On May 9, 2018 Lodo Therapeutics Corporation, a drug discovery and development company focused on identifying and producing unique, bioactive natural products directly from the microbial DNA sequence information contained in soil, reported that it has formed a strategic drug discovery collaboration with Genentech, a member of the Roche Group (Press release, Lodo Therapeutics, MAY 9, 2018, View Source [SID1234526409]).

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

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

                  Schedule Your 30 min Free Demo!

Under the terms of the agreement, Genentech will utilize Lodo Therapeutics’ proprietary genome mining and biosynthetic cluster assembly platform to identify novel molecules with therapeutic potential against multiple disease-related targets of interest to Genentech. Lodo will receive an undisclosed upfront payment and is eligible to receive research, development and commercialization milestone payments up to $969 million based on achievement of certain predetermined milestones. In addition, Lodo is eligible to receive tiered-royalties on sales of certain products resulting from the collaboration.

"Lodo Therapeutics’ proprietary drug discovery platform is a powerful engine for identifying novel compounds with important therapeutic potential," said Thong Q. Le, chief executive officer at Lodo Therapeutics and Accelerator Life Science Partners. "We are incredibly excited to work with Genentech, and we look forward to demonstrating the power and utility of Lodo’s unique technology for the benefit of global human health."

Compounds derived from natural products comprise a significant proportion of the small molecule drugs used to treat cancer, infections and chronic illnesses such as Type 2 diabetes. Rather than relying on culturing known strains of bacteria, Lodo Therapeutics’ genome-based approach leverages the power of microbial evolution to identify novel, naturally occurring compounds that have therapeutic potential in the treatment of cancer and drug-resistant bacterial infections. This approach is expected to reduce the time and cost of drug discovery.

"Our ability to enter into a strategic collaboration with one of the leaders in innovating wholly new classes of drugs just two years after Lodo Therapeutics was founded reflects the potential of our proprietary platform to be a valuable resource to advance their drug discovery initiatives," said David Pompliano, Ph.D., co-founder and chief scientific officer of Lodo Therapeutics.

"We are excited to work with Genentech in their quest to discover novel, next-generation natural products derived from the microbiome of the soil using this innovative platform developed by Lodo," said Sean Brady, Ph.D., co-founder of Lodo Therapeutics and Associate Professor at The Rockefeller University. James Sabry, M.D., Ph.D., senior vice president and global head of Genentech Partnering, commented,

"Genentech is committed to accessing innovative technologies and we are excited to collaborate with Lodo Therapeutics to apply their Metagenomics Technology Platform to potentially discover therapeutics for difficult drug targets.

On May 9, 2018 Akebia Therapeutics, Inc. (NASDAQ:AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients with kidney disease through the biology of hypoxia-inducible factor (HIF), reported financial results for the first quarter ended March 31, 2018 (Press release, Akebia, MAY 9, 2018, View Source [SID1234526408]).

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

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

                  Schedule Your 30 min Free Demo!

"We began 2018 with strong momentum, focused on advancing our vadadustat clinical program, and we are targeting full enrollment in our PRO2TECT and INNO2VATE registration studies by year end," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We drive our business forward from a position of financial strength. Our existing cash resources and committed capital from our collaboration partners are expected to fund our current operating plan into early 2020."

First Quarter 2018 and Recent Corporate Highlights

Announced targeting of full enrollment in the Phase 3 PRO2TECT and INNO2VATE studies by the end of 2018, with top-line results anticipated in 2019, subject to the accrual of major adverse cardiovascular events, or MACE, and market launch planned in 2020, subject to regulatory approval;
Announced enhanced study designs for FO2RWARD and TRILO2GY, now referred to as FO2RWARD-2 and TRILO2GY-2, which Akebia believes will provide additional characterization and differentiation of vadadustat and further strengthen the company’s commercial position, subject to vadadustat’s approval. Top-line results from FO2RWARD-2 are expected in the first half of 2019;
Following a positive consultation with the Japanese regulatory authority, PMDA, collaboration partner Mitsubishi Tanabe Pharma Corporation (MTPC) initiated Phase 3 studies of vadadustat in patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis dependent chronic kidney disease (DD-CKD) in Japan, which generated $10.0 million in milestone payments to Akebia. Data read-outs are expected in 2019;
Raised $89.3 million in gross proceeds through a public offering of common stock; existing cash resources and cost-share funding from collaborators are expected to fund Akebia’s current operating plan into the first quarter of 2020;
Announced positive top-line results from a Phase 2 study of vadadustat in Japanese patients with anemia associated with DD-CKD; the data from this study were consistent with findings from previous studies of vadadustat; and
The Independent Data Monitoring Committee for Akebia’s global Phase 3 PRO2TECT and INNO2VATE programs held another meeting and recommended continuing the studies without modification.
Financial Results

Akebia reported a net loss of $23.4 million, or ($0.48) per share, for the first quarter of 2018 as compared to a net loss for the first quarter of 2017 of $44.5 million or ($1.15) per share.

