacodynamics: vecabrutinib inhibition of BTK phosphorylation was rapid and sustained in the 5 patients who had adequate baseline signal for analysis. Decreases in serum concentrations of key cytokines associated with B-cell malignancies, CCL2, CCL3, and CCL4, were also observed in 7 patients, consistent with inhibition of BTK signaling.

Webcast Information

The data will be further discussed as part of an analyst and investor event being held in San Diego today, December 2, at 8:00 p.m. PT, with the slide webcast commencing at 8:30 p.m. PT. The event is intended for institutional investors and sell-side analysts only. Please contact [email protected] for more information. The live webcast of the event, with slides, will be available to all on the Investors section of the Sunesis website at www.sunesis.com and will be archived for 90 days.

About Vecabrutinib

Vecabrutinib (SNS-062) is a selective, oral, reversible, non-covalent inhibitor of Bruton’s tyrosine kinase (BTK). BTK is a validated target for the treatment of B-cell malignancies driven by B-cell receptor signaling. Vecabrutinib retains its activity in the presence of a BTK C481S mutation, the most common mutation seen in ibrutinib-resistant CLL patients. In preclinical studies, vecabrutinib demonstrated potent activity in both wild-type and C481S-mutant BTK. Vecabrutinib has also been shown to inhibit a select number of other kinases including IL2-inducible T-cell kinase (ITK), which may improve T cell function. In a Phase 1a randomized, double-blind, placebo-controlled single ascending dose study in healthy volunteers, vecabrutinib demonstrated improved pharmacokinetics over ibrutinib, and sustained inhibition of BTK. Vecabrutinib is now being investigated in a Phase 1b/2 study in patients with relapsed CLL and other B-cell malignancies.

On December 3, 2018 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported the presentation of preclinical data from the Company’s Menin-Mixed Lineage Leukemia (MLL) inhibitor program at the 60thAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, California (Press release, Syndax, DEC 3, 2018, View Source [SID1234531831]).

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"Acute leukemias characterized by MLL-rearrangements (MLL-r) and nucleophosmin (NPM1) mutations represent areas of high unmet medical need, with 5-year survival rates falling below 50%," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Data presented on VTP-50469, a Syndax Menin-MLL inhibitor, provides further support for developing this class of molecules for specific, genetically-defined acute leukemias. The robustness and consistency of the accumulated preclinical data seen with our Menin-MLL inhibitors provide support for an anticipated IND filing on our lead compound, SNDX-5613, in the second quarter of 2019."

In an oral presentation at ASH (Free ASH Whitepaper), Hannah Uckelmann, Ph.D., Dana-Farber Cancer Institute, presented new preclinical data demonstrating that NPM1 mutant progenitor cells can act as drivers of leukemic transformation, and that VTP-50469 can block their pathological capacity by disrupting the menin-MLL interaction. In addition, data generated using a mouse PDX model of NPM1 mutant acute myeloid leukemia (AML) demonstrated that single agent treatment with VTP-50469 eradicated pre-leukemic NPM1 mutant cells and prevented leukemia development, resulting in a marked survival benefit. These data lend further support to the potential therapeutic utility of menin inhibitors in the setting of NPM1 mutant AML.

Furthermore, during a Scientific Spotlight Session at ASH (Free ASH Whitepaper), Scott Armstrong, M.D., Ph.D., Dana-Farber Cancer Institute, provided an overview of the multiple complexes that influence gene expression in MLL-r leukemias and NPM1 AML. Additional preclinical data generated in his laboratory further substantiate that inhibition of the Menin-MLL interaction can decrease leukemic burden and prolong survival in mouse PDX models of both MLL-r and NPM1 mutant leukemias. These data build on prior encouraging preclinical results presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April, and further underscore the potential therapeutic utility of menin inhibitors as modulators of critical epigenetic mechanisms in acute human leukemias, including subsets of both acute lymphoblastic leukemia (ALL) and AML.

Oral Presentation:

Title: MLL-Menin Inhibition Reverses Pre-Leukemic Progenitor Self-Renewal Induced By NPM1 Mutations and Prevents AML Development
Presenter: Hannah Uckelmann, Ph.D., Research Fellow at the Dana-Farber Cancer Institute
Publication Number: 546

Scientific Spotlight Session:

Title: Targeting Chromatin Complexes in MLL Rearranged Leukemia
Presenter: Scott Armstrong, M.D., Ph.D., Chairman of the Department of Pediatric Oncology at the Dana-Farber Cancer Institute, Associate Chief of the Division Hematology/Oncology at Boston Children’s Hospital, and the David G. Nathan Professor of Pediatrics at Harvard Medical School

Both presentations are available in the Publications section of the Company’s website, www.syndax.com.

