On May 9, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported financial results for the first quarter ended March 31, 2019 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, MAY 9, 2019, View Source [SID1234536075]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The accomplishments and performance of UroGen during the first quarter have set the stage for a pivotal and exciting year ahead as we prepare for the potential approval and commercialization of UGN-101, our first investigational candidate. Our top priority remains completion of our rolling New Drug Application (NDA) for UGN-101 for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC) and, with our commercial preparations well underway, we are confident in our readiness to deliver on a strong launch in the first half of 2020," said Liz Barrett, President and Chief Executive Officer of UroGen. "Our company sees great possibilities for the RTGel platform within uro-oncology, especially around new therapeutic modalities. Our goal is to leverage that and continue to build a company with sustainable growth via a robust pipeline as well as assessing opportunities via external partnerships."

Recent Highlights

UGN-101 Clinical Development:

At the 114th American Urological Association (AUA) Annual Meeting in Chicago, Seth Lerner, M.D. delivered a presentation during the plenary session that highlighted the unmet need and potential for UGN-101 to change the treatment paradigm for patients with LG UTUC.

The updated analysis demonstrated that in the OLYMPUS intent-to-treat population, 71 patients had undergone primary disease evaluation (PDE) at the time of the analysis and 42 of the 71 patients (59 percent) achieved a complete response (CR). Forty-one patients entered follow-up. At the time of the analysis, 27 patients underwent a six-month evaluation, and 24 out of 27 patients (89 percent) have remained disease free at six months. The most common adverse events observed were urinary tract infection, ureteral narrowing and stricture formation. The majority of ureteral events were reported as mild to moderate and have resolved. Full Phase 3 data from the OLYMPUS trial is anticipated for 2H 2019.

The Company remains on track to complete its rolling NDA for UGN-101 in 2H 2019 and is planning for approval in 1H 2020. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC. Full data is planned for 2H 2019.

Pipeline Advancement:

UGN-102:

The Company continues to enroll patients in its Phase 2b OPTIMA II clinical trial of UGN-102 (mitomycin gel) for intravesical instillation as a first-line chemoablation agent in the treatment of patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC), a form of disease associated with a high risk of recurrence.

Initial data from the OPTIMA II trial of UGN-102 is expected in 2H 2019.

There are currently no drugs approved by the FDA as first-line treatment for LG NMIBC. UGN-102 represents a substantial opportunity in UroGen’s pipeline and has the potential to be a treatment option for up to approximately 80,000 patients for whom repetitive surgical resection via Transurethral Resection of Bladder Tumor (TURBT) remains the standard of care.

UGN-201:

UroGen is currently evaluating the optimal pathway to advance UGN-201, a TLR7/8 immunomodulatory agent for the treatment of high-grade bladder disease.

Commercial Preparations:

The Company continues to accelerate its pre-commercial activities and infrastructure build-out to support the anticipated U.S. approval and launch of UGN-101 targeted for 1H 2020. The focus is on building awareness of our RTGel technology and unmet needs in UTUC to support rapid adoption and seamless integration of UGN-101 into the urologist practice following regulatory approval.

A strong team of seven medical science liaisons (MSLs) have been strategically deployed across the U.S. to engage in scientific exchange and clinical support.

The Company launched www.UTUC.com, the first resource designed to address a void in the urology space by educating patients about UTUC and available treatment options.

Business Development:

UroGen recently entered into an agreement with Janssen Research & Development, LLC (Janssen) to conduct an early-stage feasibility evaluation in a therapeutic area of mutual interest. UroGen and Janssen will each conduct certain activities under the terms of the agreement.

First Quarter 2019 Financial Results; 2019 Guidance

As of March 31, 2019, cash and cash equivalents totaled $246.7 million. This includes net proceeds of approximately $161.4 million from a public offering of ordinary shares in January 2019.

Research and development expenses for the three months ended March 31, 2019 were $9.7 million, including non-cash share-based compensation expense of $2.3 million.

General and administrative expenses for the three months ended March 31, 2019 were $12.7 million, including non-cash share-based compensation expense of $5.1 million.

The Company reported a net loss of $21.4 million, or basic and diluted net loss per ordinary share of $1.11, for the three months ended March 31, 2019.

The 2019 financial guidance set forth during the Company’s year-end earnings call on February 28th remains the same based on current business goals and anticipated activities.

Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 am Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 48486174. An archive of the webcast will be available for two weeks on the Company’s website.