Collaboration revenue was $45.9 million for the first quarter of 2018 compared to $20.9 million for the first quarter of 2017. Collaboration revenue recognized in the first quarter of 2018 related to revenue recognized under both the U.S. collaboration agreement with Otsuka Pharmaceutical Co. Ltd., or Otsuka, and the collaboration agreement with Otsuka related to Europe, China and certain other regions, which was consummated in April 2017, as well as revenue recognized in connection with the collaboration agreement with MTPC. Collaboration revenue recognized in the first quarter of 2017 related only to the U.S. collaboration agreement with Otsuka, which was consummated in December 2016.

On January 1, 2018, Akebia was required to retrospectively adopt the new revenue recognition standard, ASC 606, Revenue from Contracts with Customers, as if the standard had been in effect during all prior periods. The adoption of ASC 606 resulted in a retrospective $3.2 million of additional collaboration revenue for the year ended December 31, 2017. As required by ASC 606, Akebia will adjust the comparative 2017 financial results to reflect the retrospective additional revenue in its Annual Report on Form 10-K for the fiscal year ended December 31, 2018.

Research and development expenses were $61.4 million for the first quarter of 2018 compared to $60.0 million for the first quarter of 2017. The increase is primarily attributable to external costs related to the development of vadadustat, including the manufacture of drug substance and drug product for the global Phase 3 program, and regulatory activities as well as other clinical and preclinical activities. Research and development expenses were further increased by headcount, consulting and facility-related costs related to additional resources required to support the company’s expanding research and development programs.

General and administrative expenses were $9.0 million for the first quarter of 2018 compared to $5.8 million for the first quarter of 2017. The increase is primarily attributable to an increase in costs to support the company’s research and development programs, including headcount and compensation-related costs and associated facility-related costs.

Akebia ended the first quarter of 2018 with cash, cash equivalents and available for sale securities of $393.0 million. The company also generally receives cost-share funding from its collaboration agreements with Otsuka on a prepaid quarterly basis. Akebia expects its existing cash resources, including the prepaid quarterly committed cost-share funding from its collaborators, to fund its current operating plan into the first quarter of 2020.

On May 9, 2018 Novavax, Inc., (Nasdaq:NVAX) reported its financial results for the first quarter ended March 31, 2018 (Press release, Novavax, MAY 9, 2018, View Source [SID1234526407]).

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

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

                  Schedule Your 30 min Free Demo!

First Quarter and Subsequent Achievements:

RSV F Vaccine

In May 2018, Novavax reached enrollment of approximately 4,600 pregnant women in its Prepare Phase 3 clinical trial of its RSV F Vaccine for infants via maternal immunization. This milestone enables Novavax to initiate a prespecified interim efficacy analysis after approximately six months of follow-up of the last infant born to the approximately 4,600 women enrolled (including 3,000 actively vaccinated women). Completion of this analysis is expected in the first quarter of 2019. Since 2015, the Prepare trial is supported by an $89 million grant from the Bill & Melinda Gates Foundation (BMGF).

In April 2018, Novavax presented at the World Vaccine Congress on the status of its Phase 3 clinical trial of its RSV F Vaccine.
NanoFlu

In April 2018, Novavax presented clinical data at the World Vaccine Congress from the Phase 1/2 clinical trial in older adults comparing trivalent formulations of NanoFlu to the market-leading licensed egg-based, high-dose influenza vaccine for older adults.

In February 2018, Novavax reported positive top-line results from its Phase 1/2 clinical trial of its trivalent NanoFlu.
Corporate

In April 2018, Novavax completed an underwritten public offering of approximately 34.8 million shares of its common stock, including 4.5 million shares pursuant to the underwriters’ option to purchase additional shares. The shares resulted in net proceeds of $54 million.

Effective on March 14, 2018, John J. Trizzino, former Senior Vice President, Commercial Operations since 2014, was promoted to Senior Vice President, Chief Business Officer and Chief Financial Officer.
Anticipated Events:

Initiation of the Phase 2 clinical trial of quadrivalent formulations of NanoFlu scheduled to begin in the third quarter of 2018.

Top-line data from the Phase 2 NanoFlu trial and End of Phase 2 meeting with the FDA expected in the first quarter of 2019.

Results of the Prepare Phase 3 interim efficacy analysis for our RSV F Vaccine expected in the first quarter of 2019.
Summary

"We had an extremely productive first quarter, including making important advances in our two lead clinical vaccine programs. We are pleased to have reached the enrollment target for our Prepare Phase 3 RSV F Vaccine trial, which clears the path for following these most recent participants and their babies, and subsequently announcing top-line results of our planned interim efficacy analysis in the first quarter of 2019," said Stanley C. Erck, President and CEO of Novavax, Inc. "We also continue to make significant progress on NanoFlu and plan to initiate a Phase 2 clinical trial in the third quarter of 2018."

