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.

On November 7, 2018 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported that an overview of the company’s business strategy and commercial and development-stage programs will be given at the Jefferies 2018 London Healthcare Conference being held in London (Press release, Spectrum Pharmaceuticals, NOV 7, 2018, http://investor.sppirx.com/news-releases/news-release-details/spectrum-pharmaceuticals-present-corporate-update-jefferies-2 [SID1234530969]). The company presentation is on Wednesday, November 14, 2018, at 3:20 PM GMT.

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A live webcast of Spectrum’s presentation will be available at www.sppirx.com.

On November 7, 2018 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported financial results for the third quarter ended September 30, 2018 (Press release, Compugen, NOV 7, 2018, View Source [SID1234530995]).

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"The last few months have been highly productive for Compugen, with strong execution marked by multiple key accomplishments. In September, we dosed the first patient in our Phase 1 COM701 study, which continues to progress as planned. Shortly thereafter, we signed a clinical collaboration agreement with Bristol-Myers Squibb, providing for the supply of Opdivo for the combination arms of the trial, as well as establishing the opportunity to accelerate the clinical evaluation of COM701 through other combination studies," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "With Bayer’s first patient dosing in their Phase 1 study of BAY 1905254 this quarter, there are now two ongoing clinical trials addressing novel targets we discovered through computer prediction; further evidence of the power and value of our computational discovery platform."

"Our preclinical data demonstrate that PVRIG and TIGIT are the primary inhibitory components of the foundational immuno-oncology DNAM axis, and that PVRIG is a key missing piece for cancer immunotherapy approaches that involve solely targeting the TIGIT pathway in this axis. Our research demonstrates that targeting PVRIG will be necessary to maximize the anti-tumor activity of TIGIT inhibitors, with or without PD-1 inhibitors, in various cancer types and patient populations. This is supported by initial clinical data released by others that show encouraging but modest effects of anti-TIGIT antibodies in Phase 1 clinical trials."

"As the only anti-PVRIG drug candidate currently available for clinical testing, COM701 stands out in the crowded field of immuno-oncology and offers a compelling opportunity to counteract the inhibition of the DNAM axis and potentially address the significant unmet medical need of cancer patients who are relapsed or refractory to other immunotherapies. Moreover, this program illustrates our differentiated approach to drug discovery and development; one that is guided by three principals – a focus on new pathways aimed at addressing a significant unmet need, a science-driven approach to select optimal drug combinations; and a robust rationale and strategy for patient selection based on a deep scientific understanding of the new pathways."

"We are confident that this differentiated approach will continue to produce high-potential, first-in-class therapeutic candidates in our earlier stage immuno-oncology programs," Dr. Cohen-Dayag concluded.

Recent highlights:
·
Entered into a clinical trial collaboration with Bristol-Myers Squibb. The collaboration includes the supply of Opdivo for the Phase 1 dual combination arm of COM701 and Opdivo and a framework for expansion to additional combination studies, such as PVRIG and TIGIT blockers. In addition, Bristol-Myers Squibb made a $12 million equity investment in Compugen.
·
First patient dosed in the Phase 1 study for COM701.
·
First patient dosed in the Phase 1 study for BAY 1905254 which triggered a milestone payment of $7.8 million from Bayer.

Financial Results
Revenues for the third quarter of 2018 were $7.8 million, compared with $0 in the comparable period of 2017. The revenues for the quarter reflect the milestone achieved from Bayer AG in connection with the dosing of the first patient in the Phase 1 study of BAY 1905254.

R&D expenses for the third quarter ended September 30, 2018, were $7.8 million, compared with $7.6 million for the comparable period in 2017. R&D expenses continue to reflect pre-clinical expenses associated with COM902 in preparation of the IND application filing anticipated in 2019 and clinical expenses associated with the COM701 Phase 1 trial which was initiated in September 2018.

Net loss for the third quarter of 2018 was $3.1 million, or $0.05 per diluted share, compared with a net loss of $9.9 million, or $0.19 per diluted share, in the comparable period of 2017.

As of September 30, 2018, cash, cash related accounts, short-term and long-term bank deposits totaled $34.9 million, compared with $30.4 million at December 31, 2017. The quarter-end cash balance does not include the $12 million equity investment by Bristol-Myers Squibb or the $7.8 million for the milestone from Bayer AG. The Company has no debt.

