On August 6, 2020 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on the registration and commercialization of momelotinib, a JAK1, JAK2 & ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis, reported its financial and operational results for the second quarter ended June 30, 2020 (Press release, Sierra Oncology, AUG 6, 2020, View Source [SID1234562990]).

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"We believe momelotinib, if approved, may provide an important treatment option for underserved myelofibrosis patients, in particular those with anemia and thrombocytopenia, and as such is well-positioned to generate significant value," said Dr. Stephen Dilly, President and CEO of Sierra Oncology. "During the second quarter, we continued to advance the MOMENTUM Phase 3 trial and are on track to deliver top-line results in the first half of 2022. In anticipation of these pivotal data, we are preparing for the regulatory submission process and the potential commercialization of momelotinib, and subsequent to the end of the quarter, we substantially strengthened our senior management team to support these activities."

"We made significant progress during the first half of 2020 operationalizing the global MOMENTUM Phase 3 trial and, while the potential impact of the COVID-19 pandemic continues to be uncertain, we are pleased with the current pace of enrollment," said Dr. Barbara Klencke, Chief Development Officer, Sierra Oncology. "During the EHA (Free EHA Whitepaper) virtual conference, two world-leading physicians in the treatment of myelofibrosis reported long-term data that continue to reinforce momelotinib’s differentiated durability, safety and efficacy profile. We plan to report updated analyses in late 2020 comparing the symptomatic benefits of momelotinib to ruxolitinib from the SIMPLIFY-1 Phase 3 trial that will further emphasize momelotinib’s differentiated and competitive profile."

"As the MOMENTUM trial ramps up, we’ve managed our resources prudently and continue to anticipate our current cash runway will extend beyond top-line data and into the second half of 2022, subject to the potential impact of COVID-19," said Mr. Sukhi Jagpal, Chief Financial Officer of Sierra Oncology. "In addition, our Series B warrants will expire on the 75th day following the announcement of top-line data and may only be exercised by paying the exercise price in cash, which would amount to approximately $34.0 million in proceeds to the Company if fully exercised. We are also starting to explore non-dilutive options that could provide additional capital to support our North American commercialization strategy."

Second Quarter Highlights:

Hosted an Analyst & Investor Call featuring a presentation by renowned myelofibrosis expert Dr. Ruben Mesa, Director of the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center, who discussed momelotinib’s ability to address anemia and transfusion dependency, two critical unmet medical needs in treating patients with myelofibrosis.
Reported favorable Long-Term Safety and Dose Intensity data for momelotinib from more than 550 patients across the two previously conducted SIMPLIFY Phase 3 studies and their subsequent ongoing extended treatment periods, at the 25th EHA (Free EHA Whitepaper) Virtual Congress.
Professor Claire Harrison, Guy’s and St. Thomas’ NHS Foundation Trust, London, United Kingdom presented a poster on the long-term safety profile of momelotinib, which demonstrated a lack of emergent or cumulative toxicity with extended daily administration. More than 90 SIMPLIFY-1 and SIMPLIFY-2 patients continued to receive momelotinib for 3.5 years or longer. Patients treated with momelotinib experienced rapid and sustained increases in hemoglobin, in contrast to the significant decrease in hemoglobin for patients receiving ruxolitinib. Patients treated with momelotinib also experienced significantly higher mean platelet counts compared to those receiving ruxolitinib. Importantly, patients who switched from ruxolitinib to momelotinib also achieved a sustained improvement in hemoglobin in both studies, and platelets in SIMPLIFY-1.
Dr. Vikas Gupta, Princess Margaret Cancer Centre, Toronto, Canada, presented a poster highlighting the sustained dose intensity and prolonged clinical activity of momelotinib across the continuum of JAK inhibitor naïve and previously JAK inhibitor treated myelofibrosis patients. While the starting doses for ruxolitinib were often attenuated due to low platelets, further reductions in dose intensity were also commonly required for ruxolitinib. In contrast, momelotinib was initiated at full dose for all patients enrolled to the SIMPLIFY studies and high dose intensity was maintained in the majority over extended dosing durations. Patients who switched from ruxolitinib to momelotinib saw an immediate and sustained improvement in dose intensity.
The data from the two interrelated presentations suggest that the favorable effect on hemoglobin and platelets allows momelotinib to be initiated at high dose intensity and maintained at high dose intensity over extended durations while retaining a favorable long-term safety profile. Notably, some patients continue to receive momelotinib 10 years after enrolling in the initial momelotinib Phase 2 trials, while 90 Phase 3 SIMPLIFY patients who enrolled into those trials 4 to 6 years ago continue to receive momelotinib, suggesting that the dosing and safety profile contributes to momelotinib’s potential ability to provide sustained benefits over extended durations.
Second Quarter 2020 Financial Results (all amounts reported in U.S. currency)
Research and development expenses were $10.2 million for the three months ended June 30, 2020 compared to $11.7 million for the three months ended June 30, 2019. The decrease was primarily due to a $2.1 million decrease in clinical trial, third-party manufacturing, research and preclinical costs for SRA737, a $1.1 million decrease in personnel-related and allocated overhead costs, and a $0.9 million decrease in third-party manufacturing costs for momelotinib. These decreases were partially offset by a $2.6 million increase in clinical trial and development costs for momelotinib. Research and development expenses included non-cash stock-based compensation of $0.9 million and $1.2 million for the three months ended June 30, 2020 and 2019, respectively.

