On August 6, 2020 PDL BioPharma, Inc. ("PDL" or "the Company") (Nasdaq: PDLI) reported financial results for the three and six months ended June 30, 2020 and provides an update on important milestones achieved in the execution of its monetization plan (Press release, PDL BioPharma, AUG 6, 2020, View Source [SID1234563081]):

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"I am pleased with the significant progress we have made over the past couple of months in the execution of our monetization strategy," commented PDL’s President and CEO Dominique Monnet. "We continue to focus on maximizing the net proceeds from the monetization of our assets for our stockholders, and I believe the actions we have taken so far have served them well. I would like to thank the PDL Board and team, our advisors and our LENSAR and Noden colleagues for their continued engagement, dedication and support."

•On May 21, 2020, the Company distributed 100% of its shares of Evofem Biosciences, Inc. ("Evofem") common stock to the PDL stockholders.
•On July 17, 2020, the Company announced that its majority owned subsidiary, LENSAR, Inc. ("LENSAR"), confidentially submitted a registration statement on Form 10 to the Securities and Exchange Commission relating to a potential spin-off of LENSAR as a stand-alone publicly traded company. The Company continues to pursue various strategic alternatives for LENSAR in addition to a spin-off.
•On July 30, 2020, the Company announced the signing of a definitive agreement for the sale of 100% of the outstanding stock in its wholly owned subsidiaries Noden Pharma DAC and Noden Pharma USA (collectively "Noden"). The total value of the transaction will result in payments to PDL of up to $48.25 million in cash. Upon closing, which we expect to occur by mid-August, PDL will be released of its guarantee to Novartis under Noden’s supply agreement.

In February 2020, the Board of Directors of PDL BioPharma (the "Board") approved a Plan of Complete Liquidation ("Plan of Liquidation"). The Company is seeking stockholder approval at its 2020 Annual Meeting of Stockholders on August 19, 2020 to dissolve the Company under Delaware state law. If stockholders approve the dissolution proposal, the Board would have the authority to cause the Company to file a Certificate of Dissolution to begin the process of winding down and dissolving the Company if and when the Board decides that it would serve best the interest of PDL stockholders.

As announced previously, the Company has engaged financial advisors and initiated processes either to sell its remaining assets separately or to sell the Company as a whole. The Company intends to pursue its monetization strategy in a disciplined and cost-effective manner seeking to maximize net proceeds to stockholders. While the Company cannot provide a definitive

timeline for the liquidation process, it is targeting to complete the monetization or distribution of its key assets over the next 12 months.

Discontinued Operations Classified as Assets Held for Sale

As a result of the Company’s plans to monetize its assets and the actions put in place in the first quarter of 2020, as of March 31, 2020 the assets held for sale and discontinued operations criteria were met for the Company’s royalty assets and for its Pharmaceutical segment, which consisted of Noden. The royalty assets are a component of the Income Generating Assets segment. In the second quarter of 2020, upon the distribution of the Evofem common stock to the Company’s stockholders, the discontinued operations criteria were met for the Strategic Positions segment. The Strategic Positions segment was comprised solely of the Company’s investment in Evofem.

During the period in which a component meets the assets held for sale and discontinued operations criteria, an entity must present the assets and liabilities of the discontinued operation separately in the asset and liability sections of the balance sheet for the current and comparative reporting periods. The prior period balance sheet is reclassified for the held for sale items. For statements of operations, the current and prior periods report the results of operations of the component in discontinued operations.

Second Quarter Financial Highlights

•Total revenues were $5.2 million, consisting primarily of LENSAR product, lease and service revenues.
•LENSAR revenues were $5.1 million, a decrease of 31% over the prior-year period, with procedure volume also declining 31%.
•Net cash from all royalty rights was $11.5 million, down 43% from $20.1 million for the prior-year period.
•Revenue from our Pharmaceutical segment was $8.2 million, compared with $10.4 million for the prior-year period.
•GAAP net loss was $50.0 million. Non-GAAP net loss was $23.0 million. A reconciliation of GAAP to non-GAAP financial results can be found in Table 4 at the end of this news release.

