On November 6, 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 third quarter ended September 30, 2019 (Press release, Acceleron Pharma, NOV 6, 2019, View Source [SID1234550412]).

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"With the FDA’s decision on the luspatercept regulatory submission for the treatment of beta-thalassemia expected in less than one month, we are close to achieving a major company milestone-the potential approval for the first Acceleron-discovered medicine," said Habib Dable, President and Chief Executive Officer of Acceleron. "Alongside our global collaboration partner, Celgene, we are preparing for luspatercept’s potential commercial launch in the U.S. We also await the FDA’s decision on our BLA for the MDS indication in April 2020 and the European Medicines Agency’s decision on the MAA for both indications, which is expected in the second half of 2020. We continue to advance our ongoing clinical trials in first-line lower-risk MDS-, non-transfusion-dependent beta-thalassemia- and myelofibrosis-associated anemia. In addition, we are looking forward to presenting luspatercept updates on our Phase 3 MEDALIST and BELIEVE trials, as well as interim results from the ongoing Phase 2 myelofibrosis trial at the upcoming ASH (Free ASH Whitepaper) meeting in December."

Added Mr. Dable: "While our hematology program continues to grow, we are also advancing our two Acceleron-led clinical programs in pulmonary and neuromuscular disease. We anticipate reporting results in the first quarter of 2020 for both our PULSAR Phase 2 sotatercept trial in patients with PAH, and our ACE-083 trial in patients with CMT."

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid maturation agent 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.

The U.S. Food and Drug Administration’s (FDA) review of the Biologics License Application (BLA) for luspatercept is ongoing for the treatment of anemia in adult patients with very low- to intermediate-risk MDS, who have ring sideroblasts and require red blood cell (RBC) transfusions, and for the treatment of anemia in adult patients with beta-thalassemia, who require regular RBC transfusions.

The FDA’s target action date for the beta-thalassemia indication is December 4, 2019, and April 4, 2020 for the MDS indication.

The European Medicines Agency’s (EMA) review of the Marketing Authorization Application (MAA) for luspatercept for the treatment of anemia in adult patients with MDS or beta-thalassemia is ongoing. The EMA decision on the MAA is expected in the second half of 2020.

Six clinical abstracts of luspatercept have been accepted for presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando on December 7-10, 2019, including

Five clinical abstracts highlighting new and updated analyses from the MEDALIST and BELIEVE Phase 3 trials;

One clinical abstract including interim results from the ongoing Phase 2 trial in MF.

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Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve, lower-risk MDS.

The BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia is ongoing, with topline results expected by year-end 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 (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.

The FDA granted Orphan Drug Designation to sotatercept for the treatment of patients with PAH.

Topline results from the PULSAR Phase 2 trial in patients with PAH are expected in the first quarter of 2020.

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

Neuromuscular Disease

ACE-083: Charcot-Marie-Tooth Disease (CMT) and Facioscapulohumeral Muscular Dystrophy (FSHD)
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.

Topline results from Part 2 of the Phase 2 trial in patients with CMT are expected in the first quarter of 2020.

Clinical development of ACE-083 in patients with FSHD has been discontinued following the Phase 2 topline results.
Financial Results

Cash Position – Cash, cash equivalents and investments as of September 30, 2019 were $468.3 million, as compared to $291.3 million as of December 31, 2018.
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 – Revenue for the third quarter of 2019 was $4.2 million. This revenue was derived from the Company’s collaboration partnership with Celgene and is largely related to expenses incurred by the Company in support of luspatercept.

Costs and Expenses – Total costs and expenses for the third quarter of 2019 were $53.1 million. This includes R&D expenses of $37.6 million and G&A expenses of $15.5 million.

Net Loss – The Company’s net loss for the third quarter of 2019 was $45.4 million.

Conference Call and Webcast

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The Company will host a webcast and conference call to discuss its third quarter 2019 financial results and provide an update on recent corporate activities on November 6, 2019, at 10:00 a.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at 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 Third Quarter 2019 Earnings Call."

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

On November 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results and business highlights from Q3 2019 (Press release, Adaptimmune, NOV 6, 2019, View Source [SID1234550411]).

