On March 28, 2019 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is generating red blood cells and bioengineering them into an entirely new class of cellular medicines, reported fourth quarter and full-year 2018 financial results (Press release, Rubius Therapeutics, MAR 28, 2019, View Source [SID1234534703]).

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"2018 was a transformative year for Rubius as we transitioned from a private, preclinical company into a public company on the brink of entering the clinic with our first program, RTX-134, for the treatment of patients with phenylketonuria," said Pablo J. Cagnoni, M.D., chief executive officer. "With our first Investigational New Drug application for RTX-134 now cleared by the U.S. Food and Drug Administration, we plan to begin treating patients in our Phase 1b trial during the second quarter with initial clinical data expected during the second half of the year. We will also be presenting preclinical data at the AACR (Free AACR Whitepaper) 2019 Annual Meeting, supporting our growing oncology pipeline. We stand well capitalized and well positioned to achieve our goal of filing four to five IND’s by the end of 2020, as we work to advance this new class of cellular medicines on behalf of patients."

Recent Business Highlights and Anticipated Upcoming Milestones

· In March 2019, Rubius announced that the U.S. FDA cleared the Company’s Investigational New Drug (IND) application for RTX-134, an allogeneic, off-the-shelf cellular therapy for the potential treatment of patients with phenylketonuria (PKU), an inherited metabolic disorder that is characterized by the body’s inability to metabolize the essential dietary amino acid, phenylalanine.

· Rubius expects to begin treating patients in a Phase 1b clinical trial in the second quarter of 2019 and plans to report initial clinical data from the trial during the second half of 2019.

·The primary objectives of the Phase 1b study are to evaluate preliminary safety, longevity of the RTX-134 cells in circulation, to obtain proof-of-mechanism as measured by production of trans-cinnamic acid (the byproduct of PAL) and select a preliminary dose and schedule.

·At this year’s American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, being held March 29-April 3, Rubius will present preclinical data from its oncology pipeline, including its lead oncology candidates RTX-240 (formerly RTX-212) and RTX-224 for the

treatment of solid tumors and RTX-aAPC (HPV+), an artificial antigen presenting cell, for the treatment of HPV+ tumors, including head and neck cancers.

·Rubius strengthened its leadership by appointing Natalie Holles to its board of directors and Greg Whitehead as senior vice president and chief quality officer.

·The Company plans to continue investing in its pipeline and expects to file its first oncology IND for RTX-240 for the treatment of solid tumors by early 2020, with two to three additional IND filings following in 2020.

·Rubius intends to continue renovating and hiring key personnel for its Smithfield, RI manufacturing facility with the goal of being operational in 1000L bioreactors by the end of 2020.

Successful Execution on Key Priorities in 2018

·Purchased 135,000 sq. ft. manufacturing facility in Smithfield, RI and initiated renovations

·Scaled manufacturing of RCTs from 50L to 200L bioreactors

· Transferred RTX-134 manufacturing process to contract manufacturing organization

·Executed three successful financings, including a $101.0 million crossover round, a $254.3 million initial public offering after expenses, and a recent debt financing of up to $75.0 million, which further extends cash runway into 2021

·Increased RCT storage stability from 28 days to 42 days with additional studies ongoing to explore extending RCT storage stability even further

·Continued to strengthen the board of directors, build a leading scientific team and attract experienced leadership to deliver against Rubius’ objectives

·Strengthened internal capabilities in discovery, platform development, technical operations and manufacturing

Fourth Quarter Financial Results

Net loss for the fourth quarter of 2018 was $27.2 million or $0.35 per common share, compared to $17.5 million or $2.07 per common share in the fourth quarter of 2017.

In the fourth quarter of 2018, Rubius invested $16.5 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $6.6 million in the fourth quarter of 2017. The year-over-year increase was due to an additional $2.4 million of costs incurred in preparation for the Phase 1b clinical trial for RTX-134, and $6.7 million was associated with personnel costs, stock-based compensation, facility and laboratory costs driven by increases in R&D headcount and expanded research activities to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $12.6 million during the fourth quarter of 2018, as compared to $10.9 million for the fourth quarter of 2017. The higher costs were primarily driven by increases in

professional fees and infrastructure costs to support the Company’s growth and to operate as a public company.

Full Year Financial Results

Net loss for the full year 2018 was $89.2 million or $2.27 per common share, compared to $44.5 million or $5.55 per common share for the full year 2017.

For the full year 2018, Rubius invested $51.8 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $21.2 million for the full year 2017. The year-over-year increase was primarily driven by an increase of $8.3 million of costs incurred in preparation for the Phase 1b clinical trial for RTX-134, and $19.1 million in personnel costs, stock-based compensation, facility and laboratory costs driven by increases in R&D headcount and expanded research activities to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $39.9 million during the twelve months of 2018, as compared to $22.0 million for the same period in 2017. The higher costs were primarily driven by an $7.6 million increase in stock-based compensation and $10.3 million increase in personnel costs, facility and professional fees to support the Company’s growth and to operate as a public company.

