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Unum Therapeutics Inc. corporate presentation.

On January 13, 2020 Unum Therapeutics Inc. (the "Company") presented a corporate presentation (Presentation, Unum Therapeutics, JAN 13, 2020, View Source [SID1234553153]).

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Momenta Provides Year-End 2019 Update and 2020 Outlook

On January 13, 2020 Momenta Pharmaceuticals, Inc. (NASDAQ: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, reported a 2019 year-end corporate update and key anticipated milestones for 2020 (Press release, Momenta Pharmaceuticals, JAN 13, 2020, View Source [SID1234553152]).

"We expect 2020 to be a significant year of value inflection for Momenta, as we approach key data readouts across our novel auto- and alloimmune programs. In particular, we start the year with promising interim data for our M254 program in ITP, with additional data expected in Q2, and look forward to sharing nipocalimab data in MG in Q3," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals. "We also continue to grow our pipeline, with the goal to initiate a Phase 2 study for M254 in CIDP later this year and the nomination of a new SIFbody development candidate. With a strong balance sheet supporting our efforts, we have multiple new product and potential partnering opportunities ahead of us.

It has been a pivotal year for Momenta, due to the dedication of our team and their focus on unraveling the complexity of immune-mediated diseases. Today we are proud to reveal our new corporate identity. With a renewed purpose and a strong drive to make a meaningful difference in the lives of patients with unmet needs, this new brand is a great way to kickoff 2020."

Overview of Programs and Anticipated 2020 Milestones

M254 (hsIgG): a hypersialylated immunoglobulin designed as a high potency alternative for intravenous immunoglobulin (IVIg)

·The Company’s multi-part Phase 1/2 clinical trial in idiopathic thrombocytopenic purpura (ITP) has completed Part A and is progressing through Part B, which is evaluating M254 in a single ascending dose (SAD) cohort of ITP patients, followed by 1,000 mg/kg of IVIg.

·Five of six ITP patients administered M254 (43 mg/kg to 250 mg/kg) responded and exhibited platelet counts >=50 x 109/L.

·Based on early favorable responses to M254 and variable responses to IVIg, the Company is augmenting the number of patients in the Part B cohorts and including a lower dose cohort.

·Enrollment is ongoing and the Company expects to report additional data from Part B of the study in the second quarter of 2020.

·The Company also plans to initiate a clinical study of M254 in chronic inflammatory demyelinating polyneuropathy (CIDP) later in 2020.

Nipocalimab (M281): a fully human anti-neonatal Fc receptor (FcRn) aglycosylated immunoglobulin G (IgG1) monoclonal antibody (mAb)

·Vivacity-MG, the Company’s Phase 2 clinical study of nipocalimab in generalized myasthenia gravis (gMG), continues to enroll patients, with 80% of the trial enrolled.The Company expects to report top-line data from this study in the third quarter of 2020.

·Unity, the Company’s global multi-center Phase 2 clinical study of nipocalimab in hemolytic disease of the fetus and newborn (HDFN), is actively enrolling patients,with four patients currently enrolled. The Company expects to report top-line data from this study in 2021.

·Energy, the Company’s adaptive Phase 2/3 clinical study of nipocalimab in warm autoimmune hemolytic anemia (wAIHA) commenced in the third quarter 2019. The Company is activating clinical sites in both the United States and European Union and is enrolling patients. Nipocalimab has been granted Fast Track and Orphan Drug designation by the FDA in this indication and the Company expects to report top-line data from this study around the end of 2021.

M230 (CSL730): a recombinant Fc multimer being developed in collaboration with CSL

·A Phase 1 clinical program to evaluate the safety and tolerability of M230 in healthy volunteers is continuing and Momenta’s partner, CSL, looks forward to introducing a subcutaneous formulation into the phase 1 program.

Momenta’s SIFbody platform combines multiple Fc’s with antibody fabs to optimally activate Fc and complement effector function and effectively deplete target cells. CD38 is a target on plasmacytes, immune system cells responsible for generating autoantibodies (autoimmune disease) and M protein (multiple myeloma). Pre-clinical data suggests this CD38 SIFbody has the potential to be a best-in-class therapeutic for the management of plasmacyte-mediated diseases such as multiple myeloma, AL amyloidosis and rare, auto-antibody-mediated diseases.