On May 9, 2019 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported financial results for the first quarter ended March 31, 2019 and recent business highlights (Press release, Magenta Therapeutics, MAY 9, 2019, View Source [SID1234536074]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In 2019 we are continuing to advance our portfolio of programs toward our vision of curing more patients with autoimmune diseases, blood cancers and genetic diseases. This momentum was reflected in the recent start of our Phase 1 study of MGTA-145, our first-line therapy for stem cell mobilization and second clinical program, as well as in the extended evidence of disease benefit we see in our Phase 2 study of our MGTA-456 cell therapy in patients with inherited metabolic disorders," said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. "We are positioned to build on this momentum through additional important milestones across each of our programs and to deliver value for patients and shareholders."

Upcoming Anticipated Milestones:

The Company plans to achieve the following key milestones in 2019:

Present preclinical data on C100 anti-CD45 targeted conditioning program in autoimmune disease and declare a development candidate

Present preclinical data on C200 anti-CD117 targeted conditioning program in gene therapy

Present clinical data from the Phase 1 study of MGTA-145

Present additional clinical data from the Phase 2 study of MGTA-456 in inherited metabolic disorders (IMDs)

Recent Business Highlights:

Dosed first subjects in Phase 1 clinical trial of MGTA-145 first-line stem cell mobilization product candidate: In April 2019, Magenta announced that it had dosed the first subjects in a Phase 1 study of MGTA-145. Magenta intends to develop MGTA-145 in autoimmune diseases, blood cancers and genetic diseases. The Phase 1 study will investigate the safety and tolerability of MGTA-145 alone and in combination with plerixafor in healthy volunteers and establish recommended Phase 2 doses. The study will also measure the number of hematopoietic stem cells in the blood after dosing with MGTA-145 alone and in combination with plerixafor. Magenta expects to present data from the study in the second half of 2019. Depending on the Phase 1 data, the Company plans to move MGTA-145 into a Phase 2 study in multiple myeloma and non-Hodgkin lymphoma in 2020.

Updated clinical data for MGTA-456 cell therapy showed continued signs of durable clinical benefit in patients with IMDs: Magenta presented updated data from the Phase 2 clinical study of MGTA-456 in patients with IMDs at the American Academy of Neurology (AAN) annual meeting in May 2019. Patients with cerebral adrenoleukodystrophy (cALD) treated with MGTA-456 in the study showed stable neurological function scores and persistent resolution of brain inflammation by MRI at 6 months post-transplant, suggesting that the progression of disease has been halted. Magenta expects to update these results in the second half of 2019.

Preclinical data on E478 stem cell gene therapy expansion program show significant increase in gene-modified stem cells: At the American Society of Gene and Cell Therapy annual meeting in May 2019, Magenta presented data showing that E478 increased the number of human hematopoietic stem cells modified with either CRISPR/Cas9 or lentiviral vector by 10-fold compared to standard culture methods. Magenta is developing E478 to achieve high doses of gene-modified stem cells for better outcomes in patients with genetic disorders, including sickle cell disease and beta-thalassemia, where gene editing or viral vector technologies are used to correct stem cells. Magenta intends to develop E478 in partnership with gene therapy companies.

Presented nine abstracts at Transplant and Cellular Therapies Conference: Magenta presented data covering the breadth of the Company’s integrated portfolio of programs at the Transplant and Cellular Therapy (TCT) annual meeting in February 2019.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2019, were $127.3 million compared to $142.6 million on December 31, 2018. In addition, earlier this week Magenta announced that it completed a public offering of common stock and raised gross proceeds of $64.8 million. Magenta anticipates that its cash, cash equivalents and marketable securities, including the proceeds from this recent financing, will be sufficient to fund operations and capital expenditures into the second half of 2021.

Research and Development Expenses: Research and development (R&D) expenses were $10.5 million in the first quarter of 2019, compared to $7.8 million in the first quarter of 2018. The increase was driven by investments related to the IND filing and clinical activities for MGTA-145, as well as the on-going clinical development of MGTA-456.

General and Administrative Expenses: General and administrative (G&A) expenses were $5.8 million for the first quarter of 2019, compared to $3.5 million for the first quarter in 2018. The increase was primarily due to increased personnel and facility costs associated with the growth of the Company.

Net Loss: Net loss was $14.8 million for the first quarter of 2019, compared to net loss of $11.2 million for the first quarter of 2018.

