On May 7, 2019 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the first quarter ended March 31, 2019 (Press release, QLT, MAY 7, 2019, View Source [SID1234535833]).

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Novelion’s Interim Chief Executive Officer Ben Harshbarger commented, "We continue to execute on our goals of achieving target revenues, maximizing the impact of the operating cost reductions that we implemented in late 2018, working with the FDA on the development program for the potential expansion of the metreleptin label in the U.S. to include the partial lipodystrophy (PL) indication, and pursuing a comprehensive capital restructuring."

Business Update

Sales growth was supported by the launch of MYALEPTA (metreleptin) in Germany in the fourth quarter of 2018. Following marketing authorization of MYALEPTA for generalized lipodystrophy (GL) and PL by the EMA, Novelion’s subsidiary Aegerion Pharmaceuticals commenced the pricing and reimbursement processes in key EU markets. Reimbursement decisions in many of the key EU markets are anticipated throughout 2019.

In January 2019, Aegerion held a meeting with the U.S. Food and Drug Administration (FDA) to obtain feedback on the design of the placebo-controlled study for the PL indication for MYALEPT in the U.S. Aegerion is assessing feedback on the study design and integrating it into the Phase 3 study protocol.

Aegerion plans to file for regulatory approvals for metreleptin in GL and PL in certain key markets outside the U.S. and EU, including Brazil, in 2019.

As previously announced, Novelion and Aegerion have each engaged advisors to independently explore and advise them on all available strategic alternatives regarding the Company’s capital structure, such as a restructuring of Aegerion’s Convertible Notes due August 2019 (including a restructuring that would likely involve a debt for equity exchange), a sale or merger of Novelion or Aegerion, or the sale or other disposition of certain businesses or assets. The implementation of one or more of such transactions (or the failure to complete any such transaction or transactions) will likely require Aegerion, and could require Novelion, to seek the protections of applicable bankruptcy laws allowing for corporations to seek to restructure their debts and other affairs under a court supervised reorganization process.

First Quarter 2019 Financial Results

JUXTAPID: The Company reported net revenues of JUXTAPID of $14.2 million in the first quarter of 2019, compared to $13.4 million for the same period in 2018, $9.1 million, or 64.1%, of which was from prescriptions written in the U.S. and $0.8 million of which was royalty revenue from sales in the EMEA region. Growth in JUXTAPID revenue in the first quarter of 2019 compared to the same period in 2018 was due to an increase in revenues in the U.S., primarily resulting from a price increase, and a greater number of patients on therapy in Japan.

MYALEPT/MYALEPTA: The Company reported net revenues of MYALEPT/MYALEPTA of $18.0 million in the first quarter of 2019, compared to $14.1 million for the same period in 2018, $11.4 million, or 63.3%, of which was from prescriptions written in the U.S. MYALEPT/MYALEPTA revenue growth in the first quarter was driven by increased sales in Germany, as a result of the recent launch, and the U.S., as a result of a greater number of shipments to patients as well as a price increase, partially offset by a decline in sales in Brazil.

GAAP total net revenues for the first quarter of 2019 were $32.2 million compared to $27.5 million for the same period in 2018.

Cost of product sales for the first quarter of 2019 was $15.2 million compared to $13.5 million for the same period in 2018. The increase is primarily attributed to higher stability testing costs and higher supply chain costs as well as a higher royalty rate on U.S. sales of metreleptin, partially offset by a lower inventory cost basis in the first quarter of 2019. Despite the increase, gross margin improved from 50.9% to 52.7% as a result of higher revenues in comparison to certain fixed costs.

GAAP total operating expenses for the first quarter of 2019 were $33.0 million compared to total operating expenses of $35.5 million, a 7.0% reduction compared to the same period in 2018. GAAP selling, general and administrative (SG&A) expenses were $26.0 million in the first quarter of 2019, including approximately $13.8 million of costs related to the Company’s ongoing debt restructuring and strategic review processes, compared to $23.7 million for the same period in 2018. GAAP R&D expenses were $6.9 million in the first quarter of 2019 compared to $11.8 million for the same period in 2018.

On a non-GAAP basis, during the first quarter of 2019, SG&A expenses were $11.7 million compared to $21.6 million for the same period in 2018. In the first quarter of 2019, we incurred $12.3 million of non-ordinary course expenses, which primarily consist of legal, financial/restructuring advisory, and consulting fees, in connection with our ongoing strategic alternatives review, as well as a $1.5 million payment to extend the maturity date of Aegerion’s bridge loans. The 45.8% decrease in non-GAAP SG&A expenses in the first quarter of 2019 compared with the same period in 2018 is primarily the result of cost reduction initiatives implemented in late 2018 as well as the delay of certain ordinary course projects and initiatives to later in the year.