Conference Call and Webcast Information
Compugen will hold a conference call to discuss its third quarter 2018 results today, November 7, 2018, at 8:30 a.m. ET. To access the live conference call by telephone, please dial 1-888-407-2553 from the U.S., or +972-3-918-0644 internationally. The conference call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website www.cgen.com.

On November 7, 2018 Protalix BioTherapeutics, Inc. (NYSE American:PLX, TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the three months and nine months ended September 30, 2018, and provided a corporate update (Press release, Protalix, NOV 7, 2018, View Source [SID1234531160]).

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"In the third quarter we achieved two important milestone events for pegunigalsidase alfa, or PRX-102, our differentiated enzyme replacement therapy in development for the treatment of Fabry disease. First, we expanded our partnership with Chiesi Farmaceutici S.p.A. to include exclusive rights to commercialize and develop PRX-102 in the United States, which significantly strengthened our financial position. Second, we reported positive preliminary results from our BRIDGE study on key kidney function," commented Moshe Manor, Protalix’s President and Chief Executive Officer. "Based on the promising preliminary BRIDGE study results, and taking into account the newly issued guidance from the U.S. Food and Drug Administration (FDA), we plan to engage with the FDA during the first half of 2019 to discuss the most optimal regulatory path forward for PRX-102. While we continue to enroll patients in all of our currently ongoing Fabry disease studies, we believe that with over 110 Fabry patients enrolled across the studies included in our PRX-102 clinical program to date, we have a sufficient number of patients for expedited review, including filing an application for accelerated approval," continued Mr. Manor.

2018 Third Quarter Highlights

·Presented preliminary positive data from the BRIDGE study showing Improvement in kidney function in patients switched from agalsidase alfa (Replagal) to pegunigalsidase alfa and further showing the reversal of a deterioration trend in kidney function to an improvement trend when switched.

·Expanded partnership with Chiesi Farmaceutici S.p.A., or Chiesi, to include U.S. rights for the development and commercialization of PRX-102. Terms of the agreement included an up-front payment of $25 million, up to $20 million in development costs and tiered royalties ranging from 15-40% of net sales.

·Expanded our Board of Directors with the addition of Mr. David Granot as an independent director.

·Continued exploring the potential for partnership opportunities mainly for OPRX-106, and for PRX-110. In parallel, the Company is exploring the option of conducting a controlled phase IIb study of OPRX-106 for the treatment of ulcerative colitis in order to maximize the value of this asset in a manner that will best serve the stockholders’ interest.

Financial Results for the Nine Months ended September 30, 2018

·The Company reported a net loss of $36.2 million, or $0.25 per share, basic and diluted for the nine-month period ended September 30, 2018 compared to a net loss of $32.1 million, or $0.25 per share, basic and diluted for the same period of 2017 excluding remeasurement of a derivative.

The Company recorded total revenues of $7.2 million for the nine-month period ended September 30, 2018, compared to $16.8 million for the same period of 2017. The decrease resulted primarily from decreased shipments of alfataliglicerase to Brazil despite the increase in the number of patients treated with alfataliglicerase, and decreased sales of drug substance to Pfizer.

The $25.0 million in proceeds received from Chiesi during the three-month period ended September 30, 2018 as an upfront payment were not recorded as revenues, and were deferred according to the revenue recognition rules of U.S. generally accepted accounting principles. Such proceeds should be recorded upon the commencement of commercial manufacturing. The same accounting treatment was applied to the $25.0 million upfront payment received by the Company in the fourth quarter of 2017, and the $11.8 million of research and development reimbursement payments the Company has received from Chiesi to date.

·Research and development expenses were $23.8 million for the nine-month period ended September 30, 2018, compared to $19.8 million for the same period of 2017.

Selling, general and administrative expenses were $7.3 million for the nine-month period ended September 30, 2018 compared to $8.2 million for the same period of 2017.

As of September 30, 2018, the Company had $41.9 million of cash and cash equivalents. With the expected decrease in cash consumption resulting primarily from the Company’s U.S. license transaction with Chiesi, the Company expects the cash balance to fund the Company through significant regulatory achievements of PRX-102.

Conference Call and Webcast Information

The Company will host a conference call on Wednesday, November 7, 2018 at 8:30 am ET to review the clinical, corporate and financial highlights.

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: +1-844-358-6760; International: +1-478-219-0004. Conference ID number 8567317.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.