On May 9, 2019 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the first quarter of 2019 and recent business highlights (Press release, Ionis Pharmaceuticals, MAY 9, 2019, View Source [SID1234536073]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our strong first quarter results put us on track to achieve our 2019 goals. We added commercial revenue from the first full quarter of the TEGSEDI launch to our growing commercial revenue from SPINRAZA. WAYLIVRA is now our third antisense medicine approved in just over two years, and we look forward to launching in Europe next quarter through our affiliate, Akcea," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer of Ionis. "This week, data presented from our medicines targeting Huntington’s disease and SOD1-ALS once again demonstrate the potential for our antisense technology to provide benefit in disease measures for patients with serious and previously untreatable diseases. Both medicines are in Phase 3 clinical trials with potential to support rapid paths to patients. Novartis licensed our most advanced LICA medicine, AKCEA-APO(a)-LRx, targeting the millions of patients worldwide with Lp(a)-driven cardiovascular disease. Novartis’ decision to advance AKCEA-APO(a)-LRx into a Phase 3 cardiovascular outcomes study further validates the potential of our rapidly expanding LICA pipeline to treat a broad range of diseases, including those for large patient populations. We plan to advance our next LICA medicine, AKCEA-TTR-LRx targeting TTR amyloidosis, into Phase 3 development in the second half of this year. We believe our accomplishments in the first quarter of 2019 position us for continued success. The power of our efficient technology and business strategy give us confidence that we can continue to deliver sustainable financial growth while aggressively investing in our commercial medicines and advancing our pipeline and our technology."

First Quarter 2019 Financial Results and Highlights

Revenues more than doubled compared to Q1 2018

Total revenue was $297 million compared to $144 million in Q1 2018.

Commercial revenue from SPINRAZA (nusinersen) was $60 million compared to $41 million in Q1 2018.

TEGSEDI (inotersen) product sales were $7 million in its first full quarter on the market and $9 million since launching in Q4 2018.

R&D revenue included $150 million from Novartis for its license of AKCEA-APO(a)-LRx and $35 million from Roche when it enrolled the first patient in the Phase 3 study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease.

Achieved substantial operating income and net income

Operating income and net income were $121 million and $84 million, respectively, compared to an operating loss and net loss of $3 million and $1 million, respectively, in Q1 2018, all on a GAAP basis.

Non-GAAP operating income and net income were $167 million and $126 million, respectively, compared to $25 million for both non-GAAP operating income and net income in Q1 2018.

Operating expenses increased in the first quarter primarily due to Ionis’ investment in commercializing TEGSEDI.

Substantial cash position grew to $2.3 billion enabling aggressive investment broadly across Ionis’ business

"We achieved another quarter of strong financial performance with both operating income and net income in the first quarter of 2019, substantially outperforming the same quarter in 2018. Our revenues in the first quarter were composed of growing commercial revenues from SPINRAZA royalties and TEGSEDI product sales, on top of substantial R&D revenues, driven in large part by the one-time $150 million license fee from Novartis for AKCEA-APO(a)-LRx. Looking ahead, we expect growing revenues this year from SPINRAZA, TEGSEDI and our partnered programs. And we also look forward to adding revenue following the EU launch of WAYLIVRA," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to achieve our 2019 financial guidance of net income and more than $100 million in operating income, both on a non-GAAP basis. Our goal is to continue to be profitable while investing in our commercial products, our pipeline and our technology."

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

Recent Business Highlights

SPINRAZA – the worldwide standard-of-care for the treatment of people with all forms of spinal muscular atrophy

Biogen reported worldwide sales of SPINRAZA of $518 million in the first quarter of 2019, a 42 percent increase compared to Q1 2018, driven primarily by increased penetration in existing markets, new country launches and continued uptake in the U.S. by children and adult patients.

There were more than 7,500 SMA patients from over 40 countries on SPINRAZA treatment at the end of the first quarter of 2019, including commercial patients and patients in the expanded access program and clinical trials.

SPINRAZA data from the ongoing NURTURE and SHINE open-label extension studies demonstrated continued durable efficacy and reinforced the safety profile of SPINRAZA in patients treated for up to 6 years, as presented by Biogen at the 2019 AAN Annual Meeting.

TEGSEDI – launch underway in multiple markets for the treatment of polyneuropathy of hereditary transthyretin amyloidosis (hATTR) in adult patients

TEGSEDI product sales were $7 million in its first full quarter on the market and $9 million since launching in Q4 2018.

TEGSEDI received a positive Final Evaluation Document (FED) from the National Institute for Health and Care Excellence (NICE) authorizing reimbursement for the treatment of patients with polyneuropathy due to hATTR amyloidosis in England.