On a non-GAAP basis, during the first quarter of 2019, R&D expenses decreased 41.4% to $6.8 million compared to $11.6 million for the same period in 2018, reflecting cost reduction initiatives, as well as the delay of certain ordinary course projects and initiatives to later in the year, partially offset by higher spending related to pharmacovigilance activities.

On a non-GAAP basis, during the first quarter of 2019, total operating expenses were $18.6 million compared to $33.3 million for the same period in 2018, reflecting a decrease in expenses for the reasons set forth above.

GAAP net loss in the first quarter of 2019 was $31.8 million, an improvement of approximately 3.0% compared to GAAP net loss of $32.8 million during the same period in 2018.

On a non-GAAP basis, net income was $1.9 million in the first quarter of 2019 compared to a net loss of $13.5 million for the same period in 2018.

A full reconciliation of the GAAP financial results to non-GAAP financial results is included in the financial information tables below.

Debt and Government Settlement Payments

As of March 31, 2019, Aegerion’s debt liabilities and government settlement payments included $302.5 million in outstanding principal under Aegerion’s Convertible Notes due August 15, 2019, $74.4 million in outstanding principal (including paid in kind fees and interest) under Aegerion’s secured term loans having a maturity date of June 30, 2019, $35.7 million outstanding under Aegerion’s secured intercompany term loan with Novelion, as lender, which has a maturity date of July 1, 2019, as well as $29.3 million owed under Aegerion’s settlements with the Department of Justice and the U.S. Securities and Exchange Commission (the "Commission"), payable in prescribed installments until the first quarter of 2021.

Financial Guidance

Novelion expects total net revenues in 2019 to be between $160.0 and $175.0 million, including approximately $30.0 million of licensing revenues, in the form of the $25.0 million upfront licensing payment and $5.0 million payment upon transfer of the marketing authorization to Recordati Rare Diseases Inc. ("Recordati"), resulting from the licensing agreement entered into between Aegerion and Recordati for the commercialization of JUXTAPID in Japan in February 2019.

About Novelion Therapeutics

Novelion Therapeutics is a global biopharmaceutical company dedicated to developing and commercializing therapies that deliver new standards of care for people living with rare and underserved diseases. With a global footprint and an established commercial portfolio, including MYALEPT (metreleptin) and JUXTAPID (lomitapide), our business is supported by differentiated treatments that treat severe and rare diseases.

Cautionary Note

Novelion is the parent company of Aegerion, our operating subsidiary and the source of the consolidated company’s revenues. References to "we," "our" and the "Company" refer to the entire enterprise, whose assets and operations reside primarily at Aegerion. As described above, Aegerion has a substantial amount of debt, including a secured term loan entered into in November 2018, which matures on June 30, 2019, its 2% Convertible Notes, which mature on August 15, 2019, and a secured intercompany term loan from Novelion, which matures on July 1, 2019. In light of these arrangements and their provisions, which prohibit Aegerion from making certain payments,

including payments in cash, to Novelion (including for out-of-pocket costs incurred, and services rendered, by or on behalf of Novelion, for the benefit of Aegerion), investors are cautioned that Aegerion’s interests may not always be aligned, and may in certain circumstances be in conflict, with those of Novelion or its shareholders. The risks attendant to these conflicts of interest are described below under the caption "Forward Looking Statements and Risk Factors," which section you should read carefully and in its entirety.

Non-GAAP Results

The non-GAAP results in this press release, including, without limitation, non-GAAP R&D expenses, non-GAAP SG&A expenses, non-GAAP total operating expenses and non-GAAP net income (loss), are provided as a complement to results provided in accordance with GAAP because management believes, when considered together with the GAAP information, these non-GAAP financial measures help indicate underlying trends in the Company’s business, are important in comparing current results with prior period results and provide additional information regarding the Company’s financial performance. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, and to manage the Company’s business and evaluate its performance. The non-GAAP financial measures have no standardized meaning under GAAP and therefore may not be comparable to similar measures presented by other companies. The non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, the financial measures prepared and presented in accordance with GAAP and should be reviewed in conjunction with the relevant GAAP financial measures. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

On May 7, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported financial results for the quarter ended March 31, 2019 (Press release, Gamida Cell, MAY 7, 2019, View Source [SID1234535850]). The company also highlighted continued progress in advancing its clinical development candidates: omidubicel1 (formerly known as NiCord), an investigational advanced cell therapy in Phase 3 clinical development designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant, and GDA-201 (formerly known as NAM-NK), an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma and multiple myeloma.