About MLL Rearranged Leukemias

Rearrangements of the MLL gene give rise to an acute leukemia, MLL-r. MLL-r occurs in ~80% of infant acute leukemias and up to 10% of adult acute leukemias. It is associated with a poor prognosis, with less than 50% of infants with MLL-r surviving past 5 years. MLL rearrangements produce fusion proteins that require interaction with a protein called Menin in order to drive leukemic cancer growth. Disruption of the Menin-MLL-r interaction has been shown to halt the growth of MLL-r leukemic cells. MLL-r leukemias are routinely diagnosed through currently available cytogenetic screening techniques in leukemic cells, but there are currently no approved therapies indicated for MLL-r leukemias.

About NPM1c Acute Myeloid Leukemia

NPM1c represents another discrete form of acute myeloid leukemia (AML) distinguished by point mutations in the NPM1 gene that drives the leukemic phenotype. NPM1c is the most common type of cytogenetically normal AML and represents ~30% of all diagnosed AML. This subtype of AML has a poor prognosis, with a 5-year overall survival rate of ~50%. Similar to MLL-r leukemias, NPM1c AML is highly dependent on the expression of specific developmental genes, shown to be negatively impacted by inhibitors of the menin-MLL1 interaction. NPM1c AML is routinely diagnosed through currently available screening techniques in leukemic cells, but there are currently no approved therapies indicated for NPM1c AML.

On December 3, 2018 Celgene Corporation (NASDAQ:CELG) reported initial safety data from its ongoing proof-of-concept trial of JCARH125 in patients with relapsed/refractory multiple myeloma. JCARH125 is an investigational BCMA-targeting CAR T cell therapy being developed by Juno Therapeutics, A Celgene Company (Press release, Celgene, DEC 3, 2018, View Source [SID1234531848]). Results were presented by Sham Mailankody, MBBS, in an oral presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, CA (Abstract #957).

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The data reported from the multicenter, phase 1/2 EVOLVE trial includes patients who have been treated with JCARH125 in the dose escalation study. The primary objectives of the phase 1 portion of the trial are safety and identification of a recommended phase 2 dose. The patients enrolled in the study had to have received at least three prior lines of multiple myeloma therapy, including an autologous stem cell transplant for transplant eligible patients, a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Dose escalation is currently ongoing.

"We believe that cellular therapies targeting BCMA will play an important role in the future treatment of patients with multiple myeloma," said Mark Gilbert, M.D., Chief Medical Officer for Juno Therapeutics, A Celgene Company. "These data from 44 patients in the EVOLVE trial further support our commitment to innovation in multiple myeloma clinical research."

At data cut off, 44 patients have been infused with JCARH125 in three dose escalation cohorts. These patients were heavily pretreated, with a median of seven prior lines of therapies (range, 3-23), and 77% had high-risk cytogenetics. Seventy-one percent of patients experienced grade 1 and 2 cytokine release syndrome (CRS) with 9% of patients experiencing grade 3/4 CRS. In addition, 18% of patients experienced grade 1 and 2 neurological events with 7% of patients experiencing a grade 3/4 event. Other frequent grade 3/4 AEs included neutropenia (86%), anemia (50%), thrombocytopenia (43%) and infection (14%).

In this first report of JCARH125 data, the median follow up was only 11 weeks, yet among infused patients, the overall response rate (ORR) was 82%. At the lowest dose level of 50×106 CAR T cells, the ORR was 79% and 43% of patients achieved stringent complete response (sCR) or complete response (CR).

JCARH125 is investigational and has not been approved in any country

On December 3, 2018 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that Professor Paul G. Richardson presented updated interim data with melflufen (Ygalo) from the ongoing HORIZON trial at the 60th ASH (Free ASH Whitepaper) meeting in San Diego, California, USA (Press release, Oncopeptides, DEC 3, 2018, View Source [SID1234531865]).

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Overall conclusions

The updated phase II data with melflufen in late-stage relapsed-refractory multiple myeloma patients refractory to pomalidomide and/or daratumumab after failing on immunomodulatory drugs (IMiDs) and proteasome inhibitors (PIs) shows:

An Overall Response Rate (ORR) of 33%, in patients where 46% had received 3+ regimens of treatments over the last 12 months
A vast majority of patients, 84%, achieved disease stabilization (SD or better)
The first evaluation of Progression Free Survival (PFS) shows a median of 4.0 months in the ongoing trial with a median of 6.4 months for responding patients
Activity was observed regardless of underlying refractory status and the traditional prognostic factor albumin was a strong predictor of ORR
The treatment was well tolerated with mainly reversible and manageable hematological adverse events. The incidence of non-hematological toxicity was low
Professor Paul G. Richardson comments

"Over the last decade, initial treatments in myeloma have been radically improved, and in particular with the advent of the widespread adoption of novel agent combination approaches as well as continuous therapy. However, the number of patients who become resistant to immunomodulatory agents, proteasome inhibitors and anti-CD38 monoclonal antibodies is increasing, and there is thus a clear need for effective treatment options with new mechanisms of action. Melflufen is a first in class peptidase-enhanced compound that does not share resistance pathways seen with currently used treatments and has general manageable toxicity with favorable tolerability. Melflufen is showing considerable promise in the relapsed and refractory setting, with clinical development ongoing" said Paul Richardson MD, the RJ Corman Professor of Medicine at Harvard Medical School and Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute, Boston, USA.