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"The sarcoma community has been very supportive following the ESMO (Free ESMO Whitepaper) presentation in which we showed a clear benefit for ADP-A2M4. This increases our confidence that we can accelerate enrollment in SPEARHEAD-1 as we progress toward commercialization in 2022," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We continue to see evidence of antitumor activity with our SPEAR T-cells across other indications. We are executing with our ADP-A2AFP trial, now dosing in Cohort 3, as well as our next-gen SURPASS trial, and we plan on updating data throughout 2020. We continue to prioritize resources to enable us to deliver data from our ongoing trials."

COMMERCIAL READINESSS FOR ADP-A2M4 IN SARCOMA WITH AIM TO LAUNCH IN 2022

·Data presented at ESMO (Free ESMO Whitepaper) 2019 demonstrating a clear benefit of ADP-A2M4 for patients with synovial sarcoma showing:

oOverall response rate of 58% with 7 out of 12 evaluable patients with clinical responses

oDisease control rate (best overall responses of partial response or stable disease) of 92% with 11 out of 12 evaluable patients showing clinical benefit

These data will be updated at CTOS on November 16th

·Enrolling patients in SPEARHEAD-1 (Phase 2 trial in synovial sarcoma and myxoid/ round cell liposarcoma) across several leading clinical trial centers

·US FDA granted Orphan Drug Designation to SPEAR T-cells targeting MAGE-A4 (the ADP-A2M4 program) for the treatment of soft tissue sarcomas

·Produced first in-house GMP viral vector batch, augmenting the Company’s external supply with internal manufacturing capabilities

FOCUSED ON EXECUTION IN OTHER CLINICAL TRIALS

·Dosing in the SURPASS trial with ADP-A2M4CD8 – the first next-generation trial at several clinical trial centers with data updates anticipated in 1H 2020

oPreclinical data show that these next-generation SPEAR T-cells may improve long term T-cell function as well as antitumor activity

·Enrolling in Cohort 3 in the ADP-A2AFP trial in liver cancer at target doses of 5 billion SPEAR T-cells (range 1.2 to 6 billion), after Safety Review Committee endorsed dose escalation

Continuing enrollment in the radiation sub-study of the Phase 1 ADP-A2M4 trial

·Based on emerging translational data to be presented at SITC (Free SITC Whitepaper) on November 8th, will initiate a clinical trial combining ADP-A2M4 with a PD-1 pathway inhibitor in 2020

PRECLINICAL PROGRESS

·Continued progress with stem-cell derived allogeneic program, which is applicable to all T-cells (including both CAR-T and TCR T-cells) with data updates next year

·Initiated collaboration with Noile-Immune to develop further next-generation products

OTHER CORPORATE NEWS

·Michael Garone appointed as Interim Chief Financial Officer

·The Company is recruiting for a permanent Chief Medical Officer and Chief Financial Officer

Financial Results for the three-month period ended September 30, 2019

·Cash / liquidity position: As of September 30, 2019, Adaptimmune had cash and cash equivalents of $39.4 million and Total Liquidity1 of $102.9 million. The Company also received $15.8 million for UK R&D Tax Credits in October 2019.

·Revenue: Revenue for the three and nine months ended September 30, 2019 was $0.2 million and $0.4 million respectively, compared to $40.8 million and $58.0 million for the same periods in 2018. The revenue in the three and nine months ended 2019 is due to the commencement of development under the third target nominated by GSK under the Collaboration and License Agreement, whereas the development and license revenue for the same periods in 2018 was recognized due to the performance under the NY-ESO transition program and the PRAME development plan, which were completed in 2018.

·Research and development (R&D) expenses: R&D expenses for the three and nine months ended September 30, 2019 were $29.6 million and $77.1 million, respectively, compared to $23.5 million and $75.5 million for the same periods of 2018. The three and nine month periods ended September 30, 2019 include the impact of recognized purchase commitments for clinical materials of $5.0 million, and in process research and development payments relating to the collaboration agreements with Alpine Immune Sciences, Inc of $2.0 million in May 2019 and Noile-Immune Biotech, Inc of $2.5 million in August 2019. These increases were partially offset by a decrease in share-based compensation expense due to option forfeitures in both periods, an increase in reimbursements for research and development tax and expenditure credits in both periods, and, during the nine months ended September 30, 2019, a reduction in expenditure associated with the NY-ESO program, which was transferred to GSK on July 23, 2018.