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"Gamida Cell is focused on transforming the treatment landscape for patients with blood cancers and rare, serious hematologic diseases. We are pleased that omidubicel has been selected as the nonproprietary name for NiCord, highlighting our progress toward bringing this important cell therapy to patients in need of a bone marrow transplant," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We have also made several key personnel appointments this year that reflect our strategic focus on commercial preparedness, including hiring our first chief commercial officer and nominating new board members who bring commercial, operational and financial experience to Gamida Cell’s board of directors."

Dr. Adams continued, "We are pleased that the multi-center, randomized Phase 3 study of omidubicel is progressing, with patient enrollment expected to be complete by the end of this year and topline data anticipated in the first half of 2020. Positive data from the study would enable the submission of our first biologics license application next year, which would be a significant achievement."

"Earlier this year, we also reported encouraging data from the Phase 1 clinical study of our natural killer cell product candidate, GDA-201, previously known as NAM-NK. The multiple complete responses observed emboldened us to begin scaling up our manufacturing process to enable the evaluation of a cryopreserved formulation of GDA-201 in a multi-center, multi-dose Phase 1/2 clinical study in patients with non-Hodgkin lymphoma next year," Dr. Adams concluded.

Company Highlights

Omidubicel selected as nonproprietary name for NiCord: Today Gamida Cell announced that the United States Adopted Names (USAN) Council selected omidubicel as the nonproprietary name for Gamida Cell’s investigational hematopoietic stem cell expanded through the company’s proprietary nicotinamide-based, or NAM, technology. The USAN Council aims for global standardization and unification of drug nomenclature to ensure that drug information is communicated accurately and unambiguously. Gamida Cell’s lead investigational product has two components: omidubicel (hematopoietic stem cells expanded through the company’s proprietary nicotinamide-based, or NAM, technology) and differentiated immune cells, including T cells. Gamida Cell refers to the two components collectively as "omidubicel." Going forward, Gamida Cell will use the name "omidubicel" in publications and public statements, at conferences and other forums, and in medical and commercial-related materials.
Reported encouraging data for omidubicel and GDA-201 at TCT Annual Meeting: In February, data from the omidubicel and GDA-201 clinical programs were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation and Center for International Blood and Marrow Transplant. Research from the completed Phase 1/2 clinical study of omidubicel demonstrated that recipients who received omidubicel had rapid and robust reconstitution of key immune cells. Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant.

Data were also reported from the ongoing Phase 1/2 study of omidubicel in patients with severe aplastic anemia. In the initial cohort of three patients, all successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The results enable the initiation of a second cohort of patients to be treated with omidubicel as a stand-alone graft. Patient enrollment in the second cohort is expected to begin in the first half of 2019.

Additionally, data reported from the ongoing Phase 1 study of GDA-201 in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM) demonstrated that GDA-201 was clinically active, with three complete responses observed in patients with NHL and one complete response in a patient with MM. These data, along with safety data showing that GDA-201 was generally well tolerated, support continued clinical development. Gamida Cell is planning to initiate a multi-center, Phase 1/2 clinical study of GDA-201 in patients with NHL in 2020.
Evolved Board of Directors to reflect company’s progress toward commercialization: In March, the company announced the nominations of Shawn Cline Tomasello and Stephen T. Wills to its board of directors. These nominations require approval at the Annual Shareholders Meeting, which will take place in June 2019. Ms. Tomasello has extensive experience in commercializing first-in-class medicines for the treatment of cancer, including Yescarta (at Kite Pharma, now part of Gilead Sciences) and Imbruvica (at Pharmacyclics, now part of AbbVie). Mr. Wills has extensive operational, financial and transactional experience over nearly three decades in the life sciences and accounting industries. He has served as chief financial officer of Palatin Technologies, a publicly-traded biotechnology company developing peptide therapeutics, since 1997 and also serves as Palatin’s chief operating officer and executive vice president.