·The Company has nominated an early development CD38 SIFbody candidate and expects to initiate IND enabling studies in 2020.

Financial Guidance

Momenta provides non-GAAP operating expense guidance, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP financial measures. Non-GAAP operating expense is total operating expense, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenue.

Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion of this subject.

·Momenta expects that its non-GAAP operating expense for the fourth quarter of 2019 will be in line with its previously announced guidance, which was outlined in its third-quarter 2019 earnings release.

·The Company expects to report approximately $545.1 million of cash, cash equivalents and marketable securities at December 31, 2019.

·Momenta is providing 2020 full year estimated non-GAAP operating expense guidance of $220-240 milion.

Non-GAAP Financial Information and Other Disclosures

Momenta uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Momenta believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Momenta’s operating performance as it excludes non-cash stock compensation expense, restructuring expense and collaborative reimbursement revenue. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Momenta’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Momenta’s operating results as reported under GAAP. Momenta has not provided forward-looking GAAP operating expense or GAAP reconciliation for its forward-looking non-GAAP annual operating expense because Momenta cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure. The Company does not expect restructuring expense and collaboration reimbursement revenue to be material.

The preliminary, unaudited financial information provided or referred to in this release is based on Momenta’s current estimate of its results for the quarter ended December 31, 2019 and its cash, cash equivalents, and marketable securities as of December 31, 2019, and remains subject to change based on closing and audit procedures, and the execution of Momenta’s internal control over financial reporting.

Alkermes plc corporate presentation.

On January 13, 2020 Alkermes Presented the corporate presentation (Presentation, Alkermes, JAN 13, 2020, View Source [SID1234553151]).

Investor Presentation dated January 2020

On January 13, 2020 Moleculin Presented the corporate presentation (Presentation, Moleculin, JAN 13, 2020, View Source [SID1234553150]).

Replimune Provides 2019 Year End Corporate Update and Reviews Expected 2020 Milestones

On January 13, 2020 Replimune Group Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported a corporate update, highlighting the progress of its key programs (Press release, Replimune, JAN 13, 2020, View Source [SID1234553149]).

"Replimune had a very productive 2019 where a key highlight included the presentation of initial clinical data with RP1 as monotherapy and combined with Opdivo at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)," said Philip Astley-Sparke, Chief Executive Officer of Replimune. "We believe the data provides strong support for our ongoing registration-directed trial in our lead indication of CSCC as well as the decision to initiate a new clinical trial in organ transplant recipients who have developed the disease. Similarly, the data in melanoma led to the decision to expand the clinical trial to include a registration-directed cohort of melanoma patients who are refractory to prior treatment with anti-PD1 therapy. We also initiated our first clinical trial of RP2, the next therapy in our pipeline. Overall we are thrilled with our progress and are excited for the year ahead as we release further data sets, bring our third product into the clinic to address less immune-responsive tumors and as we continue to execute upon our mission to make our oncolytic immuno-gene therapies a cornerstone of cancer treatment."

Program Updates

RP1: RP1 is Replimune’s first clinical product candidate and is based on a proprietary new strain of herpes simplex virus armed with a gene encoding a potent fusogenic protein (GALV-GP-R-), intended to enhance tumor killing potency, immunogenic cell death and the activation of systemic anti-tumor immune responses, and with a gene encoding the cytokine GM-CSF. The Company presented initial data from the Phase 1/2 clinical trial of RP1 alone and in combination with Opdivo in November 2019 at SITC (Free SITC Whitepaper) 2019. The Company believes the data demonstrates that RP1 used alone and in combination with Opdivo is well tolerated and showed preliminary anti-tumor efficacy in target tumor types providing strong support for its ongoing and planned development programs in melanoma and CSCC. This clinical trial is being conducted under a clinical trial collaboration and agreement with Bristol-Myers Squibb (BMS) for the supply of Opdivo.