Cash Flow Highlights

As of December 31, 2018, cash, cash equivalents and investments were $404.1 million as compared to $104.3 million as of December 31, 2017, providing Rubius with a cash runway into 2021. The year-end cash balance reflects $101.0 million of net proceeds received from its Series C preferred stock financing during the first quarter of 2018, $254.3 million of net proceeds from the Company’s initial public offering during the third quarter of 2018, and, in the fourth quarter, $25.0 million from a debt financing. During the fourth quarter, the Company used $19.7 million of cash in operations and used $3.3 million for capital purchases. During the full year, the Company used $58.3 million of cash in operations and $15.0 million for capital purchases, including $8.0 million to acquire the manufacturing facility in Smithfield, Rhode Island.

On March 28, 2019 Imbrium Therapeutics L.P., a clinical-stage biopharmaceutical company and operating subsidiary of Purdue Pharma L.P., in conjunction with Mundipharma EDO GmbH, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its investigational drug tinostamustine, a potentially first-in-class alkylating deacetylase inhibiting molecule being studied in early phase clinical trials, for the treatment of T-cell prolymphocytic leukemia (T-PLL) (Press release, Imbrium Therapeutics, MAR 28, 2019, View Source [SID1234534719]).

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T-PLL is an extremely rare and aggressive T-cell leukemia that is characterized by out of control growth of mature T-cells. There are very limited effective treatment options for T-PLL. The disease typically progresses rapidly and does not respond well to standard multi-agent chemotherapy.

"This orphan drug designation represents an important step not just for Imbrium and the development of tinostamustine, but also for the patients suffering from T-PLL who do not currently have sufficient treatment options," said Richard Fanelli, PhD, head of Regulatory Affairs, Imbrium Therapeutics.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation is intended to facilitate drug development for rare diseases and may provide certain incentives to drug developers.1,2 T-PLL is an extremely rare and typically aggressive blood cancer. It is so rare that healthcare professionals may only see one case of T-PLL every five to 10 years.3 Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes with a median survival of around one year. 3,4 There is no guarantee that tinostamustine, an investigational agent, will successfully complete clinical development or gain FDA approval.

Craig Landau, MD, president and CEO, Purdue Pharma L.P. added, "This marks the second orphan drug designation we have received from the U.S. FDA in just the last two months and demonstrates our commitment to rapidly advancing our pipeline of oncology chemotherapeutics for rare and difficult to treat cancers."

In addition to T-PLL, Imbrium Therapeutics has initiated the early phase clinical development of tinostamustine in a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

About T-cell prolymphocytic leukemia
T-cell prolymphocytic leukemia (T-PLL) affects approximately 2 percent of all patients with mature lymphocytic leukemias.5 It is characterized by the out of control growth of mature T-cells (T-lymphocytes). T-cells are a type of white blood cell that protects the body from infections.6 The majority of patients present with hepatosplenomegaly and generalized lymphadenopathy, with skin infiltration, anemia and thrombocytopenia often seen.5 T-PLL affects older adults with a median age at diagnosis of 61 years, and it is more common in men than in women.6

T-PLL typically has rapid progression and does not respond well to standard multi-agent chemotherapy.

About tinostamustine
Tinostamustine (EDO-S101), is a novel multi-action therapy in Phase 2 clinical development for a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

Preclinical studies have shown that tinostamustine has the potential to improve access to the DNA strands within cancer cells, break them, and counteract damage repair.7,8,9,10 The preclinical data also suggest that these complementary and simultaneous modes of action have the potential to overcome resistance toward some other cancer treatments.7,8,9,10

Tinostamustine is currently being studied in multiple myeloma, Hodgkin lymphoma, peripheral T-cell lymphoma, cutaneous T-cell lymphoma, T-cell prolymphocytic leukemia, soft tissue sarcoma, small cell lung cancer, triple-negative breast cancer, ovarian cancer, endometrial cancer and MGMT-unmethylated glioblastoma.

On March 28, 2019 Queen’s University Belfast and Domainex are reported that their joint project team has successfully identified novel first-in-class small molecule inhibitors of the anti-apoptotic protein FLIP (Press release, Queen’s University Belfast, MAR 28, 2019, View Source [SID1234554005]). The team has generated data suggesting multiple therapeutic opportunities for a FLIP inhibitor in both the single agent and combination settings and is now seeking a commercial partner for further development of the novel inhibitors.