Revenue Highlights

•Total revenues for the second quarter of 2020 were $5.2 million and consisted primarily of LENSAR product, lease and service revenues.
•Product revenue from LENSAR was $5.1 million, a 31% decrease from the second quarter of 2019. LENSAR procedure volume for the second quarter of 2020 also declined 31% from the prior-year period, primarily due to lower system sales and procedures driven by the negative impact of the COVID-19 pandemic and the associated decline in elective surgical procedures. LENSAR operating results are expected to improve as elective surgical procedures progressively ramp to pre-COVID-19 levels as the pandemic subsides.
•Total revenues for the first half of 2020 were $11.2 million, compared with $14.2 million for the first half of 2019.
◦Revenues from LENSAR for the six months ended June 30, 2020 decreased by $3.0 million, or 21%, to $11.1 million from $14.1 million for the six months ended June 30, 2019. LENSAR procedure volume for the six months ended June 30, 2020 declined by 18% from the prior-year period.

Operating Expense Highlights

•Operating expenses from continuing operations of the Company include general and administrative expenses for corporate overhead. A significant amount of these costs had historically not been allocated to individual segments.
•Operating expenses for the three months ended June 30, 2020 were $19.0 million, a $2.3 million increase from $16.7 million for the three months ended June 30, 2019. The increase was primarily a result of:
◦higher general and administrative expenses, primarily due to increased professional fees associated with the ongoing monetization efforts,
◦higher research & development expenses in our Medical Devices segment as LENSAR pursues its next-generation workstation, ALLY, which integrates an enhanced femtosecond laser with a phacoemulsification system in a compact, mobile workstation, partially offset by
◦lower cost of product revenue, due to decreased sales in our Medical Devices segment, and
◦lower sales and marketing expenses in our Medical Devices segment.

•Operating expenses for the six months ended June 30, 2020 were $56.7 million, a $25.1 million increase from $31.6 million for the six months ended June 30, 2019. The increase was primarily a result of:
◦provisions under our Wind-Down Retention Plan, which, as a result of the adoption of the Plan of Liquidation, accelerated the vesting of outstanding stock awards for employees in the first quarter of 2020,
◦higher general and administrative expenses of $5.5 million, or 32% from the prior period, primarily due to increased professional fees, and
◦higher research & development expenses in our Medical Devices segment, partially offset by
◦lower cost of product revenue, due to decreased sales in our Medical Devices segment.

Discontinued Operations Highlights

•Loss from discontinued operations for the three months ended June 30, 2020 was $37.4 million, a $40.9 million decrease from the $3.5 million of income recognized for the three months ended June 30, 2019. The change was primarily a result of:
◦A $58.4 million change in the fair value of our equity affiliate from an unrecognized gain of $45.5 million in the three months ended June 30, 2019, compared with a $12.9 million loss in the three months ended June 30, 2020.
◦A $16.8 million loss recorded in the three months ended June 30, 2020 associated with reducing the estimated fair value of Noden as informed by negotiations and terms for the disposition of the entity.
◦A $2.2 million, or 22%, decline in revenue from our Pharmaceutical segment for the three months ended June 30, 2020, compared with the same period in the prior year. The decrease in revenue from our Pharmaceutical segment is primarily due to lower net revenues in the United States.
▪The decrease in revenue from our Pharmaceutical segment in the U.S. for the three months ended June 30, 2020 is due to the increased sales of our authorized generic and lower sales of our branded Tekturna, compared with the second quarter of 2019.
▪U.S. market share for branded Tekturna and the authorized generic of Tekturna of approximately 65% at June 30, 2020 declined from 68% as of March 31, 2020.

These amounts were partially offset by:
◦Revenue from our royalty right assets of negative $16.3 million in the three months ended June 30, 2020, compared with a negative $40.4 million for the three months ended June 30, 2019. The difference was primarily due to a larger decrease in fair value in the second quarter of 2019 primarily resulting from the $60.0 million AcelRx write-down, compared with a $22.9 million write down in the three months ended June 30, 2020 for certain royalty assets, as informed by bids received during our monetization process.
▪The royalty right assets in our Income Generating Assets segment generated cash flows of $11.5 million and a loss from the net change in fair value of $27.8 million in the three months ended June 30, 2020, compared with cash flows of $20.1 million and a loss in the net change in fair value of $60.5 million in the three month period ended June 30, 2019.
▪See Table 3 for a rollforward of royalty assets for the second quarter of 2020, compared with the comparable period in 2019.