In January, the company appointed Nurit Benjamini to Gamida Cell’s board of directors and chair of the board’s audit committee. Ms. Benjamini has served as chief financial officer of TabTale Ltd. since 2013. Previously, she held a number of chief financial officer positions, including at Wix.com Ltd., Sigma Designs Israel Ltd. and Compugen Ltd.
Appointed Thomas Klima as chief commercial officer: In January, the company announced the appointment of Thomas Klima as chief commercial officer. In this newly created role, Mr. Klima will be responsible for building the team and executing the strategy to potentially bring omidubicel to patients, including oversight of reimbursement and patient services. Klima brings nearly 20 years of global experience in the pharmaceutical industry with expertise in cellular therapy, hematology, oncology and transplantation. During his career, he has played key roles in building commercial organizations and leading multiple successful product launches.
Anticipated 2019-2020 Milestones
Gamida Cell’s anticipated program milestones in 2019-2020 are as follows:

Omidubicel

Initiate Cohort 2 in the Phase 1/2 study evaluating omidubicel as stand-alone graft in severe aplastic anemia in the first half of 2019
Complete enrollment in Phase 3 study of omidubicel in patients with hematologic malignancies in the second half of 2019
Report topline data from the Phase 3 study of omidubicel in patients with hematologic malignancies in the first half of 2020
Complete BLA submission for omidubicel in hematologic malignancies in the second half of 2020, should Phase 3 data be positive
GDA-201

Complete patient enrollment in the ongoing Phase 1 study in the second half of 2019
Present additional data at a medical meeting in the second half of 2019
Initiate multi-center, Phase 1/2 clinical study in patients with NHL in 2020
First Quarter 2019 Financial Results

As of March 31, 2019, Gamida Cell had total cash, cash equivalents and available-for-sale securities of $50.3 million, compared to $60.7 million as of December 31, 2018.
Research and development expenses in the first quarter of 2019 were $7.3 million, compared to $5.1 million in the same period in 2018. The difference was attributable mainly to a $1.2 million increase in clinical activities relate to the advancement of omidubicel and GDA-201, $0.5 million reduction in grants received from the Israeli Innovation Authority (IIA) and an increase of $0.5 million in compensation and other R&D expenses.
General and administrative expenses were $3.8 million for the first quarter of 2019, compared to $1.7 million in the same period in 2018. The increase was due mainly to a $1.0 million increase in expenses related to hiring and establishing the U.S. headquarters, an increase of $0.5 million in non-cash stock-based compensation expenses, and $0.6 million in professional services, rent and other expenses.
Finance expenses, net, were $4.4 million for the three months ended March 31, 2019, compared to $0.7 million in income in the same period in 2018. The increase was primarily due to noncash expenses resulting from revaluation of warrants and the revaluation of royalty-bearing grant IIA liability.
Net loss for the first quarter of 2019 was $15.5 million, compared to a net loss of $7.4 million in the same period in 2018.
2019 Financial Guidance
Gamida Cell continues to expect cash used for ongoing operating activities in 2019 to range from $35-$40 million, reflecting anticipated expenditures to advance the company’s clinical programs.

Gamida Cell expects that its cash, cash equivalents, available-for-sale securities and short-term debt will support the company’s capital needs through the data readout for the Phase 3 clinical study of omidubicel, which is expected in the first half of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

Conference Call Information
Gamida Cell will host a conference call today, May 7, 2019, at 8:30 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 2277888. A replay of the webcast will be available for approximately 30 days.

About Omidubicel
Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers).1 Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.2 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.3 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.4 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.5

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On May 7, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, reported its financial results for the first quarter ended March 31, 2019 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, MAY 7, 2019, View Source [SID1234535866]).

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"We are excited by the continued strong momentum across our business," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "We recently presented new positive data from ASPIRO, the Phase 1/2 study of AT132 for the treatment of XLMTM, at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). These data further support the compelling profile of this important product candidate and help further our progress toward the goal of making AT132 globally available to patients living with XLMTM as rapidly as possible."

Mr. Patterson continued, "In our Pompe disease program, this quarter we successfully completed an initial NHP safety study of our product candidate AT845 and the encouraging results give us added confidence as we complete GLP toxicology and dose-ranging studies to support a third quarter IND submission. Finally, we are thrilled to have recently announced the expansion of our technology platform and development pipeline with the addition of programs utilizing vectorized antisense to address Duchenne muscular dystrophy and myotonic dystrophy, two devastating neuromuscular diseases."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy (XLMTM):

