On November 7, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended September 30, 2018 and provided a corporate update (Press release, Portola Pharmaceuticals, NOV 7, 2018, View Source;p=RssLanding&cat=news&id=2376001 [SID1234530994]).

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"I am excited to join Portola at such a pivotal moment in the Company’s history. Having just completed my first month in role, I am very encouraged by the progress and momentum we’ve achieved since prioritizing Andexxa, including our preparations for the potential approval of Generation 2 supply in the United States and in Europe," said Scott Garland, Portola’s president and chief executive officer. "The early feedback from our first full quarter of sales gives us confidence in the significant long-term potential for Andexxa, and we are now implementing our new focused strategy for Bevyxxa. I am also excited about the potential for cerdulatinib, which recently received Orphan Drug Designation. We have a number of critical milestones ahead that I expect we will achieve and we look forward to delivering our life-saving medications to more patients, and driving sustainable growth and value for shareholders."

Third Quarter 2018 Financial Results
Total revenue for the third quarter of 2018 was $14.2 million, compared with $3.8 million for the third quarter of 2017. This includes $7.7 million in product revenue for our first full quarter of Andexxa sales and $7.0 million in collaboration and license revenues. Bevyxxa product orders remained relatively flat in the third quarter, during which we recognized a net product loss for Bevyxxa of $552 thousand. This adjustment was primarily related to potential returns of initial launch quantities provided to wholesalers.

Total operating expenses for the third quarter of 2018 were $83.3 million, compared with $84.3 million for the same period in 2017. Total operating expenses for the third quarter of 2018 included $11.4 million in stock-based compensation expense, compared with $10.1 million for the same period in 2017.

Research and development expenses were $40.2 million for the third quarter of 2018, compared with $55.3 million for the third quarter of 2017. The decrease is driven primarily by the timing of manufacturing costs for Andexxa Generation 2 campaigns. Approximately $12 million, or 30 percent, of R&D expense for the quarter and $75 million, or 45 percent, for nine months year-to-date of total R&D expense was related to Andexxa manufacturing.

Selling, general and administrative expenses for the third quarter of 2018 were $38.8 million, compared with $28.9 million for the same period in 2017, reflecting the investment in our field force and marketing for our product launches.

For the third quarter of 2018, Portola reported a net loss of $71.3 million, or $1.08 net loss per share, compared with a net loss of $82.9 million, or $1.41 net loss per share, for the same period in 2017.

Cash, cash equivalents and investments at September 30, 2018 totaled $380.9 million, compared with $534.2 million as of December 31, 2017.

2018 Annual Financial Guidance
For the fiscal year 2018, Portola is updating its guidance for GAAP operating expenses, which are now expected to be between $355 million and $365 million, a decrease from the prior guidance range of between $390 million and $430 million, both including stock-based compensation. The updated guidance reflects the Company’s narrowed focus on a group of key hospitals for the Bevyxxa launch, as well as additional cost savings for the year.

Recent Achievements and Events

Appointed industry veteran Scott Garland as president and chief executive officer.
Submitted Prior Approval Supplement (PAS) to U.S. FDA for large-scale Generation 2 Andexxa manufacturing process; assigned a PDUFA date of December 31, 2018.
Transitioned commercial focus to the ongoing Andexxa U.S. launch under the Early Supply Program.
Secured New Technology Add-on Payment (NTAP) for Andexxa, which became effective as of October 1, 2018.
Implemented new strategy for Bevyxxa U.S. launch focused on establishing 10 Centers of Excellence to provide a model for driving broader adoption.
Received Orphan Drug Designation from the U.S. FDA for cerdulatinib for the treatment of peripheral T-cell lymphoma.
Eight abstracts presented at the European Society of Cardiology (ESC) meeting.
Upcoming Milestones

Anticipated broad launch of Andexxa upon U.S. FDA approval of Generation 2 supply.
Committee for Medicinal Products for Human Use (CHMP) opinion on andexanet alfa, with potential for European approval of andexanet alfa in the first half of 2019.
End-of-Phase 2 meeting and determination of regulatory path forward for cerdulatinib in Q1 2019.
New interim Phase 2a study results for cerdulatinib to be presented in an oral session at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Also accepted for presentation: outcomes-based research on the burden of hospital readmissions for venous thromboembolism among patients with cancer (oral) and two poster presentations on andexanet alfa.
Conference Call Details
Portola will host a conference call today, Wednesday, November 7, 2018, at 4:30 p.m. ET, during which time management will discuss the third quarter 2018 financial results, updates on the U.S. launches of Andexxa and Bevyxxa, and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the U.S. and Canada or 1 (765) 507-2588 internationally and using the passcode 3387315. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

On November 7, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported that it will present a poster highlighting data demonstrating the potential of sd-rxRNA to improve NK cell potency in adoptive cell transfer (ACT) at SITC (Free SITC Whitepaper) 2018, the 33rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, RXi Pharmaceuticals, NOV 7, 2018, View Source [SID1234531026]).