Financial Results for the First Quarter Ended March 31, 2018

Novavax reported a net loss of $46.4 million, or $0.14 per share, for the first quarter of 2018, compared to a net loss of $43.9 million, or $0.16 per share, for the first quarter of 2017.

Novavax revenue in the first quarter of 2018 was $9.7 million, compared to $5.7 million in the same period in 2017. This 70% increase was driven by higher revenue recorded under the BMGF grant corresponding to the increased enrollment in the Prepare trial.

Research and development expenses increased 18% to $44.5 million in the first quarter of 2018, compared to $37.7 million for the same period in 2017. The increase was primarily due to increased development activities of the RSV F Vaccine for infants via maternal immunization.

Interest income (expense), net for the first quarter of 2018 was ($2.9) million, compared to ($3.0) million for the same period of 2017.

As of March 31, 2018, Novavax had $164.2 million in cash, cash equivalents, marketable securities and restricted cash, compared to $186.4 million as of December 31, 2017. Net cash used in operating activities for the first quarter of 2018 was $66.1 million, compared to $44.5 million for same period in 2017. The increase in cash usage was primarily due to approximately $16 million of one-time payments, as well as the adoption of a new accounting standard that requires restricted cash to be included in the beginning and ending balances on the statements of cash flows, thus increasing Novavax’ cash usage in the first quarter of 2018 and 2017 by approximately $9 million and $6 million, respectively. We expect our cash used in operating activities to significantly decrease for the subsequent quarters of 2018 as compared to the first quarter of 2018.

Conference Call

Novavax management will host its quarterly conference call today at 4:30 p.m. ET. The dial-in number for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 3687883. A replay of the conference call will be available starting at 7:30 p.m. ET on May 9, 2018 until 7:30 p.m. ET on May 16, 2018. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 3687883.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website at www.novavax.com or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available on the Novavax website until August 9, 2018.

About RSV

RSV is the most common cause of lower respiratory tract infections and the leading viral cause of severe lower respiratory tract disease in infants and young children worldwide, with estimated annual infection and mortality rates of 64 million and 160,000, respectively.1 In the U.S., RSV is the leading cause of hospitalization of infants.2 Despite the induction of post-infection immunity, repeat infection and lifelong susceptibility to RSV is common.3 Currently, there is no approved RSV vaccine available.

About RSV F Vaccine for Infants via Maternal Immunization

Novavax is developing a vaccine that targets the fusion protein, or F protein, of the RSV virus. The F protein has highly conserved amino acid sequences, called antigenic sites, which are the target of neutralizing antibodies and are believed to be ideal vaccine targets. Novavax’ genetically engineered novel F protein antigen exposes a range of these antigenic sites, and can evoke immune responses to them in human vaccine recipients. In a previous Phase 2 clinical trial of the RSV F Vaccine, which assessed the transplacental transfer of maternal antibodies induced by the vaccine, immunized women demonstrated meaningful fold rises in anti-F IgG, palivizumab-competing antibodies and microneutralization titers. In addition, infants’ antibody levels at delivery averaged 90-100% of the mothers’ levels, indicating efficient transplacental transfer of antibodies from mother to infant.

About Influenza

Influenza is a world-wide infectious disease that causes illness in humans with symptoms ranging from mild to life-threatening or even death. Serious illness occurs not only in susceptible populations such as infants, young children and older adults, but also in the general population largely because of infection by continuously evolving strains of influenza which can evade the existing protective antibodies in humans. An estimated one million deaths each year are attributed to influenza.4 Current estimates for seasonal influenza vaccine growth in the top seven markets (U.S., Japan, France, Germany, Italy, Spain and UK), show a potential increase from approximately $3.2 billion in 2015 to $5.3 billion by 2025.5

About NanoFlu

NanoFlu is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA amino acid protein sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu contains Novavax’ patented saponin-based Matrix-M adjuvant, which has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes.

On May 9, 2018 Savara Inc. (NASDAQ:SVRA), an orphan lung disease company, reported financial results for the first quarter ended March 31, 2018 and provided a business update (Press release, Savara, MAY 9, 2018, View Source [SID1234526406]).

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

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

                  Schedule Your 30 min Free Demo!

"It has been an incredibly productive quarter, including the launch of two new clinical studies, OPTIMA and IMPALA-X, with our lead product candidate Molgradex," stated Rob Neville, chief executive officer of Savara. "With a total of four ongoing clinical studies, our focus for the remainder of the year will continue to be the advancement of our core programs, while actively exploring the further expansion of our pipeline."