Presented new positive data from ASPIRO at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper).
The ASGCT (Free ASGCT Whitepaper) data set included safety and efficacy assessments out to 48 weeks of follow-up for six treated patients in dose Cohort 1 (1×1014 vg/kg) and 24 weeks for three treated patients in dose Cohort 2 (3×1014 vg/kg).
Patients receiving AT132 achieved reductions in ventilator dependence not previously observed in chronically ventilated patients with neuromuscular disorders, including four patients achieving ventilator independence and all other treated patients demonstrating sustained and clinically meaningful reductions in ventilator use.
The data also demonstrated sustained improvements in neuromuscular function in both dose cohorts with corresponding achievement of clinically meaningful motor milestones. Additionally, muscle biopsy data continued to show robust tissue transduction, protein expression, and histological improvements in 24 and 48-week muscle biopsies in all patients, with evidence of more rapid pathological improvement by week 24 in the Cohort 2 biopsy samples.
AT132 has been generally well-tolerated and has shown a manageable safety profile across both dose cohorts with no clinically meaningful safety differences between doses to date.
Enrollment of an additional three to five patients in Cohort 2 (3×1014 vg/kg) of ASPIRO is ongoing.
On track to select optimal dose of AT132 in the second quarter of 2019. Plan to provide an updated data package to FDA and on track to provide update on the license application submission plans for AT132 in the third quarter of 2019.
Next clinical data presentation planned at the 24th International Annual Congress of the World Muscle Society (WMS) in Copenhagen, Denmark, October 1-5, 2019.
AT845 for Pompe Disease:

Successfully completed an initial non-GLP NHP study, which showed a clean safety profile.
Previously planned GLP toxicology and dose-ranging studies in progress.
On track to file IND in the third quarter of 2019.
AT702/AT751/AT753 for Duchenne Muscular Dystrophy (DMD):

Announced exclusive license agreement with the Research Institute at Nationwide Children’s Hospital to develop AT702, an AAV-antisense candidate designed to induce exon 2 skipping for DMD caused by duplications of exon 2 and mutations in exons 1-5 of the dystrophin gene. Conducting additional preclinical work of AT702 and plan to commence a Phase 1/2 study at Nationwide Children’s in the fourth quarter of 2019.
Separate from the Nationwide Children’s collaboration, conducting preclinical work to advance AT751 and AT753, additional vectorized exon skipping candidates designed to treat DMD patients with genotypes amenable to exon 51 and exon 53 skipping. Both AT751 and AT753 utilize the same vector construct backbone as AT702, enabling a potentially accelerated path into clinical development.
Initial programs target more than 25% of patients with DMD. Plan to leverage vectorized exon skipping platform to develop further product candidates to address up to 80% of DMD patients over time.
AT466 for Myotonic Dystrophy (DM1):

Collaboration announced with Nationwide Children’s to evaluate vectorized RNA knockdown and vectorized exon skipping for DM1.
Preclinical studies are underway, and IND planned for 2020.
AT342 for Crigler-Najjar Syndrome and AT307 for CASQ2-CPVT:

Completed a strategic review of our product candidates and plan to focus on those programs that have the potential to create the greatest long-term value for patients and shareholders.
Plan to explore outlicensing opportunities to continue to advance these important and promising gene therapy programs.
Manufacturing:

Announced new internal cGMP plasmid manufacturing facility, further establishing the company’s leadership in AAV manufacturing and enabling the rapid advancement of neuromuscular programs from preclinical development to commercialization.
Made continued progress on chemistry, manufacturing, and controls (CMC) BLA and MAA-readiness efforts for AT132 based on FDA and EMA feedback on the company’s preliminary filing strategy.
Corporate:

Expanded leadership team with the additions of Mark Meltz, Senior Vice President, General Counsel, to lead the legal, compliance, and corporate governance functions and Sarah Spencer, Vice President, Corporate Communications, to lead internal and external communications to build global awareness of the company, its mission, culture, and innovative product portfolio.
First Quarter 2019 Financial Results

Cash Position: At March 31, 2019, Audentes had cash, cash equivalents and marketable securities of $375.0 million, which is expected to fund operations into 2021.
Research and Development Expenses: Research and development expenses were $39.8 million for the first quarter of 2019 compared to $19.9 million for the same period in 2018, an increase of $19.9 million. The increase in research and development expense was primarily attributable to increased R&D headcount and related facility costs, a one-time licensing fee of $7.0 million related to the AT702 program, increased internal manufacturing costs, increased clinical trial expenses for our AT132 program, increased pre-clinical and clinical trial planning costs for our AT845 program, and an increase in stock compensation expense.
General and Administrative Expenses: General and administrative expenses were $12.0 million for the first quarter of 2019 compared to $6.5 million for the same period in 2018, an increase of $5.5 million. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs, increased professional service and consulting fees, higher costs related to public company regulatory compliance and increase in stock compensation expense.
Net Loss: Net loss was $49.4 million for the first quarter of 2019 compared to $25.6 million for the same period in 2018.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its first quarter 2019 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 6839198 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

On May 7, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases and extend healthy lifespan, reported its financial results for the first quarter ended March 31, 2019 (Press release, CohBar, MAY 7, 2019, View Source [SID1234535945]).