About the HORIZON study

Patient recruitment to the study is ongoing. The interim data presented at the ASH (Free ASH Whitepaper) meeting is based on a data cut-off dated October 22ed 2018 with 83 patients treated and 82 patients included in the response analysis. The patients in the study are refractory to pomalidomide and/or daratumumab after failing on IMiDs and PIs.

Summary of the HORIZON interim data

The study continues to develop positively in this heavily pretreated patient group that is refractory to pomalidomide and/or daratumumab after failing on IMiDs and PIs with few remaining treatment options.

61% of patients in the study had high-risk cytogenetics, 36% of patients were ISS stage III, the median number of prior lines of therapy was 5 and the median time since initial diagnosis was 6.5 years.
All patients in the study were investigator assessed as non-responsive or non-tolerant to IMiDs and PIs, 100% of patients were also refractory to pomalidomide or daratumumab, 60% were refractory to IMiDs, PIs and anti-CD38, 55% were alkylator refractory and 93% had disease progression on or within 60 days of completion of the last therapy.
Analysis of the preliminary efficacy results showed an ORR of 32,9% and that 84,1% of the patients achieved disease stabilization (SD or better).
Overall response rate (N=82)
ORR sCR VGPR PR MR SD PD NE
total, N=82 32,9% 1,2% 11% 20,7% 6,1% 45,1% 14,6% 1,2%
Subgroup analysis suggests that response does not vary across refractory subsets but rather with the underlying disease and health status of the patient (confirming the observation made in Oncopeptides phase II study O-12-M1).
This study confirms earlier results from the O-12-M1 study in a more resistant patient population. The efficacy results in this interim analysis are encouraging with an ORR of 32,9%.

Melflufen showed a manageable safety and tolerability profile. Treatment-related grade 3/4 AEs were reported in 62 (75%) patients with the majority being hematological. Treatment-related non-hematological grade 3/4 AEs were rare with infections in only 7% of patients. 13% of the patients discontinued treatment due to AEs.

About melflufen

Melflufen (Ygalo), a peptide conjugated alkylator belonging to a novel class of peptidase-enhanced compounds, targets multiple myeloma (MM) cells with a unique mechanism of action. Aminopeptidases are enzymes found in all cells but are over-expressed in several cancers including MM. Melflufen selectively targets MM cells through aminopeptidase-driven accumulation. In vitro experiments show a 50-fold enrichment of the active substance in MM cells compared with administration of equal amount of an alkylator not enriched by aminopeptidases. The enrichment results in selective cytotoxicity (increased on-target potency and decreased off-target toxicity), and that resistance pathways of existing myeloma treatments (including alkylators) is overcome. Melflufen also demonstrates strong anti-angiogenic properties.

Melflufen in clinical development

Melflufen (Ygalo) has been used to treat late-stage RRMM patients in both phase I and phase II clinical studies (O-12-M1) with favorable results. Currently, melflufen is being studied in four clinical trials for the treatment of multiple myeloma. The current studies are OCEAN, HORIZON, ANCHOR and BRIDGE.

The current clinical study program is intended to demonstrate better results from treatment with melflufen compared to established alternative drugs for patients with late-stage multiple myeloma. Melflufen could potentially provide physicians with a new treatment option for patients suffering from this serious disease.

Melflufen has been investigated in the treatment of late-stage relapsed refractory multiple myeloma (RRMM) patients. This was done in the clinical study O-12-M1 where strong final results were reported in December 2017. Currently, four clinical studies are ongoing with melflufen.

OCEAN is Oncopeptides´ pivotal Phase III study where melflufen is compared directly with current standard of care, pomalidomide, in late-stage RRMM patients.

HORIZON is a Phase II study that studies the effect of melflufen in late-stage RRMM patients with few or no remaining established treatment options. Updated interim data from this study were presented at ASH (Free ASH Whitepaper) in December 2018.

ANCHOR is a phase I/II study where melflufen is administered in combination with either bortezomib or daratumumab in RRMM patients. The results of this study aim to create understanding and knowledge among treating physicians for how melflufen can be used in combination with these drugs. In addition, the results could open up for the use of melflufen in earlier lines of treatment. The first interim data from the study was presented in a poster presentation on December 1, 2018 at ASH (Free ASH Whitepaper).

BRIDGE is a phase II study, where melflufen is used in RRMM patients with impaired renal function. This is a positioning study to show melflufen’s treatment profile in these patients.