Data presented at AAN from the TEGSEDI NEURO-TTR open-label extension study demonstrated long-term efficacy and safety in patients with hATTR.

WAYLIVRA (volanesorsen) – approved in the EU for the treatment of adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis

Akcea’s preparations to launch in the EU are underway, beginning in Germany in Q3 2019.

Launch in additional EU countries is planned in 2020.

Earned a $6 million milestone payment from PTC Therapeutics for the EU approval of WAYLIVRA.

Roche presented nine-month data from the ongoing Phase 1/2 open-label extension study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease at AAN, demonstrating continued and sustained reductions in mutant huntingtin protein with bi-monthly dosing.

Based on these data, Roche amended the dosing regimen in the Phase 3 study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease to replace the monthly dosing regimen with a tri-annual (every four months) dosing regimen.

Biogen presented data from the Phase 1/2 study of tofersen (IONIS-SOD1Rx) in ALS patients with SOD-1 mutations (SOD1-ALS) at AAN, demonstrating benefit in clinical measures of ALS disease progression after three months of treatment.

Tofersen is in a Phase 3 clinical study that could support a rapid path to patients.

Biogen is collaborating with regulators to further define the scope of the clinical data package required to support registration.

Ionis generated a $7.5 million milestone payment for advancing a new target for an unidentified neurological disease under its 2018 strategic neurology collaboration with Biogen.

Brett P. Monia, Ph.D., chief operating officer of Ionis was appointed to the Ionis board of directors.

Key Upcoming Data Events

Open-label extension study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease

Phase 1/2 study of AKCEA-TTR-LRx in healthy volunteers

BROADEN study of WAYLIVRA in patients with familial partial lipodystrophy (FPL)

Development program targeting FXI for the treatment of patients with clotting disorders

Development program for the treatment of patients with HBV infection

Phase 2 study of IONIS-GHR-LRx in patients with acromegaly

Phase 1 study of IONIS-ENAC-2.5Rx in healthy volunteers

In the first quarter of 2019, Ionis significantly increased both commercial revenue and R&D revenue. Commercial revenue from SPINRAZA royalties increased more than 45 percent. The Company also added growing TEGSEDI product sales to its commercial revenue.

Ionis’ R&D revenue substantially increased in the first quarter of 2019 due to the $150 million and $35 million the Company earned from Novartis and Roche, respectively.

In the second quarter of 2019, Alnylam announced it licensed Ionis’ technology to Regeneron. Once the transaction closes, Ionis expects to earn $20 million in sublicensing revenue.

Operating Expenses

Operating expenses for the first quarter of 2019 on a GAAP basis were $176 million and on a non-GAAP basis were $130 million. These amounts compare to GAAP operating expenses for the first quarter of 2018 of $147 million and non-GAAP operating expenses of $119 million. The increase in operating expenses was principally due to Ionis’ investments in the global launch of TEGSEDI.

Income Tax Expense

Ionis recorded income tax expense of $31 million for the three months ended March 31, 2019, compared to $15,000 for the same period in 2018. The increase in its income tax expense was primarily due to Ionis’ expectation that it will generate U.S. federal and state taxable income in 2019. Ionis’ 2019 income tax expense has two components. The first component relates to federal income taxes. Ionis expects to utilize its deferred tax assets to offset its U.S. federal taxable income. The other component of Ionis’ income tax expense relates to the estimated cash taxes it will pay for its state income taxes. Although Ionis is recording the expense for its state income taxes in 2019, Ionis will not have to make the majority of the payment for this liability until the first quarter of 2020.

Net (Income) Loss Attributable to Noncontrolling Interest in Akcea

At March 31, 2019, Ionis owned approximately 76 percent of Akcea. The shares of Akcea third parties own represent an interest in Akcea’s equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis’ consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other owners of Akcea’s common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations. Ionis’ net income attributable to noncontrolling interest in Akcea for the first quarter of 2019 was $6 million, compared to a net loss attributable to noncontrolling interest in Akcea of $9 million for the same period in 2018.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported net income attributable to Ionis’ common stockholders of $84 million for the first quarter of 2019 compared to a net loss of $1 million for the same period in 2018, both on a GAAP basis. On a non-GAAP basis, Ionis reported net income attributable to Ionis’ common stockholders of $126 million for the first quarter of 2019 compared to $25 million for the same period in 2018. The increase was primarily due to increases in revenue.