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The poster, titled "sd-rxRNA to Enhance NK Cell Activity for Adoptive Cell Transfer" (Poster #P258), will be available for viewing from 12:20–1:50 p.m. ET and 7:00–8:30 p.m. ET on Saturday, November 10, 2018.

The poster will also be available under the "Investors – Events and Presentations" section of the Company’s website, View Source, approximately one hour following the presentation.

SITC 2018 is being held November 7-11, 2018 at the Walter E. Washington Convention Center in Washington, DC.

On November 7, 2018 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), a research and clinical development oncology company focused on biomarker-defined cancers, reported its third quarter 2018 financial results for the period ended September 30, 2018 and provided a strategic update (Press release, Merrimack, NOV 7, 2018, View Source [SID1234531175]).

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"Following a comprehensive review of our drug candidate pipeline, we have determined that a corporate restructuring provides the best path forward to reduce operational costs and maximize value. Naturally, this step was the result of an extremely difficult decision and we regret its impact on the affected members of our team, to whom we remain grateful for their contributions to Merrimack," said Richard Peters, M.D., Ph.D., President and Chief Executive Officer. "Going forward, we remain committed to the efficient development of targeted therapies for biomarker-defined cancers, as we are now focused on our clinical development program for MM-310, for which we anticipate providing another safety update in Q1 2019, and our emerging preclinical candidates, MM-401 and MM-201."

Strategic Update

The Company conducted an internal review of its pipeline, the results of which are outlined below:

Corporate: Reflective of its updated development plans, Merrimack is initiating a corporate restructuring intended to maximize value and preserve its ability to capture outstanding milestones from Ipsen, which the Company intends to pass through to shareholders, net of any taxes owed and subject to there being sufficient surplus at that time. This restructuring includes a workforce reduction of approximately 60%, including the elimination of all open positions, to be initiated immediately and completed by February 2019. This restructuring, together with other restructuring and cost cutting measures that the Company could implement in the future, provide the Company with the potential to extend its cash runway into at least the second half of 2022. In parallel, Merrimack has retained external advisors to explore strategic alternatives.

MM-121: Based on the results of the interim analysis of its randomized Phase 2 SHERLOC study that were announced on October 19, 2018, Merrimack is discontinuing development of all ongoing MM-121 programs, including terminating the SHERBOC study, its companion Phase 2 clinical trial evaluating MM-121 in metastatic breast cancer.

MM-310: After treating the first 14 patients in the first dose-escalating regimen in Merrimack’s Phase 1 clinical trial of MM-310 in solid tumors, the study has been amended to test an alternative dosing schedule. Early data from the every three week dosing schedule regimen showed signs of encouraging antitumor activity in four patients, including two non-small cell lung cancer patients, one soft tissue sarcoma patient and one ovarian cancer patient who additionally had a 70% reduction of CA-125, an associated tumor marker. However, emerging cumulative grade 3 peripheral neuropathy following multiple cycles of treatment was observed in three patients. Pharmacokinetic and preclinical data indicate that lengthening the time between dosing may improve tolerability of MM-310. Patients are now being screened under an every four week schedule amendment and will be dosed starting at the highest dose level reached in the prior regimen. Merrimack plans to provide an additional safety update from the study in Q1 2019.

Preclinical Programs: Merrimack is narrowing the scope of its preclinical efforts to prudently advance its two most promising programs, which are MM-401, its previously undisclosed immuno-oncology program, an agonistic antibody targeting a novel immuno-oncology target, TNFR2, and MM-201, a highly stabilized agonist-Fc fusion protein targeting death receptors 4 and 5. The Company will feature data from the MM-401 program at the 30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on November 14, 2018 in a poster titled, "Targeting TNFR2 – A Key Regulator of the Tumor Immunosuppressive Microenvironment," which highlights robust responses to MM-401 as a monotherapy in multiple mouse tumor models, including in PD-1 antibody-resistant tumors, as well as in combination with checkpoint inhibition.