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The team is presenting the FLIP inhibitor programme at the AACR (Free AACR Whitepaper) annual meeting in Atlanta on 31 March 2019 (New Molecular Targets poster session, poster 382 /14). The poster showcases the on-target effects of the inhibitors, their drug-like properties, their potency against B Cell Lymphomas, triple negative breast cancer and KRAS and EGFR mutant non-small cell lung cancer, and their potential for combination with chemotherapy, immune oncology agents and EGFR-targeted therapeutics.

The next phase of the programme will comprise selection of a pre-clinical development candidate and completion of pre-clinical activities. Profs Dan Longley (Biology lead) and Tim Harrison (Med Chem lead) are attending the AACR (Free AACR Whitepaper) meeting and will be actively engaging in discussions with potential partners at the meeting.

This FLIP programme has been funded by the Wellcome Trust and has focussed on the identification and optimisation of novel first-in-class small molecule FLIP-FADD protein-protein interaction inhibitors. FLIP is a non-redundant inhibitor of Caspase 8 and functional FLIP allows tumour cells to evade cell death and promotes tumour growth and therapy resistance. The novel FLIP inhibitors activate Caspase 8 and have shown efficacy in a number of pre-clinical models including clinically challenging KRAS and EGFR mutant non-small cell lung cancer.

To arrange to meet the team at AACR (Free AACR Whitepaper) please contact Enda Gribbon: ([email protected])

Trevor Perrior, Chief Executive Officer of Domainex, commented: "Our medicinal chemists have been delighted to work on this exciting target and enable Prof. Dan Longley and his team to identify and optimise the FLIP inhibitors. There were several challenges that we had to solve in order to identify hits and develop a series of potential drug compounds that are potent and selective. We look forward to at least one of these compounds progressing towards the clinic for the benefit of patients. The funding secured from the Wellcome Trust is a clear endorsement of the strength of the integrated drug discovery platform of Domainex to deliver molecules with disease-modifying potential and we remain deeply committed to supporting academic translational research."

Prof. Dan Longley from the Centre for Cancer Research and Cell Biology (CCRCB) at Queen’s University Belfast, added: "Resistance to cancer therapeutics such as chemotherapy is a major clinical problem that limits the effectiveness of many current cancer treatments. Very often, this is caused by the failure of anti-cancer therapies to kill the cancer cells. FLIP is a cellular protein that cancer cells frequently express at high levels, and this increases their resistance to chemotherapy and other types of therapy used to treat cancer, such as radiotherapy. The agents that we are developing target FLIP and prevent it from causing therapy resistance. In addition, some tumour-promoting immune cells also need FLIP, so our FLIP inhibitors may also have beneficial effects in the tumour microenvironment by reactivating immune cells to attack cancer cells."

On March 28, 2019 AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, reported that the Company will be presenting new data at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, Georgia (Press release, AIVITA Biomedical, MAR 28, 2019, View Source [SID1234534704]). Chief Medical Officer Robert O. Dillman, M.D. will give a presentation concerning prognostic and predictive markers of efficacy for patients receiving AIVITA’s next-generation immunotherapy targeting tumor-initiating stem cells.

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The American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting is a six-day meeting covering the latest discoveries in cancer research. The presentation details are as follows:

Title: Soluble programmed cell death molecule-1 (sPD-1) as a prognostic and predictive biomarker in patients with metastatic melanoma randomized for treatment with autologous dendritic cell or tumor cell vaccines.
Session Category: Clinical Research
Session Title: Circulating and Cell-free Biomarkers for Diagnosis and Monitoring of Cancer 2
Session Date and Time: Monday Apr 1, 2019 8:00 AM – 12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 18
Poster Board Number: 26
Permanent Abstract Number: 1257

AIVITA is currently conducting three clinical studies investigating its platform ROOT OF CANCER therapy in patients with ovarian cancer, glioblastoma and melanoma. AIVITA uses 100% of proceeds from the sale of its ROOT of SKIN skincare line to support the treatment of women with ovarian cancer.

About ROOT OF CANCER

OVARIAN CANCER

AIVITA’s treatment is a platform technology applicable to most solid tumor types and consists of autologous dendritic cells loaded with autologous tumor antigens from purified autologous self-renewing tumor-initiating cells.

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous cancer stem cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the cancer stem cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with the cancer stem cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

For additional information about AIVITA’s AV-MEL-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03743298

On March 28, 2019 Veracyte, Inc. (Nasdaq: VCYT), a leading genomic diagnostics company, reported that Bonnie H. Anderson, chairman and chief executive officer, is scheduled to present at the 18th Annual Needham Healthcare Conference in New York City on Wednesday, April 10, 2019 at 8:00 a.m. EDT (Press release, Veracyte, MAR 28, 2019, View Source [SID1234534720]).

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A live audio webcast of the company’s presentation will be available by visiting Veracyte’s website at View Source A replay of the webcast will be available for 90 days following the conclusion of the live presentation broadcast.