Research and development expenses were $21.8 million for the six months ended June 30, 2020, compared with $21.9 million for the six months ended June 30, 2019. The decrease was primarily due to a $5.2 million decrease in clinical trial, third-party manufacturing, research and preclinical costs for SRA737, a $2.1 million decrease in personnel-related and allocated overhead costs, and a $0.6 million decrease in third-party manufacturing costs for momelotinib. These decreases were offset by a $6.3 million increase in clinical trial and development costs for momelotinib, and a non-cash charge of $1.5 million pertaining to the change in fair value of an obligation to issue common stock and a warrant to Gilead Sciences, Inc. (Gilead), which were issued during the first quarter of 2020. Research and development expenses included non-cash stock-based compensation of $1.5 million and $2.4 million for the six months ended June 30, 2020 and 2019, respectively.

General and administrative expenses were $6.3 million for the three months ended June 30, 2020, compared to $3.5 million for the three months ended June 30, 2019. The increase was primarily due to a non-cash $2.2 million stock-based compensation charge and a $0.6 million severance charge pertaining to an executive resignation. General and administrative expenses included non-cash stock-based compensation of $2.7 million and $0.5 million for the three months ended June 30, 2020 and 2019, respectively.

General and administrative expenses were $10.8 million for the six months ended June 30, 2020, compared to $6.8 million for the six months ended June 30, 2019. The increase was primarily due to a $3.0 million increase in personnel-related and allocated overhead costs, including a non-cash $2.2 million stock-based compensation charge noted above and $1.0 million of severance charges, offset by a decrease of $0.2 million in other personnel-related and allocated overhead costs. There was also an increase of $1.0 million in professional fees, including pre-commercial planning costs for momelotinib. General and administrative expenses included non-cash stock-based compensation of $3.1 million and $1.0 million for the six months ended June 30, 2020 and 2019, respectively.

Other income (expense), net was $24,000 of other expense, net for the three months ended June 30, 2020, compared to $0.3 million of other income, net for the three months ended June 30, 2019. The difference was primarily attributable to a decrease in interest income due to lower interest rates. Other income (expense), net was $15.7 million of other expense, net for the six months ended June 30, 2020, compared to $0.7 million of other income, net for the six months ended June 30, 2019. The difference was primarily attributable to a non-cash charge of $16.2 million related to the change in fair value of warrant liabilities which were reclassified to equity in January 2020.

For the three months ended June 30, 2020, Sierra incurred a GAAP net loss of $16.5 million compared to a GAAP net loss of $14.9 million for the months ended June 30, 2019. For the six months ended June 30, 2020, Sierra incurred a GAAP net loss of $48.4 million compared to a GAAP net loss of $27.9 million for the six months ended June 30, 2019. The GAAP net loss for the six months ended June 30, 2020 includes a non-cash charge of $16.2 million related to the change in fair value of warrant liabilities included in other income (expense), net and a $1.5 million non-cash charge pertaining to the obligation to issue securities to Gilead included in research and development expenses as mentioned above.