•Loss from discontinued operations for the six months ended June 30, 2020 was $50.2 million, a $68.7 million decrease from the $18.6 million of income recognized for the six months ended June 30, 2019. The unfavorable change was primarily a result of:
◦A $72.2 million change in the fair value of our equity affiliate from an unrecognized gain of $45.5 million in the six months ended June 30, 2019, compared with a $26.7 million loss in the six months ended June 30, 2020.
◦A $23.5 million write down of our Pharmaceutical segment in the current year due to a decrease in the estimated fair value of the entity.
◦A $7.2 million, or 24%, decline in revenue from our Pharmaceutical segment for the six months ended June 30, 2020, compared with the same period in the prior year.

3

These amounts were partially offset by:
◦Revenue from our royalty right assets in our Income Generating Assets segment of negative $6.9 million for the six months ended June 30, 2020, compared with negative $28.1 million for the corresponding period of the prior year. The difference was primarily due to a larger decrease in fair value in the second quarter of 2019 resulting from the $60 million AcelRx write-down, compared with the six months ended June 30, 2020, which includes the fair value adjustments for certain royalty assets as informed by the bids received during our monetization process.
▪The royalty right assets generated cash flows of $25.0 million in the current period, compared with $32.7 million in the prior-year period.

Other Financial Highlights

•On a GAAP basis, the net loss attributable to PDL’s shareholders for the second quarter of 2020 was $50.0 million, or $0.43 per share, compared with a GAAP net loss attributable to PDL’s shareholders of $4.4 million, or $0.04 per share, for the prior-year period. Non-GAAP net loss attributable to PDL’s shareholders was $23.0 million for the second quarter of 2020, compared with non-GAAP net income for PDL’s shareholders of $12.7 million for the second quarter of 2019.
•On a GAAP basis, the net loss attributable to PDL’s shareholders for the first half of 2020 was $81.7 million, or $0.68 per share, compared with GAAP net income attributable to PDL’s shareholders of $2.3 million, or $0.02 per share, for the prior-year period. Non-GAAP net loss attributable to PDL’s shareholders was $29.7 million for the first half of 2020, compared with non-GAAP net income for PDL’s shareholders of $24.5 million for the first half of 2019.
•PDL had cash and cash equivalents from continuing operations of $105.4 million as of June 30, 2020, compared with $169.0 million as of December 31, 2019.
◦The $63.6 million reduction was primarily the result of common stock repurchases of $39.4 million, the net cash used for the repurchase of convertible debt of $18.0 million and net cash used in operations of $33.8 million. This reduction was partially offset by the proceeds from royalty rights of $25.0 million.

Stock and Convertible Note Repurchase Program

•In January 2020, PDL began repurchasing shares of its common stock in the open market pursuant to the 10b5-1 program entered into in December 2019 following a $275 million repurchase plan approved by the Board. In the first half of 2020, the Company acquired 12.3 million shares of its common stock for $39.4 million, at an average cost of $3.20 per share, including commissions.
•Under this same program, in the first half of 2020, the Company also repurchased $15.9 million par value of convertible notes.
•In consideration of the impact and uncertainty introduced by the COVID-19 pandemic on the Company’s monetization process, the Company discontinued its 10b5-1 program on May 31, 2020.
•Through June 30, 2020, the total amount spent of the $275 million Board authorized repurchase program, including the value of the Company’s stock issued in connection with the December 2019 convertible debt exchange, was $213.0 million.
•As of July 31, 2020, the Company had approximately 114.0 million shares of common stock outstanding.

Conference Call and Webcast

PDL will hold a conference call to discuss financial results and provide a business update at 4:30 p.m. Eastern time today. Slides to accompany the conference call will be available in the Investor Relations section of View Source." target="_blank" title="View Source." rel="nofollow">View Source

To access the live conference call via phone, please dial (866) 777-2509 from the United States or (412) 312-5413 internationally. The conference ID is 10146007. A telephone replay will be available for one week beginning approximately one hour after the completion of the call and can be accessed by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada or (412) 317-0088 internationally. The replay passcode is 10146007.

To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of View Source and select "Events & Presentations."

On August 6, 2020 Monopar Therapeutics Inc. (Monopar or the Company) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company primarily focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported second quarter 2020 financial results and business updates (Press release, Monopar Therapeutics, AUG 6, 2020, View Source [SID1234563064]).