Third Quarter 2018 Financial Results

In the third quarter and more recently, Merrimack received three ONIVYDE-related milestone payments:

$18.0 million resulting from the sale of ONIVYDE in two additional major European countries, which was received and announced in August 2018;

$5.0 million resulting from the sale of ONIVYDE in the first major non-European, non-Asian country, which was received and announced in September 2018; and

$5.0 million of the $10.0 million milestone for the first patient dosed in a pivotal clinical trial of ONIVYDE in an indication other than pancreatic cancer, which was received in October 2018, subsequent to the end of the third quarter. Merrimack received $5.0 million triggered by the commencement of a multi-part study that Ipsen and Servier are conducting in small cell lung cancer (SCLC). The remaining $5.0 million would be paid if and when a decision is made to progress to the randomized part of the study focused on efficacy.

The following summarizes Merrimack’s financial results for the quarter ended September 30, 2018:

Research and development expenses for the third quarter ended September 30, 2018 were $13.0 million, compared to $13.6 million for the comparable period of 2017. Research and development spending for the third quarter of 2018 was lower versus the comparable period in 2017 primarily due to the phasing of clinical development programs;

General and administrative expenses for the third quarter ended September 30, 2018 were $3.8 million, compared to $3.4 million for the comparable period of 2017. General and administrative spending for the third quarter of 2018 was higher versus the comparable period in 2017 primarily due to the timing of external corporate expenses;

Net loss for the third quarter ended September 30, 2018 from continuing operations was $12.3 million, or $0.92 per share, compared to a net loss of $5.4 million from continuing operations, or $0.40 per share, for the comparable period of 2017. The comparable period of 2017 included a one-time non-cash gain of approximately $10.8 million related to the deconsolidation of a subsidiary; and

As of September 30, 2018, Merrimack had 13.3 million shares of common stock, $0.01 par value per share, outstanding.

Financial Outlook

As a result of the corporate restructuring, Merrimack believes that its cash, cash equivalents and marketable securities of $84.8 million as of September 30, 2018, plus the $5.0 million ONIVYDE milestone received in October but excluding any potential additional milestone payments, together with other restructuring and cost cutting measures that Merrimack could implement in the future, provide Merrimack with the potential to fund its operations into at least the second half of 2022.

Merrimack remains eligible to receive additional milestone payments resulting from the Company’s asset sale to Ipsen in 2017:

Merrimack is entitled to receive up to $5.0 million in milestone payments triggered by Ipsen’s and Servier’s decision to progress their ongoing multi-part clinical trial evaluating ONIVYDE in SCLC into the randomized part of the study focused on efficacy. This milestone payment is excluded from the Company’s cash runway guidance until achieved; and

Merrimack is entitled to receive an aggregate of $450.0 million in regulatory-based milestones from Ipsen, which Merrimack has said it expects to pass through to stockholders, net of any taxes owed and subject to there being sufficient surplus at that time, consisting of:

$225.0 million upon approval by the FDA of ONIVYDE for the first-line treatment of metastatic adenocarcinoma of the pancreas, subject to certain conditions;

$150.0 million upon approval by the FDA of ONIVYDE for the treatment of small cell lung cancer after failure of first-line chemotherapy; and

$75.0 million upon approval by the FDA of ONIVYDE for an additional indication unrelated to those described above.

Conference Call and Webcast

Merrimack will host a live conference call and webcast today, Wednesday, November 7, 2018 at 8:30 am ET, to provide an update on the Company, including the results of its portfolio review, and a summary of these financial results.

Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 4685885. A listen-only webcast of the call can be accessed in the Investors section of Merrimack’s website, investors.merrimack.com, and a replay of the call will be archived there for six weeks following the call.

On November 7, 2018 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported financial results for the third quarter ending September 30, 2018 and provided a business update (Press release, Savara, NOV 7, 2018, View Source [SID1234530912]).

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"We are creating a differentiated orphan lung disease company through a portfolio of investigational programs addressing significant unmet need in rare respiratory diseases," said Rob Neville, Chief Executive Officer, Savara. "This past quarter’s achievements demonstrate considerable progress with our lead candidate, Molgradex, and in the coming year we look forward to numerous important data read-outs from our three clinical studies, two of which are pivotal. Additionally, we expect Savara’s pipeline, buoyed by indication expansion and product acquisitions, to facilitate sustainable growth now and in the future."