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"The clear highlight since our last quarterly update was our announcement last week that we will be resuming the clinical trial of our lead therapeutic candidate, CB4211, for NASH and obesity," said Philippe Calais, CohBar’s interim CEO. "At the same time, we are expanding our evaluation efforts with novel analogs of mitochondrial encoded peptides targeting new potential indications, notably type 2 diabetes, cancer and fibrotic diseases. We continue to identify interactions between our novel analogs and new disease models, further validating our belief that mitochondria based therapeutics have significant potential to treat a broad range of age-related diseases with unmet medical needs."

First Quarter 2019 and Recent Clinical, Research and Business Highlights:

Upcoming Resumption of CB4211 Clinical Trial. The company recently announced that it will be resuming its Phase 1a/1b clinical trial of CB4211, its lead MBT candidate under development for the treatment of nonalcoholic steatohepatitis (NASH) and obesity. The company previously announced the suspension of the study in November 2018, to address mild but persistent injection site reactions.

New potential disease targets and mechanisms of action. During the first quarter and more recently, the company continued to advance its optimization and evaluation of novel analogs of mitochondrially encoded peptides with the identification of new disease targets and potential mechanisms of action. Recent studies have provided preliminary data on the evidence of antifibrotic activity of a novel peptide analog in in vitro and in vivo models of lung fibrosis, while another study demonstrated significant progress in exploring the potential for CohBar’s novel peptide analogs to enhance cancer cell killing in the setting of immuno-oncology.

Upcoming Presentation at American Diabetes Association, 79th Scientific Sessions. CohBar will present its recent discovery of the mechanism of action of a family of novel peptides with effects on glucose tolerance in animal models of type 2 diabetes. The mechanism involves interaction with a key cell surface receptor that plays an important role in a number of age-related diseases. We expect that the abstract title and session information will be available to the public on the ADA website on May 10, 2019.

Expansion of Board of Directors. David Greenwood joined the CohBar Board in April 2019. Mr. Greenwood brings to CohBar more than 40 years of financial and operational experience in biotechnology and investment banking. Mr. Greenwood served in leadership roles and on boards at both public and private biotechnology companies including Corium Inc. (formerly NASDAQ: CORI) and Geron Corporation.

Hosted Key Opinion Leader Call, "Mitochondria, a Source for Novel Therapeutics." The company hosted a Key Opinion Leader conference call and webcast on May 2, 2019, featuring world-leading experts and CohBar founders Dr. David Sinclair and Dr. Pinchas Cohen, and CohBar CSO Dr. Ken Cundy, discussing the role of mitochondria in health and aging and mitochondrial-derived peptides (MDPs) as a novel source of potential therapeutics for a host of major age-related diseases. A replay of the call is available on the homepage of the CohBar website, (www.cohbar.com).

Expanded IP Portfolio. The company’s IP portfolio recently expanded with the grant of a U.S. patent for its licensed asset MOTS-c. The company also converted its PCT application that describes CB4211 and its analogs into individual patent filings in the U.S. and in multiple international jurisdictions. The applications claim CB4211 composition of matter and methods for using it for the treatment of various diseases.

Continued Investment Community Outreach. In addition to meetings and presentations earlier in the quarter at the JP Morgan Healthcare Conference and at the BIO Investor Forum, company management met with investors at the Roth Conference in March, where interim CEO Philippe Calais also participated in a panel entitled "Why Knowing NASH as a Generalist Can Make You Rich in 2019."
During the first quarter and more recently, CohBar’s founders, Dr. Pinchas Cohen and Dr. Nir Barzilai, continued to be recognized as international leaders in the study of aging, age-related diseases and mitochondrial science.

Dr. Cohen delivered a presentation entitled "Novel Aspects of Mitochondrial Biology and its Implications for Healthy Aging" at the International Conference on Frailty and Sarcopenia Research in Miami Beach, Florida, in February 2019. More recently, in April, Dr. Cohen co-authored an article published in Mitochondrion entitled "Effects of air pollution on mitochondrial function, mitochondrial DNA methylation, and mitochondrial peptide expression."