Upcoming Milestones and Recent Developments

Anticipating completion of enrollment in the Molgradex Phase 3 IMPALA study in Q3 2018. The IMPALA study is evaluating our inhaled formulation of granulocyte-macrophage colony-stimulating factor, or GM-CSF, for the treatment of autoimmune pulmonary alveolar proteinosis, or aPAP. At the end of Q1, enrollment was at 96 patients out of a total target of 135 patients, with completion of enrollment currently on track for Q3 2018 and topline data anticipated in Q2 2019.

Anticipating completion of enrollment in the AeroVanc Phase 3 AVAIL study in Q1 2019. The AVAIL study is evaluating our vancomycin hydrochloride inhalation powder for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in individuals living with cystic fibrosis. At the end of Q1, enrollment was at 62 patients out of a total target of 200 patients, with completion of enrollment currently on track for Q1 2019 and topline data anticipated in H2 2019.

Anticipating completion of enrollment in the Molgradex Phase 2a OPTIMA study in Q3 2018. The OPTIMA study is evaluating our inhaled GM-CSF for the treatment of nontuberculous mycobacterial (NTM) lung infection. The study was initiated in mid-March 2018 and is expected to be completed in Q3 2018. As OPTIMA is an open-label study, depending on enrollment and other factors, interim results from the study may be provided during 2018.

Announcing first patient enrolled in the Molgradex IMPALA-X safety extension study. The IMPALA-X study is an open-label, multicenter study designed to determine the long-term safety and utilization of Molgradex in patients with aPAP. IMPALA-X offers patients the opportunity to continue treatment with Molgradex for up to three years after completion of the pivotal Phase 3 IMPALA study. Savara plans to initiate the IMPALA-X study in a rolling fashion in 12 of the countries participating in the IMPALA study by the end of 2018.
First Quarter Financial Results

Savara’s net loss attributable to common shareholders for the three months ended March 31, 2018 was $26.8 million, or $(0.88) per share, compared with a net loss attributable to common shareholders of $5.0 million, or $(1.65) per share, for the first quarter of 2017, which represents the historical financial information of the private company Savara Inc., which completed its merger transaction with Mast Therapeutics, Inc. on April 27, 2017 (the "Merger").

Research and development expenses were $8.5 million for the three months ended March 31, 2018, compared with $2.9 million for the first quarter of 2017. The increase was primarily due to $2.7 million in increased expenses associated with the development of Molgradex, including the expansion of the IMPALA study in the U.S. and other countries and the commencement of the Molgradex NTM study, an increase of $2.2 million in AeroVanc study costs related to Phase 3 activities, and $0.7 million related to non-recurring milestone payments relating to the Aironite study acquired in the Merger, which was not a part of our product pipeline in the first quarter of 2017.

General and administrative expenses for the three months ended March 31, 2018 were $1.8 million, compared with $1.7 million for the first quarter of 2017.

Also, during the three months ended March 31, 2018, we recognized a one-time noncash impairment charge of $21.7 million to the carrying value of IPR&D related to the Aironite product candidate assumed in the Merger due to the unfavorable results from a Phase 2 study in which Aironite failed to meet the endpoints of the study and showed limited effectiveness in patients. We do not intend to further support or pursue the development of Aironite.

We reported a $4.6 million tax benefit for the first quarter of 2018 related to the reversal of a deferred tax liability resulting from the impairment of IPR&D acquired in the Merger.

As of March 31, 2018, Savara had cash, cash equivalents and short-term investments of approximately $85.0 million. The company’s operating expenses for the first quarter of 2018 were approximately $32.1 million which included the one-time noncash impairment charge of $21.7 million to the carrying value of IPR&D acquired in the Merger. Savara ended the first quarter of 2018 with approximately $14.9 million in debt.

Conference Call and Webcast
Savara will hold a conference call today beginning at 5:30 p.m. Eastern Time / 4:30 p.m. 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 should request the Savara Inc. call. A live webcast of the conference call will be available online from the Investors section of Savara’s website at View Source Replays of the webcast will be available on Savara’s website for 30 days and a telephone replay will be available through May 16th, 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. by entering replay access code 10119917.

On May 9, 2018 Clovis Oncology, Inc. (Nasdaq: CLVS) reported that Patrick J. Mahaffy, Chief Executive Officer and President, will present at the Bank of America Merrill Lynch Healthcare Conference 2018 on Wednesday, May 16, 2018 at 11:20 AM local time (Press release, Clovis Oncology, MAY 9, 2018, View Source [SID1234526405]). The conference will be held at the Encore Hotel in Las Vegas, Nevada.

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

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

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation can be accessed through the investor relations section of the Company’s website at www.clovisoncology.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 30 days.