·General and administrative (G&A) expenses: G&A expenses for the three and nine months ended September 30, 2019 were $10.7 million and $32.7 million respectively, compared to $10.3 million and $32.8 million for the same periods of 2018.

·Other income (expense), net: Other income (expense), net for the three and nine months ended September 30, 2019 was income of $0.3 million and an expense of $0.6 million respectively, compared to expenses of $2.2 million and $10.5 million for the same periods of 2018. Other income (expense), net primarily comprises unrealized foreign exchange losses, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities. Management determined that, effective from July 1, 2019, an intercompany loan was of a long-term investment nature and therefore, from that date, foreign exchange differences on this intercompany loan have subsequently been reported within other comprehensive income (loss).

·Net (loss) income: Net (loss) income attributable to holders of the Company’s ordinary shares for the three and nine month periods ended September 30, 2019 was losses of $39.3 million and $107.8 million respectively, and $(0.06) and $(0.17) per ordinary share respectively, compared to income of $5.2 million and a loss of $59.3 million respectively, and $0.01 and $(0.10) per ordinary share respectively in the same periods of 2018.

1 Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

Financial guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations through the third quarter of 2020. As further detailed in the Company’s Quarterly Report on Form 10-Q for the fiscal quarter ended September 30, 2019, to be filed with the Securities and Exchange Commission following this earnings release, the Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date the financial statements are issued. The Company concluded that there are factors that raise substantial doubt about its ability to continue as a going concern for at least one year from the issuance of the September 30, 2019 quarterly condensed consolidated financial statements. In the immediate future, the Company plans to continue to prioritize and review ongoing clinical development projects and costs with the aim of focusing operations on the ADP-A2M4 SPEARHEAD-1, ADP-A2M4CD8 SURPASS, and ADP-A2AFP trials. The Company believes that a combination of targeted clinical progress together with accessing additional capital and/or cost reductions will enable the Company to continue as a going concern for the next twelve months. The Company’s financial results are presented as though it would continue as a going concern.

Conference Call and Webcast Information
The Company will host a live teleconference at 8:00 a.m. EST (1:00 p.m. GMT) today, November 6, 2019. The live webcast of the conference call will be available in the investor section of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, please dial (833) 652-5917 (U.S. or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (3997944).

On November 6, 2019 Arbutus Biopharma Corporation (Nasdaq: ABUS), a Hepatitis B Virus (HBV) therapeutic solutions company, reported its third quarter 2019 financial results and provides a corporate update (Press release, Arbutus Biopharma, NOV 6, 2019, View Source [SID1234550410]).

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"We remain committed to our mission of developing a portfolio of assets with differing mechanisms of action that we believe will form the basis for a functional cure of chronic Hepatitis B", said William Collier, Arbutus’ President and Chief Executive Officer. "Our current efforts are focused on completing the Phase 1a/b clinical trial of AB-729, rapidly selecting a next-generation capsid inhibitor for IND-enabling studies to replace our recently discontinued AB-506, evaluating our oral RNA destabilizer, AB-452, as well as next-generation compounds in this class, and research on compounds that inhibit PD-L1."

Recent Corporate Updates

AB-729

In July 2019, the Company initiated a single and multiple dose Phase 1a/1b clinical trial for AB-729, a subcutaneously delivered RNAi agent which has been shown in preclinical models to span all HBV transcripts, reduce all viral antigens, including hepatitis B surface antigen (HBsAg) expression, and inhibit HBV replication. In this trial, which is designed to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AB-729 in healthy volunteers and in subjects with chronic hepatitis B (CHB) infection, AB-729 will be dosed monthly.

Preliminary safety data in single-dose cohorts of healthy subjects and safety and efficacy data in single-dose cohorts of subjects with CHB infection are expected in the first quarter of 2020.

Capsid Inhibitors

In October 2019, Arbutus announced its decision to discontinue the clinical development of AB-506, an oral capsid inhibitor, in Phase 1a/1b clinical development for the treatment of CHB due to safety observations in a Phase 1a 28-day clinical trial in healthy volunteers. Arbutus intends to present results from the AB-506 Phase 1a/1b clinical trial program at the American Association for the Study of Liver Diseases meeting later this month.