Dr. Barzilai delivered keynote lectures at Unfolding Aging, in March 2019, in Berlin, Germany, and at the National Medical Research Council, in April 2019, in Singapore. He also lectured about diabetes and aging at the National Medical Research Council, in April 2019, in Singapore, and at the Wellness Summit in March 2019, at Rockefeller University in New York; and delivered the Raining Grant Annual Lectureship at the University of Minnesota in April 2019. During the quarter, Dr. Barzilai co-authored 3 published papers including "Interrogating the Genetic Determinants of Tourette’s Syndrome and Other Tic Disorders Through Genome-Wide Association Studies," in The American Journal of Psychiatry, "APOE Alleles and Extreme Human Longevity," in the Journal of Gerontology, and "Models and Studies of Aging: Executive Summary of a Report from the U13 Conference Series," in The Journal of American Geriatrics.
First Quarter 2019 Financial Highlights

Cash and Investments. CohBar had cash and investments of $19,531,260 on March 31, 2019, compared to $22,182,768 on December 31, 2018.

R&D Expenses. Research and development expenses were $1,371,848 in the three months ended March 31, 2019, compared to $2,680,983 in the prior year quarter. The decrease was primarily due to the timing of certain preclinical and clinical costs incurred in the prior year period, and lower stock-based compensation costs, partially offset by an increase in costs associated with our research programs focused on the continuing development of peptides.

G&A Expenses. General and administrative expenses were $1,456,197 for the three months ended March 31, 2019, compared to $913,088 in the prior year quarter. The increase in general and administrative expenses was primarily due to stock-based compensation costs, recruiting costs, and increased director fees.

Net Loss. For the three months ended March 31, 2019, net loss was $2,920,584, or $0.07 per basic and diluted share, compared to a net loss of $3,586,585, or $0.09 per basic and diluted share, for the three months ended March 31, 2018.
First Quarter Investor Call and Slide Presentation:

Date: May 7, 2019
Time: 5:00 p.m. EDT (2:00 p.m. PDT)

Conference Audio

Dial-in U.S. and Canada: (800) 289-0438
Dial-in International: (323) 794-2423
Conference ID No.: 1071020
Slide Presentation

Go to www.webex.com, click on the ‘Join’ button and enter meeting number 924 256 010 and Password CWBR, or
Go to www.cohbar.com and click on Q1 2019 Shareholder Presentation at top of homepage.
We kindly request that you please call into the conference audio and log into WebEx approximately 10 minutes prior to the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 8:00 p.m. EDT on May 7, 2019, through 11:59 p.m. EDT on May 28, 2019. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 1071020. The audio replay along with the slide presentation will also be available on the homepage at www.cohbar.com from May 7, 2019 through May 28, 2019.

About CB4211

CohBar’s lead program is based on CB4211, a first-in-class mitochondria based therapeutic (MBT) that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide (MDP) which was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators and has been shown to play a significant role in the regulation of metabolism. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes a potential activity readout relevant to NASH and obesity. In November 2018, the company announced the temporary suspension of the trial to address mild but persistent injection site reactions, and announced the anticipated resumption of the clinical trial in May 2019. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is currently no approved treatment for the disease.

On May 7, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), reported its quarterly results for the first quarter ended March 31, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 7, 2019, View Source [SID1234535818]). BTI is a clinical-stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology.

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First Quarter 2019 and Recent Highlights:

(BXCL501)- Neuroscience Program-

·Completed dosing of multiple cohorts in Phase 1 pharmacokinetic (bioavailability) and safety study of BXCL501; data announcement expected in coming weeks

·Finalized formulation development and transitioned to automated process for manufacturing of BXCL501 thin film formulation for pivotal study

· Announced proof-of-concept data demonstrating high response rates from three independent Phase 1 studies of intravenously administered dexmedetomidine (Dex) for acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease / dementia and opioid withdrawal symptoms

(BXCL701)- Immuno-Oncology Program-

·Filed investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) on proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer along with our partners Pfizer and Merck KGaA, Darmstadt, Germany, and Nektar Therapeutics

·Filed a clinical trial application (CTA) with U.K. health authorities to advance global development of BXCL701 and pembrolizumab (Keytruda) in neuroendocrine prostate cancer (tNEPC)

· Multiple sites opened for tNEPC clinical trial and two sites selected for BXCL701 proof of mechanism study for previously opened INDs

·Presented positive preclinical data on combination of BXCL701 with OX40 agonist at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, showcasing synergistic anti-tumor activity and survival benefit in tumor models

Dr. Vimal Mehta, Chief Executive Officer of BTI said, "During the first quarter of 2019 a number of positive initiatives continued to drive the ongoing clinical advancement of our lead programs, BXCL501 and BXL701."