Second Quarter and Recent Highlights

Collaboration with NorthStar Medical Radioisotopes to Develop Potential Therapeutic for Severe COVID-19 and Other Respiratory Diseases

oMonopar and NorthStar entered into a 50/50 collaboration to develop potential Radio-Immuno-Therapeutics (RITs) to treat severe COVID-19.

oMonopar will provide its pre-IND stage humanized uPAR targeted monoclonal antibody, known as MNPR-101, and NorthStar will supply a cytotoxic radioisotope. The aim is to create a highly selective agent that has the potential to kill aberrantly activated cytokine-producing immune cells thought to be largely responsible for the severe lung injury that contributes to poor outcomes and death in patients with severe COVID-19.

oProvisional patent has been filed that covers novel compositions and uses of cytotoxic radioisotopes attached to antibodies that bind to uPAR, thereby creating precision targeted radio-immuno-therapeutics (uPRITs) for the treatment of severe COVID-19 and other respiratory diseases.

Validive Phase 2b/3 Clinical Trial and Camsirubicin Phase 2 Clinical Trial Continue on Schedule

oMonopar’s two clinical-stage programs both continue to be on schedule for first patient dosing in the second half of 2020.

Second Quarter Summary Financial Results

Results for the Quarter Ended June 30, 2020 Compared to the Quarter Ended June 30, 2019

Cash and cash equivalents as of June 30, 2020 were $12.5 million. Net loss for the three months ended June 30, 2020 was $1.4 million or $0.14 per share compared to net loss of $0.9 million or $0.10 per share for the three months ended June 30, 2019.
R&D expenses for the three months ended June 30, 2020 were $0.8 million, compared to $0.3 million, for the three months ended June 30, 2019. This represents an increase of $0.5 million primarily attributed to increases in camsirubicin and Validive clinical trial planning and materials manufacturing costs and increases in personnel expenses for three new R&D employees and higher salary and stock-based (non-cash) compensation for 2020.

General and Administrative (G&A) Expenses

G&A expenses for the three months ended June 30, 2020 were $0.6 million, compared to $0.6 million, for the three months ended June 30, 2019.

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On August 6, 2020 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported an update on its financials for the second quarter of 2020, and a corporate update (Press release, Aviragen Therapeutics, AUG 6, 2020, View Source [SID1234563062]).

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"With $140 million in cash at the end of July, Vaxart is now laser focused on preparing to enter a Phase 1 clinical trial with our lead COVID-19 vaccine candidate," said Andrei Floroiu, chief executive officer of Vaxart, "We believe the convenience of our oral tablet, coupled with the potential for better protection than that of injectable vaccines due to the activation of mucosal immunity, positions our COVID-19 vaccine as one of the most promising candidates for successful mass vaccination campaigns, both here in the US and abroad."

Corporate Highlights:

On May 20, 2020, Vaxart announced that it had selected its lead COVID-19 vaccine candidate to move into clinical trials.

Vaxart is continuing with its manufacturing collaboration with Emergent BioSolutions Inc. (Emergent) and KindredBio and is currently producing bulk cGMP vaccine in time for initiation of a Phase 1 clinical study during the second half of 2020.

On June 25, 2020, Vaxart announced the signing of a Memorandum of Understanding with Attwill Medical Solutions Sterilflow, LP ("AMS") affirming the parties’ intent to establish AMS as a resource for lyophilization development and large-scale manufacturing including tableting and enteric coating for the oral COVID-19 vaccine.

On June 26, 2020, Vaxart announced that its oral COVID-19 vaccine was selected to participate in a non-human primate challenge study, organized and funded by Operation Warp Speed.

The universal influenza vaccine collaboration study with Janssen has been completed and a report is being compiled for Janssen.

As of July 13, 2020, Vaxart had raised approximately $97 million in net proceeds through its At-The-Market facility (the ATM Program), which constituted the maximum offering price under the ATM Program.

Financial Results for the Fiscal Period Ended June 30, 2020

Vaxart reported a net loss of $9.0 million for the second quarter of 2020 compared to $5.6 million for the second quarter of 2019. The increase was mainly due to an increase in operating expenses. Net loss per share for the second quarter was $0.12 in 2020 compared to $0.39 in 2019, in part due to an increase in the number of shares outstanding.

Vaxart ended the quarter with cash and cash equivalents of $44.4 million compared to $29.9 million at March 31, 2020. The increase was primarily due to $14.4 million received for the exercise of common stock warrants, partially offset by $3.7 million of cash used in operations.