For the first quarter of 2019, basic and diluted net income per share were $0.63 and $0.62, respectively, compared to basic and diluted net loss per share of $0.01 for the same period in 2018. All amounts are on a GAAP basis.

Balance Sheet

Ionis added to its strong balance sheet, ending the first quarter of 2019 with cash, cash equivalents and short-term investments of $2.3 billion, compared to $2.1 billion at December 31, 2018.

Webcast and Conference Call

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 877-443-5662 or access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time.

On May 9, 2019 Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2019 (Press release, Acceleron Pharma, MAY 9, 2019, View Source [SID1234536072]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With the submission of marketing applications for luspatercept in the U.S. and E.U. last month, we and our global collaboration partner, Celgene, are excited about the potential to bring a new therapy to patients with myelodysplastic syndromes and beta-thalassemia within the next year," said Habib Dable, President and Chief Executive Officer of Acceleron. "At the same time, our pulmonary program remains on track, with enrollment ongoing in two Phase 2 trials of sotatercept in PAH, and we are anticipating topline results from the placebo-controlled part of the Phase 2 trial of our locally-acting muscle agent, ACE-083, in FSHD during the second half of this year."

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid maturation agent (EMA) designed to address a late-stage erythroid maturation defect that results in chronic anemia and the need for regular red blood cell transfusions in adults with serious hematologic diseases. Luspatercept is part of the global collaboration between Acceleron and Celgene.

Celgene recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept in patients with MDS- and beta-thalassemia-associated anemia based on the safety and efficacy results of the pivotal Phase 3 studies MEDALIST and BELIEVE.

The Companies expect to announce preliminary topline results from the Phase 2 trial of luspatercept in patients with MF in the second half of 2019.

Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia, with preliminary results expected from the BEYOND trial in 2020.

Neuromuscular Disease

ACE-083: Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)
ACE-083 is an investigational locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with FSHD were highlighted in an encore presentation at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in April 2019.

1
xlrnlogoa26.gif

Preliminary results from Part 2 of the Phase 2 trial in patients with FSHD are expected in the second half of 2019.

Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with CMT will be highlighted in a platform presentation at the American Academy of Neurology (AAN) 71st Annual Meeting on May 10, 2019.

Enrollment is ongoing in Part 2 of the Phase 2 trial in patients with CMT, with preliminary results expected in the first quarter of 2020.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure.

Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with topline results expected in the first half of 2020.

Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

A preclinical abstract of sotatercept in PAH has been accepted for presentation at the American Thoracic Society (ATS) 2019 International Conference on May 21, 2019.

Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2019 were $513.1 million. As of December 31, 2018, the Company had cash, cash equivalents and investments of $291.3 million. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales.

Revenue – Collaboration revenue for the first quarter was $2.8 million. The revenue is all from the Company’s partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept.

Costs and Expenses – Total costs and expenses for the first quarter were $43.6 million. This includes R&D expenses of $32.8 million and G&A expenses of $10.8 million.

Net loss – The Company’s net loss for the first quarter ended March 31, 2019 was $38.1 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2019 financial results and provide an update on recent corporate activities on May 9, 2019, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2019 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On May 9, 2019 PDL BioPharma, Inc. ("PDL" or "the Company") (NASDAQ: PDLI) reported financial results for the three months ended March 31, 2019 (Press release, PDL BioPharma, MAY 9, 2019, View Source [SID1234536071]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

First Quarter and Recent Financial Highlights

Total revenues of $38.9 million.

GAAP net income of $6.7 million or $0.05 per diluted share.

Non-GAAP net income attributable to PDL’s shareholders of $11.9 million. A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 at the end of this news release.

Cash and cash equivalents of $366.3 million as of March 31, 2019.

Repurchased 13.1 million shares of common stock in the open market during the first quarter of 2019 for $44.4 million, or an average price of $3.38 per share.

Invested $30.0 million in Evofem Biosciences, Inc. in April 2019.

"This is a very exciting time at PDL as we report strong first quarter financial results while we consider expanding our strategic transaction with Evofem Biosciences, as announced on April 11," said Dominique Monnet, president and CEO of PDL. "The elements of this transaction fit with our commitment to creating shareholder value by entering into strategic collaborations with pharmaceutical companies with innovative products and technologies. Evofem Biosciences provides us with an attractive opportunity to make a contribution to women’s healthcare, which presents many unmet medical needs that have been largely underserved by large pharmaceutical companies. Evofem’s lead investigational drug product, Amphora, offers a novel non-hormonal approach to contraception for women. Additionally, we are confident that Evofem’s team has the talent, expertise and dedication to execute successfully its commercial plan for Amphora.