Recent RP1-specific program progress is summarized below:

The registration-directed Phase 2 clinical trial of RP1 in combination with Libtayo in CSCC is underway. This multi-center, randomized, controlled clinical trial is intended to enroll approximately 240 patients with CSCC. The clinical trial’s primary objective is to compare the response rate following treatment with RP1 in combination with Libtayo to the response rate achieved with Libtayo alone. Libtayo is an FDA-approved anti-PD-1 therapy developed by Regeneron and Sanofi for the treatment of patients with metastatic or locally-advanced CSCC who are not candidates for curative surgery or radiation. This clinical trial is being conducted under the Company’s collaboration agreement with Regeneron, whereby the Company and Regeneron split development and supply costs equally. Recruitment for this clinical trial is expected to take approximately 18 to 24 months and enroll patients in the United States, Australia, Canada, United Kingdom and European Union.

Recruitment is currently ongoing in the Phase 2 part of the Phase 1/2 clinical trial of RP1 in combination with Opdivo. The Phase 2 part of the clinical trial is currently enrolling 30-patient cohorts with melanoma, non-melanoma skin cancers, metastatic bladder cancer, and MSI-H tumors. Data from completely enrolled or ongoing skin cancer cohorts is expected to be presented during the second half of 2020 with further data from all four cohorts expected to be available by year-end.

On track to initiate a new registration-directed 125-patient cohort in the Phase 2 clinical trial of RP1 in combination with Opdivo in anti-PD-1 refractory melanoma patients in the first quarter of 2020, based on the initial clinical efficacy data with RP1 in melanoma. The additional cohort will be enrolled under an expansion of the clinical trial collaboration and Opdivo supply agreement with BMS.

On track to initiate a clinical trial of RP1 as monotherapy in solid organ transplant recipients with CSCC in the first half of 2020. The U.S. Food and Drug Administration (FDA) has accepted the protocol for this clinical trial under its previously-accepted Investigational New Drug application for RP1. The clinical trial is intended to enroll approximately 30 patients and assess the safety and efficacy of RP1 in liver and kidney transplant recipients with recurrent CSCC and is expected to provide initial data by the end of 2020.
RP2: RP2 is a version of RP1 that, in addition to expressing GALV-GP-R and GM-CSF, also expresses a genetically encoded anti-CTLA-4 antibody intended to block the inhibition of the initiation of immune response caused by CTLA-4. Similar to RP1, RP2 is intended to be used primarily in combination with anti-PD-1 therapy.

The Phase 1 clinical trial of RP2 alone and in combination with Opdivo is underway. The clinical trial is designed to assess the safety and tolerability, and to determine the optimal dose, of RP2 alone and in combination with Opdivo and is being conducted under a clinical trial collaboration and agreement with BMS for the supply of Opdivo. Initial data from this all-comers trial is expected by the end of 2020.
RP3: RP3 is a further armed oncolytic immuno-gene therapy which expresses two immune co-stimulatory activating ligands – CD40L and 4-1BBL – together with anti-CTLA-4 and GALV-GP-R-. CD40L activates CD40, which is intended to result in the broad activation of both innate and adaptive immunity, and 4-1BBL activates 4-1BB (CD137), which is intended to promote the expansion of cellular and memory immune responses.

The Phase 1 clinical trial of RP3 alone and in combination with an anti-PD-1 therapy is expected to initiate in 2020.
Corporate Updates

Manufacturing: Completed the build-out of Replimune’s state of the art manufacturing facility to support late-stage development and commercialization. The 63,000-square-foot facility in Framingham, MA is intended to provide multi-product manufacturing capabilities for Replimune’s Immulytic product candidates. The Company believes that the capacity of this facility is sufficient to support full commercialization of its product candidates. An occupancy certificate for the facility has been obtained and the first technology transfer manufacturing run has been successfully completed. Full GMP manufacturing is expected to commence in the first half of 2020.

Cash Position: Based on its current operating plan, Replimune expects that its cash, cash equivalents and short-term investments, of approximately $183 million as of December 31, 2019, will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2022.
J.P. Morgan Conference Presentation and Webcast.

As previously announced, the Company will be presenting at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14th at 5:00 p.m. PT at the Westin St. Francis Hotel in San Francisco, CA. A simultaneous webcast of the presentation will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.