Non-GAAP adjusted net loss was $12.8 million for the three months ended June 30, 2020, compared with a non-GAAP adjusted net loss of $13.1 million for the three months ended June 30, 2019. Non-GAAP adjusted net loss for the three months ended June 30, 2020 and 2019 excludes expenses related to stock-based compensation. For the six months ended June 30, 2020, Sierra incurred a non-GAAP adjusted net loss of $26.1 million compared to a non-GAAP adjusted net loss of $24.5 million for the six months ended June 30, 2019. Non-GAAP adjusted net loss for the six months ended June 30, 2020 excludes expenses related to the change in fair value of warrant liabilities, the change in fair value of the securities issuance obligation, and stock-based compensation. Non-GAAP adjusted net loss for the six months ended June 30, 2019 excludes expenses related to stock-based compensation. See "Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below for a reconciliation of this GAAP and non-GAAP financial measure.

Cash and cash equivalents totaled $123.2 million as of June 30, 2020, compared to $147.5 million as of December 31, 2019.

As of June 30, 2020, there were 10,395,732 total shares of common stock outstanding and warrants to purchase 11,102,251 shares of common stock, with an exercise price equal to $13.20 per share. There were 2,351,055 shares issuable upon exercise of stock options and an additional warrant to purchase 1,839 shares.

On August 6, 2020 Cryoport, Inc. (NASDAQ: CYRX) ("Cryoport"), a global leader in life sciences supply chain solutions, reported financial results for Second Quarter and six-month period ended June 30, 2020 (Press release, Cryoport, AUG 6, 2020, View Source [SID1234563206]).

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"For the Second Quarter of 2020, we reported revenue of $9.4 million, an increase of 11% from the Second Quarter of 2019. This was largely driven by revenue from our biopharma segment. We are pleased to have achieved these solid results and to have provided advanced temperature-controlled supply chain services to deliver high-value therapies to eligible patients across our clinical and commercial portfolios globally and without disruption," said Jerrell Shelton, Chairman and Chief Executive Officer.

"Our Second Quarter results reflect the strength and resilience of our company where we saw continued year-over-year growth despite the challenging environment caused by the COVID-19 pandemic. Thanks to the tireless work of our colleagues around the world, we continue to successfully navigate the external realities, and we remain focused on our mission of supporting life and health by delivering reliable and comprehensive temperature-controlled supply chain solutions for the life sciences through our innovation, advanced technologies, and global supply chain network.

"We now support four (4) commercial cell and gene therapies: KYMRIAH, YESCARTA, TECARTUS and ZYNTEGLO along with Lisocabtagene Maraleucel (liso-cel), which was recently validated by the European Medicines Agency ("EMA").

"Gilead’s Kite renewed our agreement for temperature-controlled supply chain support of their commercial and clinical therapies, and we are in the final stages of renewing our agreement with Novartis in support of its commercial and clinical therapies. Over the past several years, Cryoport’s advanced solutions have supported both KYMRIAH and YESCARTA through their respective clinical phases and subsequent respective commercial rollouts. The continuance of these partnerships with these pioneers in the regenerative medicine market is important as each extends its life-saving therapies to greater and greater patient populations, globally. We think that as the only company with a proven and advanced integrated suite of proprietary temperature-controlled supply chain solutions to support the global high-volume distribution of these valuable therapies, Cryoport’s position is highly entrenched and we work hard every day to maintain that position. These renewals together with the increasing number of clinical trials we support demonstrate our leading position, which we will continue to fortify as the number of cell and gene therapy trials grow and commercial therapies come to market.

"Also, during the second quarter, FedEx extended its agreement with Cryoport Systems, a relationship that allows both companies to continue to jointly deliver temperature-controlled solutions for select clients in the Biopharma, Animal Health and Reproductive Medicine markets. The continuation of this partnership is a validation of Cryoport’s innovative technologies and FedEx’s foresight and an indication of the life sciences industry’s growing demand for advanced temperature-controlled supply chain solutions.