Arbutus is evaluating a number of oral next-generation capsid inhibitor compounds with chemical scaffolding different from AB-506 that the Company believes have the potential to contribute to the inhibition of HBV replication as part of a combination regimen. The Company’s objective is to select one of several lead compounds for IND-enabling studies in December of this year.

AB 452

Arbutus remains committed to the development of oral RNA-destabilizers that have shown compelling anti-viral effects in multiple HBV preclinical models. AB-452, Arbutus’ lead oral RNA-destabilizer is being evaluated in a repeat 90-day preclinical safety study in two species before making a go/no-go decision. We expect that the results of this study will allow us to make that decision early in 2020. The Company is also continuing to advance back-up compounds with chemical scaffolding different from that of AB-452.

Early R&D Programs

Arbutus continues a focused discovery effort on follow-on compounds for its current HBV pipeline, including efforts to identify compounds potentially capable of reawakening patients’ HBV-specific immune response by inhibiting PD-L1.

New Appointment to Arbutus’ Board of Directors

Andrew Cheng, M.D., Ph.D., was appointed to the Arbutus Board of Directors. Previously, Dr. Cheng spent nearly two decades at Gilead Sciences, Inc., where he most recently served as Chief Medical Officer and Executive Vice President. Dr. Cheng is currently President and Chief Executive Officer of Akero Therapeutics (Nasdaq: AKRO).

Cash Position and Cash Guidance

The Company had approximately $90.1 million in cash and cash equivalents as of September 30, 2019. The discontinuation of the AB-506 development program is anticipated to reduce cash burn in the short term and the Company believes its existing cash and cash equivalents balance is sufficient to fund operations into early 2021.

Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and short-term investments totaling $90.1 million as of September 30, 2019, as compared to $124.6 million as of December 31, 2018. The decreased cash balance was due primarily to the $57.7 million used in operating activities during the first nine months of 2019, partially offset by $18.5 million in net proceeds from the sale of a portion of its royalty entitlement on net sales of ONPATTRO in the third quarter of 2019 and $4.7 million of net proceeds from the issuance of shares under its ATM program. Included in the $57.7 million used in operating activities is a $5.9 million payment for the award rendered in the arbitration proceeding with the University of British Columbia in the third quarter of 2019.

Net Loss

Net loss attributable to common shares for the third quarter of 2019, including non-cash charges of $43.8 million related to the impairment of an in-process research and development ("IPR&D") intangible asset and $22.5 million for the impairment of goodwill described further below, was $85.3 million ($1.50 basic and diluted loss per common share) as compared to $27.1 million ($0.49 basic and diluted loss per common share) for the third quarter of 2018. Net loss attributable to common shares also included non-cash expense for the accrual of coupon on the Company’s convertible preferred shares of $2.8 million in the third quarter of 2019 and $2.6 million in the third quarter of 2018, as well as non-cash expense for a proportionate share of Genevant’s net losses of $3.5 million in the third quarter of 2019 and $2.8 million in the third quarter of 2018.

ONPATTRO Royalty Entitlement

Arbutus has a royalty entitlement on global net sales of ONPATTRO (Patisiran) for the lipid nanoparticle delivery (LNP) technology licensed by Arbutus to Alnylam Pharmaceuticals, Inc. (Alnylam) for this product. ONPATTRO is an RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis that has been approved by the U.S. Food and Drug Administration and the European Medical Agency. In July 2019, Arbutus sold this royalty entitlement to OCM IP Healthcare Portfolio LP, an affiliate of the Ontario Municipal Employees Retirement System (collectively, OMERS), effective as of January 1, 2019, for $20 million in gross proceeds before advisory fees. OMERS will retain this royalty entitlement until it has received $30 million in royalties, at which point 100% of this royalty entitlement will revert to Arbutus. OMERS has assumed the risk of collecting up to $30 million of future royalty payments from Alnylam and Arbutus is not obligated to reimburse OMERS if they fail to collect any such future royalties. Arbutus recognized the $20 million of gross proceeds from this transaction as a liability, net of transaction costs. The Company is amortizing the liability to non-cash interest expense and will continue to recognize the royalty revenue that Alnylam pays to OMERS as non-cash royalty revenue.

In addition to the royalty entitlement from the Alnylam LNP license agreement, Arbutus is also receiving a second, lower royalty entitlement on global net sales of ONPATTRO originating from a settlement agreement and subsequent license agreement with Acuitas Therapeutics. The royalty entitlement from Acuitas has been retained by Arbutus and is not part of the royalty entitlement sale to OMERS.