"Should we make the second $30 million tranche of our proposed investment in Evofem, our team at PDL would bring significant value to the Amphora launch by contributing our capital and expertise in commercializing products in the U.S. and internationally," he added.

First Quarter Revenue Highlights

Total revenues of $38.9 million included:

Product revenue of $26.7 million, which consisted of $20.0 million from the sales of our branded prescription medicine products Tekturna and Tekturna HCT in the U.S. and Rasilez and Rasilez HCT in the rest of the world and revenue generated from the sale of an authorized generic form of Tekturna in the United States (collectively, the Noden Products), and $6.7 million of product revenue from the LENSAR Laser System.

Product revenue from the Noden Products was $12.2 million in the U.S. and $7.8 million in the rest of the world.

Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $12.3 million, primarily related to the Assertio royalty asset.

Total revenues of $38.9 million, compared with $38.5 million for the first quarter of 2018.

Product revenue of $26.7 million, increased 14.4%, compared with $23.3 million for the prior-year period. The increase is primarily due to the initial inventory stocking related to the launch of an authorized generic form of Tekturna in the United States in March 2019.

PDL recognized $12.3 million in revenue from royalty rights – change in fair value, compared with $11.1 million in the prior-year period. The increase is related to higher royalties from the Assertio royalty asset.

PDL received $12.6 million in net cash royalties from its royalty rights in the first quarter of 2019.

Royalties from PDL’s licensees to the Queen et al. patents were less than $0.1 million in the first quarter of 2019, compared with $2.8 million for the first quarter of 2018 as royalties on the sales of Tysabri are nearing completion.

Interest revenue decreased by $0.7 million from the prior-year period due to CareView Communications not making its interest payment in the first quarter of 2019.

First Quarter Operating Expense Highlights

Operating expenses were $28.4 million, a $5.8 million decrease from $34.2 million for the first quarter of 2018. The variance was primarily a result of:

a $4.7 million decline in amortization expense for the Noden intangible assets as a result of the impairment recorded for these intangible assets in the second quarter of 2018,

a $1.2 million, or 10%, decline in general and administrative expenses primarily due to lower professional fees,

a $2.8 million, or 50%, decline in sales and marketing expenses, reflecting the cost savings from the change in our marketing strategy for the Noden Products,

offset by a $2.2 million increase in Noden Products and LENSAR cost of product revenue, due to higher sales in both segments,

a $0.6 million favorable adjustment to the fair value of the contingent consideration recorded in the first quarter of 2018 with no corresponding adjustment in the first quarter of 2019, and

higher research and development expenses in our Medical Devices segment.

Stock Repurchase Programs

In November 2018, PDL began repurchasing shares of its common stock pursuant to the $100.0 million share repurchase program. During the first quarter of 2019, the Company repurchased 13.1 million shares for an aggregate purchase price of $44.4 million, or an average cost of $3.38 per share, including trading commission.

Subsequent to the close of the first quarter of 2019, the Company repurchased 2.8 million shares at an average price of $3.77 per share, for a total of $10.4 million.

To date, the Company has repurchased 24.5 million shares for a total of $80.3 million in the $100.0 million program leaving $19.7 million available to be repurchased.

Since initiating its first stock repurchase program in March 2017, the Company has used $135.3 million to repurchase a total of 46.6 million shares of its common stock.

Other Financial Highlights

PDL had cash and cash equivalents of $366.3 million as of March 31, 2019, compared with cash and cash equivalents of $394.6 million as of December 31, 2018.

The reduction in cash and cash equivalents was primarily the result of common stock repurchases of $44.4 million, partially offset by the proceeds from operations and royalty rights.

Conference Call and Webcast Details

PDL will hold a conference call to discuss financial results and provide a business update at 4:30 p.m. Eastern time today. Slides to accompany the conference call will be available in the Investor Relations section of www.pdl.com.

To access the live conference call via phone, please dial 844-535-4071 from the U.S. and Canada or 706-679-2458 internationally. The conference ID is 1099595. A telephone replay will be available beginning approximately one hour after the call through one week following the call and may be accessed by dialing 855-859-2056 from the U.S. and Canada or 404-537-3406 internationally. The replay passcode is 1099595.

To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of www.pdl.com and select "Events & Presentations."