Biopharma

"We continue to grow our leading position in the biopharma market and are happy to report strong biopharma clinical trial growth for the second quarter as a net total of 26 clinical trials were added, bringing the total number of clinical trials supported by Cryoport to a record 491. And, on another positive note, of the 56 clinical trials suspended at the end of the first quarter due to the COVID-19 pandemic, only 3 remain suspended as of the end of the second quarter. None of the remaining trials, to Cryoport’s knowledge, have been terminated.

"Cryoport’s market share leadership of temperature-controlled life science solutions continues to grow stronger as our client base expands and our competency in tailored information technology extends our market lead. The Alliance for Regenerative Medicine (ARM) reported that at the end of June there were 11 ongoing clinical trials worldwide for the development of COVID-19 treatments utilizing regenerative medicine and advanced therapies, and Cryoport supports eight of them. This area remains very active globally as many companies with the help of regulatory support are moving at a rapid pace in search for a cure. Cryoport is committed to supporting these important programs as we all seek an end to the global pandemic.

"In July 2020, another Cryoport-supported therapy received FDA approval, bringing the total number of commercial products supported by Cryoport to four, and, in addition, one more was validated by the EMA, and entered the EMA’s centralized review process. A further six (6) Cryoport supported Marketing Authorization Applications or Biologic License Applications are expected to be filed in 2020 and two additional commercial approvals are possible, based on internal information and forecasts from ARM, although the timing of some of these may be impacted by the COVID-19 pandemic and other factors."

Mr. Shelton continued, "We expect revenue from our agreement with Gilead’s Kite to increase following the recent FDA approval of TECARTUS, its chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). We are also seeing an acceleration of other Cryoport supported regenerative therapies approaching commercialization with the recent validation of Bristol Myers Squibb’s Application for CAR T-Cell Therapy Lisocabtagene Maraleucel (liso-cel) by the EMA.

"Cryoport now supports a net total of 491 clinical trials as of June 30, 2020 compared to 413 as of June 30, 2019. The number of trials in Phase III grew to 66, compared to 52 as of June 30, 2019. Of the 491 total trials Cryoport supports, 400 are in the Americas, 72 in EMEA (Europe, the Middle East and Africa) and 19 in APAC (Asia Pacific). This compares to 353 in the Americas, 53 in EMEA and 7 in APAC as of June 30, 2019. Our market leading share of clinical trials continues to climb as according to ARM there were a total of 1,078 active clinical trials globally at the end of the second quarter, with 394 in Phase I, 587 in Phase II and 97 in Phase III.

"Another sign of the strength and belief in the potential of cell and gene therapies can be seen in the financial markets. Despite the economic challenges and uncertainty presented by the pandemic regenerative medicine companies raised approximately $10.7B in financing in the first half of 2020, a 120% increase from the first half of 2019.

"To meet upcoming customer demand, further strengthen our global footprint and support capabilities, we are continuing to build out our new Cryoport Express Global Supply Chain Network and our full range of temperature-controlled supply chain solutions, which include a full range of bioservices and life sciences logistics support. Both new Supply Chain Centers in Morris Plains, NJ and Houston, TX should be online by the first quarter of 2021. Additionally, we continue to build our global alliance network as we recently announced a partnership with Medipal Holdings, one of the leading pharma wholesalers and distributors in Japan. This partnership helps us grow our abilities in the APAC region.

"Cryogene, the life sciences industry’s most trusted biostorage facility specializing in the secure storage of biological specimens, materials, and samples for research purposes, contributed revenue of $1.3 million for the second quarter. Its operations were minimally impacted by the COVID-19 pandemic and we continue to plan the geographic expansion of our bioservices offering both in the U.S. and globally."

Animal Health

Mr. Shelton continued, "Animal Health revenue remained steady at $0.2 million for the three months ended June 30, 2020; however, we are building a strong pipeline of potential new clients and expect to grow Animal Health revenue in the second half of 2020."

Reproductive Medicine

"The Reproductive Medicine market was impacted by the temporary closure of fertility clinics globally and, consequently, contributed revenue of $0.6 million in the second quarter of 2020; however, we have begun to see restrictions lifted, leading to a significant ramp in Reproductive Medicine revenue during the final month of the second quarter. We were pleased to see our partnership with Inception Fertility contribute to this revenue ramp and believe we are well-positioned, globally, for growth in this important market."