"We anticipate reporting data from our first-in-human pharmacokinetic (bioavailability) and safety study of BXCL501 in the coming weeks. The dosing of multiple cohorts has been successfully completed. We believe that these results will help lay the groundwork for launching future registration studies as well as supporting BTI’s first New Drug Application (NDA) expected to be filed with the FDA in 2020. Additionally, in order to support the Phase 2/3 registration studies planned to begin in second half of 2019, the process development for BXCL501 sublingual thin film has now been transitioned to an automated robotic aided platform. We intend to employ an automated and scalable manufacturing process for clinical and commercial supply. BXCL501 has demonstrated promising potential with intravenously administered Dex in Phase 1 trials for the acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease/ dementia and opioid withdrawal symptoms, where new effective therapies are desperately needed. We continue to believe that BXCL501 has the potential to be applied to a wide number of neuropsychiatric disorders and look forward to exploring opportunities as we advance development in our initial target indications.

We are pleased to report that we have filed an IND with the FDA for the proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer with our partners Pfizer and Merck KGaA and Nektar. The triple combination therapy can potentially be effective by activating both the innate and adaptive immunity in pancreatic cancer patients. We are diligently working with our partners to progress the development of this triple combination therapy. Additionally, our ongoing Phase 1b/2 US study of BXCL701 and pembrolizumab in tNEPC is open for enrollment in multiple clinical trial sites. With the goal of expanding the clinical development of this combination globally, we have filed a CTA with U.K. health authorities. Additionally, we have selected two sites for BXCL701 proof of mechanism study in pancreatic cancer patients.

In March of this year, we presented positive findings at the AACR (Free AACR Whitepaper) Annual Meeting from a preclinical study evaluating the combination of BXCL701 with an OX40 agonist. The findings demonstrated that the combination significantly elevated anti-cancer activity in tumor models compared to the control. These results also provide important validation on BXCL701’s ability to stimulate both innate and adaptive immunity and we expect to further investigate the applicability of this combination."

Dr. Mehta concluded, "We continued to strengthen our management team with the appointment of industry veteran, Dr. Pascal Borderies, as the Vice President, Commercial Development and Medical Affairs who is responsible for designing and executing BTI’s global commercialization and medical affairs strategy, including sales and marketing efforts. We remain intently focused on progressing our assets through clinical development throughout the remainder of the year. We believe our sound strategy, highly skilled leadership team and solid pipeline candidates make us well-positioned for continued growth in 2019 and beyond."

First Quarter 2019 Financial Results

BTI reported a net loss of $7.2 million for the first quarter of 2019, compared to a net loss of $4.3 million for the same period in 2018.

Research and development expenses were $5.6 million for the first quarter of 2019, as compared to $2.9 million for the same period in 2018. The increase was primarily due to a ramp-up of research and development costs, along with increased personnel expenses associated with BTI’s two main drug candidates.

General and administrative expenses were $1.7 million for the first quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses and costs associated with operating as a public company.

These results include approximately $682,000 in non-cash share -based compensation charges.

As of March 31, 2019, cash and cash equivalents totaled $36.3 million.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call please dial (888) 394-8218 (domestic) and (323) 794-2588 (international) and provide the passcode 9482739. A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The archived webcast will be available through June 7, 2019.

Upcoming investor conferences:

· UBS Global Healthcare Conference – May 20-22, 2019, New York City

· BXCL501 Investor Day – May 22, 2019, New York City

· BMO Capital Markets Prescription for Success Healthcare Conference – May 25, 2019, New York City

· Jefferies Global Healthcare Conference – June 4-7, 2019, New York City

About BXCL501:

BXCL501 is a first in class, sublingual film of dexmedetomidine, a selective alpha 2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism and using IV (intravenous) Dex has demonstrated anti-agitation effects in

both preclinical and clinical studies. There is precedent for FDA approval and reimbursement of a non-invasive therapy for the acute treatment of agitation in patients with schizophrenia and bipolar disease, evidenced by regulatory approval of Adasuve, an inhaled version of the antipsychotic loxapine.

About BXCL701:

BXCL701 is a first in class oral immunotherapy with dual mechanisms of action, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting fibroblast activation protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other IO agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. It is under development for tNEPC and pancreatic cancer.