Operating Expenses

Research and development expenses were $17.7 million in the third quarter of 2019 compared to $16.6 million in the third quarter of 2018. Research and development expenses in the third quarter of 2019 included costs associated with the Company’s Phase 1a/1b clinical trial for its RNAi agent (AB-729), costs associated with the Company’s Phase 1a/1b clinical trial for its oral capsid inhibitor (AB-506) that was discontinued in October 2019, and toxicology studies for its HBV RNA Destabilizer (AB-452). The increase in research and development expenses was due primarily to increased spending in 2019 for the two Phase 1a/1b clinical trials for AB-729 and AB-506. General and administrative expenses were $3.3 million in the third quarter of 2019 compared to $2.6 million in the third quarter of 2018. The increase in general and administrative expenses was due primarily to increased stock compensation expense and an increase in insurance premiums.

In the third quarter of 2019, the Company also recorded a charge of $6.5 million related to an arbitration award from the Company’s arbitration with the University of British Columbia.

Impairment of IPR&D Intangible Assets and Goodwill

The Company has historically carried IPR&D and goodwill from its acquisition of technologies and business combination as assets. All acquired IPR&D intangible assets relate to the Company’s covalently closed circular DNA ("cccDNA") program. During the three months ended September 30, 2019, the Company recorded a $43.8 million non-cash impairment expense to reduce the carrying value of its IPR&D intangible assets to zero as of September 30, 2019. The Company also recognized a corresponding income tax benefit of $12.7 million related to the decrease in its deferred tax liability associated with the IPR&D intangible assets. The impairment was due to an indefinite delay in further development of the Company’s cccDNA program while the Company focuses on its other development programs.

Goodwill represents the excess of purchase price over the value assigned to the net tangible and identifiable intangible assets in connection with the business combination that formed Arbutus. For the third quarter of 2019, the Company assessed the changes in circumstances that occurred during the quarter to determine if it was more likely than not that the fair value of the Company was below its carrying amount. Due to a sustained decrease in the Company’s share price in recent months, the Company’s market capitalization was reduced below the book value of its net assets and the Company concluded that its fair value was below its carrying amount by an amount in excess of the carrying value

of the goodwill. As a result, the Company recorded a $22.5 million non-cash impairment expense to reduce the carrying value of its goodwill asset to zero as of September 30, 2019.

Equity Investment Loss in Genevant

As of September 30, 2019, the Company owned approximately 40% of the common equity of Genevant Sciences Ltd. (Genevant), a company launched with Roivant Sciences Ltd. in April 2018. Arbutus recorded a loss of $3.5 million in the third quarter of 2019 for its proportionate share of Genevant’s net loss. Financial results of Genevant are recorded on a one-quarter lag basis.

Outstanding Shares

The Company had 56,850,172 common shares issued and outstanding as of September 30, 2019. In addition, the Company had approximately 9.1 million stock options outstanding and 1.164 million convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into approximately 23 million common shares on October 18, 2021.

Conference Call Today

Arbutus will hold a conference call and webcast today, Wednesday, November 6, 2019 at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 7279188.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 7279188.

On November 6, 2019 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported that new preclinical data from its STING agonist program will be presented at the 34th annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, Trillium Therapeutics, NOV 6, 2019, View Source [SID1234550409]). The meeting will be held November 6-10 in National Harbor, MD. The poster presentation will highlight the anti-tumor activity of TTI-10001, a novel small molecule STING agonist, when delivered intravenously or orally. Details of the presentation are listed below.

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Title: "TTI-10001, a next generation small molecule STING agonist, demonstrates potent anti-tumor activity in mice following intravenous or oral administration"

Presenter: Bob Uger, Ph.D., Chief Scientific Officer, Trillium Therapeutics Inc.

Date and Time: November 9, 2019 from 8:00 a.m. – 8:30 p.m. ET

Location: Gaylord National Hotel & Convention Center

Abstract Number: P668

On November 6, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 6, 2019, View Source [SID1234550408]).