Financial Highlights

Revenue increased 11% to $9.4 million for the three-month period ended June 30, 2020, compared with the same period in the prior year.
Revenue increased 27% to $19.2 million for the six-month period ended June 30, 2020, compared with the same period in the prior year.
Excluding revenue from the Cryogene acquisition in May of 2019, revenue grew 2.7% for the three-month period ended June 30, 2020, compared with the same period in the prior year. Excluding revenue from the Cryogene acquisition in May of 2019, revenue grew 14.2% for the six-month period ended June 30, 2020, compared with the same period in the prior year.
Biopharma revenue increased by 4.7% in the three months ended June 30, 2020 compared to the same period in 2019.
Commercial Biopharma revenue increased $0.7 million or 38%, to $2.6 million for the three months ended June 30, 2020, as compared to $1.9 million for the same period in 2019.
Gross margin for the three and six months ended June 30, 2020 was 55% and 54% respectively, compared to 51% and 52% for the same periods in the prior year.
Operating costs and expenses increased by $4.3 million for the three-month period ended June 30, 2020, compared to the same period in the prior year, as a result of strategic initiatives, such as the continued investments in infrastructure buildout, including software development, which will provide a platform for continuing the scaling of our business; engineering initiatives, which includes the development of revolutionary packaging and monitoring and communications resources, the development of our Cryoport Express Global Supply Chain Network, and the build out of competencies in support of advancing our infrastructure and the growth in demand for Cryoport’s solutions.
Net loss for the three-month period ended June 30, 2020 was $5.8 million, or $0.15 per share, compared to a net loss of $2.5 million, or $0.08 per share in the same period in 2019.
Net loss for the six-month period ended June 30, 2020 was $9.7 million, or $0.26 per share, compared with $4.9 million, or $0.16 per share, in the same six-month period in 2019.
Adjusted EBITDA for the three-month period ended June 30, 2020 was ($2.5 million), compared with $0.2 million in the same period in the prior year. Adjusted EBITDA for the six-month period ended June 30, 2020, was ($4.1 million), compared with ($0.1 million) in the same six-month period in the prior year.
Cryoport reported $208.2 million in cash, cash equivalents and short-term investments as of June 30, 2020, compared with $94.3 million as of December 31, 2019. This increase includes net proceeds of approximately $111 million received from a convertible debt offering during the three-month period ended June 30, 2020.
Further information on Cryoport’s financial results is included on the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport’s financial performance are provided in Cryoport’s quarterly report on Form 10-Q for the three months ended June 30, 2020, which will be filed with the Securities and Exchange Commission ("SEC") on or about August 10, 2020. The full report will be available on the SEC Filings section of the Investor Relations section of Cryoport’s website at www.cryoport.com.

Earnings Conference Call Information

IMPORTANT INFORMATION: A document titled "Cryoport Second Quarter 2020 in Review", providing a review of Cryoport’s recent financial and operational performance and a general business update, will be issued at 4:05 pm ET on Thursday, August 6, 2020. The document is designed to be read by investors before the questions and answers conference call and can be accessed at http://ir.cryoport.com/events-and-presentations.

Cryoport management will host a conference call at 5:00 pm ET on August 6, 2020. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company’s reported results.

Conference Call Information

Date:

August 6, 2020

Time:

5:00 p.m. ET

Dial-in numbers:

+1 (800) 496-4125 (U.S.), +1 (720) 452-9104 (International)

Confirmation code:

Request the "Cryoport Call"

Live webcast:

‘Investor Relations’ section at www.cryoport.com or at this link. Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software.

Questions and answers will be recorded and available approximately three hours after completion of the live event on the Investor Relations section of the Company’s website at www.cryoport.com for a limited time. To access the replay of the questions and answers, please follow this link. A dial-in replay of the call will also be available, to those interested, until August 13, 2020. To access the replay, dial +1 (844) 512-2921 (United States) or +1 (412) 317-6671 (International) and enter replay pin number: 7980251.