Recent Developments and Upcoming Highlights

Molgradex for autoimmune pulmonary alveolar proteinosis (aPAP)

Completed enrollment of 139 patients in the IMPALA study, a global, pivotal Phase 3 clinical study evaluating Molgradex, an inhaled formulation of granulocyte-macrophage colony-stimulating factory (GM-CSF) for the treatment of aPAP.
Expect top line results from the IMPALA study in Q2 2019. Positive results would facilitate the submission of a Biologic License Application in the first half of 2020, with an anticipated commercial launch in the U.S. and EU in 2020 or early 2021.
Continue active enrollment in IMPALA-X, an open-label, multicenter extension study to determine the long-term safety and utilization of Molgradex in patients with aPAP.
Announced a partnership with the PAP Foundation to support their efforts to unite, educate and assist the PAP patient community, including work to further expand the PAP patient registry.
Molgradex for nontuberculous mycobacterial (NTM) lung infection

Completed enrollment of 32 patients in the OPTIMA study, a Phase 2a clinical study evaluating Molgradex for the treatment of NTM lung infection.
Expect interim results from the OPTIMA study in Q4 2018.
Anticipate top line results from the OPTIMA study in Q2 2019.
The Investigational New Drug application for Molgradex in cystic fibrosis (CF)-affected individuals with chronic NTM lung infection has been accepted by the U.S. Food and Drug Administration. Savara expects to initiate a Phase 2a study of Molgradex in CF subjects with NTM lung infection in Q1 2019.
Completed license agreement with Mayo Clinic, enabling inclusion of Mayo clinical data in Savara’s patent applications related to NTM.
AeroVanc

Continue patient enrollment in the AVAIL study, a pivotal, global Phase 3 clinical study of AeroVanc, an inhaled vancomycin hydrochloride powder for the treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. At the end of Q3, patient enrollment was at 126 out of a target of 200.
Continue to target completion of patient enrollment in the AVAIL study in Q1 2019, with top line data in H2 2019.
Exploratory Pipeline

Expect to announce the initial indication for the Phase 2-ready aerosolized amikacin/fosfomycin proprietary combination antibiotic early in 2019, with an anticipated study-start later in 2019.
Financials

Successfully closed a public offering at the end of July with net proceeds to Savara of approximately $45.8 million.
Third Quarter Financial Results
Savara’s net loss attributable to common stockholders for the three months ended September 30, 2018 was $12.6 million, or $(0.37) per share, compared with a net loss attributable to common stockholders of $6.8 million, or $(0.28) per share, for the three months ended September 30, 2017.

Research and development expenses were $9.5 million for the three months ended September 30, 2018, compared with $5.0 million for the three months ended September 30, 2017. This increase was primarily due $2.5 million in additional expenses associated with AeroVanc Phase 3 study activities and $2.5 million in development costs of Molgradex, including the expansion of the aPAP study in the U.S. and costs associated with the Phase 2 NTM study. Conversely, the total research and development costs for the three months ended September 30, 2017 included approximately $0.4 million related to the Aironite program, which was assumed in the merger with Mast Therapeutics, Inc. in April 2017 and subsequently terminated in the first quarter of 2018.

General and administrative expenses for the three months ended September 30, 2018 were $3.1 million, compared with $1.5 million for the three months ended September 30, 2017. This increase was primarily due to $1.5 million additional costs related to personnel. The remaining increase in expense was associated with continued legal and accounting requirements for a public company.

Other income of $0.1 million was recognized for the three months ended September 30, 2018 as compared to other expense of $0.4 million for the three months ended September 30, 2017. The change was primarily due to additional interest income attributable to an increased balance maintained in our short-term investments for the three months ended September 30, 2018, as compared to that for the three months ended September 30, 2017.

As of September 30, 2018, Savara had a debt balance of approximately $15.0 million and had cash, cash equivalents and short-term investments of approximately $112.0 million.

Conference Call and Webcast
Savara will hold a conference call today beginning at 5:30 PM Eastern Time/4:30 PM Central Time to provide a business update. Shareholders and other interested parties may access the conference call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and request the "Savara Inc." call. A live webcast of the conference call will be available online in the Investors section of Savara’s website at View Source A replay of the webcast will be available on Savara’s website for 30 days, and a telephone replay will be available through November 12, 2018 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10125683

On November 7, 2018 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at 9:10 a.m. MST (8:10 a.m. PST, 11:10 a.m. EST) on Wednesday, November 14, at the Credit Suisse 27th Annual Healthcare Conference (Press release, Puma Biotechnology, NOV 7, 2018, View Source [SID1234530948]). The conference will be held at The Phoenician Resort in Scottsdale, Arizona.

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A live webcast of the presentation will be available on the Company’s website at www.pumabiotechnology.com. The presentation will be archived on the website and available for 30 days.