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"BioCryst is positioned for a transformational 2020, with the potential approval and launch of BCX7353 in the U.S., regulatory filings for BCX7353 in Japan and Europe, and data from our PNH proof of concept study with BCX9930 reading out in the first half of the year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We are also actively evaluating multiple approaches to add capital to the balance sheet by the end of 2019, as we did with the Japanese licensing agreement for BCX7353, which we announced earlier this week," Stonehouse added.

Upcoming Key Milestones

HAE Program – BCX7353

Submit a new drug application (NDA) for oral, once-daily BCX7353 for the prevention of hereditary angioedema (HAE) attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)

Submit a marketing authorization application for oral, once-daily BCX7353 for the prevention of HAE attacks with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and the European Medicines Agency (EMA) (Q1 2020)

Commence ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the treatment of acute HAE attacks, in 2020, pending the completion of interactions with regulators on the Phase 3 program and additional work on the acute oral formulation (2020)
Complement Oral Factor D Inhibitor Program – BCX9930

Report data from a proof of concept study in paroxysmal nocturnal hemoglobinuria (PNH) patients receiving oral BCX9930 (1H 2020)
ALK-2 Inhibitor Program – BCX9250

Continue ongoing Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of fibrodysplasia ossificans progressiva (FOP), in healthy subjects
Recent Corporate Developments

On November 6, 2019, the company provided a commercial update based on new 48-week clinical data from the APeX-2 and APeX-S trials, and detailed market research conducted with HAE patients, physicians who treat HAE and payors.

On November 5, 2019, the company announced it had licensed commercialization rights in Japan to Torii Pharmaceutical, Co. for BCX7353 for up to $42 million of upfront and potential milestone payments, including a $22 million upfront payment.
On November 1, 2019, the company announced it had begun enrollment of a Phase 1 trial of BCX9250, an oral ALK-2 inhibitor discovered and developed by BioCryst, for the treatment of FOP. The trial will evaluate the safety and tolerability and characterize the pharmacokinetic and pharmacodynamic profiles of BCX9250 in single and multiple ascending doses in healthy volunteers.
On November 1, 2019, the company announced results from a Phase 1 trial of BCX9930, an oral Factor D inhibitor discovered and developed by BioCryst, showing that BCX9930 was safe and generally well tolerated, and demonstrated rapid, sustained and >95% suppression of the alternative pathway of the complement system at 100 mg every 12 hours. Based on these results, the company is advancing the program into a proof of concept (PoC) study in PNH patients and plans to report data from the PoC study in the first half of 2020.
On September 26, 2019, the company announced the U.S. Department of Health and Human Services (HHS) had exercised its option to purchase an additional 10,000 doses of BioCryst’s approved antiviral influenza therapy, RAPIVAB (peramivir injection).
On September 23, 2019, the company announced it had appointed clinical rare disease expert, Helen Thackray, M.D., FAAP, to its board of directors.
Third Quarter 2019 Financial Results

For the three months ended September 30, 2019, total revenues were $1.8 million, compared to $1.5 million in the third quarter of 2018. The increase was primarily due to the recognition of $0.3 million of peramivir product sales to Shionogi & Co., Ltd., the company’s commercial partner in Japan.

Research and development (R&D) expenses for the third quarter of 2019 increased to $25.1 million from $22.0 million in the third quarter of 2018, primarily due to increased spending on our complement-mediated diseases programs, which entered Phase 1 clinical testing in June 2019.

Selling, general and administrative (SG&A) expenses for the third quarter of 2019 increased to $11.7 million, compared to $7.9 million in the third quarter of 2018. The increase was primarily due to increased spending on commercial activities and medical affairs to support the U.S. commercial launch of BCX7353 in 2020.

Interest expense was $3.0 million in the third quarter of 2019, compared to $2.3 million in the third quarter of 2018, and was primarily due to an increase in the outstanding balance of the company’s secured credit facility in February 2019.

Net loss for the third quarter of 2019 was $37.6 million, or $0.34 per share, compared to a net loss of $29.6 million, or $0.28 per share, for the third quarter of 2018.

Cash, cash equivalents and investments totaled $70.0 million at September 30, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Operating cash use for the third quarter of 2019 was $24.5 million. Net operating cash use for the first nine months of 2019 was $77.9 million, as compared to $70.7 million for the first nine months of 2018.

Financial Outlook

BioCryst continues to expect full year 2019 net operating cash use to be in the range of $105 to $130 million, and its operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:00 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 4891026. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 4891026.