On August 6, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported its financial results for the second quarter and six months ended June 30, 2020 (Press release, CTI BioPharma, AUG 6, 2020, View Source [SID1234563205]).

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"This past quarter we announced enrollment of the first patient in our PRE-VENT Phase 3 clinical trial of pacritinib in hospitalized patients with severe COVID-19, an important step for CTI as we work to provide a new therapeutic option for COVID-19 patients," said Adam R. Craig, M.D., Ph.D. "With regards to the PACIFICA Phase 3 trial, we continue to enroll patients but the enrollment rate is lower than planned due to the COVID-19 pandemic and we now anticipate at least a six-month delay in the trial. However, given our cash runway into Q4 2021, we remain confident in our ability to successfully execute on the development of pacritinib for the treatment of severely thrombocytopenic myelofibrosis patients."

Second Quarter Financial Results
Operating loss was $10.0 million and $21.9 million for the three and six months ended June 30, 2020, respectively, compared to operating loss of $11.0 million and $21.5 million for the respective periods in 2019. Operating loss for the three months ended June 30, 2020 as compared to the comparable period in 2019 resulted primarily from a decrease in general and administrative expenses. The increase in operating loss for the six months ended June 30, 2020 as compared to the comparable period in 2019 resulted primarily from the recording of a full allowance against certain VAT receivables due to an increased uncertainty of collectability.

No revenues were recognized for the three and six months ended June 30, 2020, while revenues of $0.4 million and $1.1 million, respectively, were recognized for the comparable periods in 2019. License and contract revenues in 2019 resulted from royalty and other revenues recognized from Les Laboratoires Servier and Institut de Recherches Internationales Servier ("Servier") related to transition period activities pursuant to the terms of the Termination and Transfer Agreement with Servier.

Net loss for the three months ended June 30, 2020 was $14.0 million, or $(0.19) for basic and diluted loss per share, compared to net loss of $11.0 million, or $(0.19) for basic and diluted loss per share, for the same period in 2019. Net loss for the six months ended June 30, 2020 was $26.2 million, or $(0.38) for basic and diluted loss per share, compared to net loss of $21.8 million, or $(0.38) for basic and diluted loss per share, for the same period in 2019.

As of June 30, 2020, cash, cash equivalents and short-term investments totaled $70.1 million, compared to $33.7 million as of December 31, 2019. We expect current cash and cash equivalents will enable us to fund our operations into the fourth quarter of 2021.

On August 6, 2020 Proteostasis Therapeutics, Inc. (Nasdaq: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF), reported financial results for the second quarter ended June 30, 2020 (Press release, Proteostasis Therapeutics, AUG 6, 2020, View Source [SID1234563204]).

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"The COVID-19 pandemic has highlighted the unmet needs of the CF community and reaffirmed our commitment to providing more treatment choices for people living with CF," said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics. "Our team continues to work tirelessly to advance our mission and we anticipate announcing data from the ex vivo portion of the CHOICES development program in the fourth quarter of 2020, and plan to move into the clinical portion by year-end."

The CHOICES (Crossover trial based on Human Organoid Individual response in CF – Efficacy Study) development program is based on testing PTI drug combinations in an ex vivo study and, subsequently, in a clinical trial to assess the predictability of the organoid assay for clinical outcomes. The ex vivo portion of the study will quantify the functional response of patient-derived organoids to PTI investigational agents dirocaftor, posenacaftor and nesolicaftor. Enrollment of 502 patients with CF in the ex vivo portion of the study is now complete and the Company expects ex vivo data in the fourth quarter of 2020. The dataset will enable targeted enrollment into the clinical portion of the CHOICES trial of subjects with rare Cystic Fibrosis transmembrane conductance regulator (CFTR) mutations based on their individual ex vivo response. The clinical portion of the trial is expected to begin by the end of 2020. PTI and its HIT-CF partners are currently in the process of activating approximately 30 clinical sites in Europe to support the CHOICES execution. The results from the CHOICES clinical trial may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency (EMA) in 2021 through a novel regulatory pathway.

Recent Highlights

In June of this year, Proteostasis announced results from in vitro studies evaluating the use of PTI-129 as a potential treatment for COVID-19. PTI-129 is a pre-clinical, once-daily, oral small molecule identified through our Disease Relevant Translation (DRT) platform and originally designed to treat protein misfolding disorders involving the unfolded protein response (UPR). In in vitro studies conducted at Calibr, the drug discovery division of Scripps Research, PTI-129 demonstrated the potential to reduce viral protein production in host cells by activating the adaptive branches of the UPR pathway and reducing the levels of misfolded cellular proteins.

Second Quarter 2020 Financial Results

Proteostasis reported a net loss of approximately $8.9 million for the three months ended June 30, 2020, as compared to a net loss of $20.0 million for the same period in the prior year.

The Company recorded no revenue in the three months ended June 30, 2020 and 2019.

Research and development expenses for the three months ended June 30, 2020 were $4.6 million, as compared to $16.9 million for the same period in the prior year. The decrease in research and development expenses for the three months ended June 30, 2020 was primarily due to a decrease in clinical-related activities and related work as the CF studies progressed to data read out in late 2019.

General and administrative expenses for the three months ended June 30, 2020 were $4.4 million, as compared to $3.7 million for the same period in the prior year. The increase in general and administrative expenses for three months ended June 30, 2020 was due primarily to an increase in professional fees supporting financing activities.

Cash, cash equivalents and short-term investments totaled $48.9 million as of June 30, 2020, compared to $69.5 million as of December 31, 2019. The Company believes that its existing cash, cash equivalents and short-term investments are sufficient to fund operations into the second half of 2021. However, additional funding will be necessary to advance the Company’s proprietary combination therapy candidates through regulatory approval and into commercialization, if approved.

On August 6, 2020 Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, reported financial results for second quarter 2020 and provided a business update (Press release, Eiger Biopharmaceuticals, AUG 6, 2020, View Source [SID1234563203]).

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"Eiger is executing toward multiple important milestones across our pipeline, including anticipated FDA approval of Zokinvy in Progeria and Progeroid Laminopathies," said David Cory, President and CEO. "Our Phase 3 HDV D-LIVR trial continues to enroll and dose patients with full enrollment expected in 2021, and we plan for end-of-treatment and end-of-study data from our Phase 2 HDV LIFT study of peginterferon lambda in combination with lonafarnib this year. In addition, we look forward to results from multiple ongoing investigator sponsored studies of peginterferon lambda in COVID-19 patients."

Recent Highlights and Upcoming Milestones

Zokinvy (lonafarnib) in Progeria and Progeroid Laminopathies

New Drug Application (NDA) accepted for filing by FDA with priority review and Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2020
Marketing Authorization Application (MAA) under review by EMA will follow a standard review timeline. EMA request for inspections, in addition to travel restrictions due to COVID-19, will delay EMA from completing its review within the framework of previously granted accelerated assessment.
Lonafarnib in Hepatitis Delta Virus (HDV)

Phase 3 D-LIVR study (N=400) continues to enroll and dose patients
Full enrollment expected in 2021
Peginterferon Lambda in HDV

Phase 2 LIFT (combo with lonafarnib) end-of-treatment data planned for EASL 2020; end-of-study data planned for AASLD 2020
Single, Phase 3 study design agreement with FDA and EMA
Peginterferon Lambda in COVID-19

Six International Investigator Sponsored Studies in progress
Second Quarter 2020 Financial Results

Cash, cash equivalents, and short-term investments as of June 30, 2020 totaled $90.8 million.

The Company reported net loss of $15.3 million, or $0.60 per share, for second quarter 2020, as compared to $17.5 million, or $0.75 per share, for second quarter 2019.

Research and Development expenses were $9.8 million for second quarter 2020, as compared to $12.9 million for second quarter 2019. The decrease was primarily due to a decrease in regulatory expenses and lower clinical trial related expenses, including clinical material costs.

General and Administrative expenses were $4.9 million for second quarter 2020, as compared to $4.2 million for second quarter 2019. The increase was primarily due to an increase in outside legal, consulting, advisory and accounting services.

Total operating expenses include total non-cash expenses of $1.8 million for second quarter 2020, as compared to $1.8 million for the same period in 2019.

As of June 30, 2020, the Company had 27,241,640 of common shares outstanding.