Revenue for the second quarter was $523,000 compared to $85,000 in the second quarter of 2019. The increase was principally due to the reversal of the reserve for sales returns for Relenza and revenue from our contract with Janssen.

Research and development expenses were $5.1 million for the second quarter compared to $3.7 million for the second quarter of 2019. The increase was mainly due to manufacturing expenses related to the COVID-19 vaccine candidate and higher stock-based compensation costs, partially offset by reductions in the cost of clinical trials for our norovirus vaccine candidate and in personnel costs after we ceased internal manufacturing as part of our December 2019 restructuring.

General and administrative expenses were $3.9 million for the second quarter compared to $1.4 million for the second quarter of 2019. The increase was mainly due to higher stock-based compensation costs and severance expenses related to our former Chief Executive Officer.

On August 6, 2020 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported financial results for the second quarter ended June 30, 2020, and provided an update on clinical development programs (Press release, Protagonist, AUG 6, 2020, View Source [SID1234563059]).

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"We have succeeded in bringing three differentiated candidates from de novo discovery into Phase 2 development," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "Each of these candidates has unique attributes that address specific unmet needs in diverse diseases, such as blood disorders with PTG-300 and inflammatory bowel disease with PTG-200 and PN-943. The hepcidin mimetic PTG-300 makes use of the iron homeostasis mechanism of a natural hormone and has demonstrated potential in the treatment of polycythemia vera. We expect to initiate a pivotal study for PTG-300 in 2021 after discussions with the regulatory agencies. PN-943 and PTG-200 are both oral, gut-restricted candidates for the potential treatment of inflammatory bowel disease. PTG-200 is an IL-23 receptor antagonist partnered with Janssen and is currently in a Phase 2 Crohn’s study. We recently initiated screening of patients for our Phase 2 study of PN-943 in ulcerative colitis. Finally, we are well financed and recently raised $122 million through a successful secondary offering and use of our ATM program, which enables us to support planned operations through mid-2023."

Product Development and Corporate Update
PTG-300: Injectable Hepcidin Mimetic for Polycythemia Vera and Other Blood Disorders

·Initial Phase 2 results reported in May 2020 in patients with polycythemia vera demonstrated clinically meaningful dose related control of hematocrit levels on individual patient basis.
·In June 2020, PTG-300 received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of polycythemia vera.
·Protagonist plans to host a webinar featuring presentations on clinical needs and market research on the potential opportunity for PTG-300 in polycythemia vera. The "PTG-300 Opportunity Update" webinar will be conducted Sept. 11, 2020. Details for participation will be publicly announced prior to the event.

PN-943: Oral Alpha-4-Beta-7 Integrin Antagonist for Inflammatory Bowel Disease

·Protagonist has initiated screening of prospective subjects in a global, randomized, double-blind, placebo-controlled Phase 2 study (the "IDEAL Study") evaluating safety, tolerability and efficacy of PN-943 in approximately 150 patients with moderate to severe active ulcerative colitis. Patients will be randomized in one of three arms (150 mg twice daily, 450 mg twice daily, or placebo) for 12 weeks of oral dosing followed by an extended treatment period of 40 weeks. During the extended treatment period all subjects will receive PN-943. The primary endpoint of the study is proportion of subjects achieving clinical remission at week 12 (as defined by rectal bleeding, stool frequency and endoscopic subscores of the Adapted Mayo Score) in the 450 mg twice daily treatment arm as compared to placebo. Secondary endpoints include additional clinical and safety assessments, as well as pharmacokinetic and pharmacodynamic measurements, and biomarker measurements related to disease activity.

Oral IL-23 Receptor Antagonists (Janssen Biotech and Protagonist Collaboration)

·Janssen Biotech is conducting a global Phase 2 study of PTG-200 (or JNJ-67864238) in moderate-to-severe Crohn’s disease.
·Joint research efforts are underway to identify second-generation oral IL-23 receptor antagonists for multiple indications.

Financial Update

·During May 2020, the Company successfully raised $105.7 million net of underwriting and offering expenses in an oversubscribed secondary offering issuing 8,050,000 shares at $14.00 per share.
·During the second quarter of 2020, the Company issued 1.2 million shares through its at-the-market (ATM) program and raised $16.8 million, at an average price of $14.02 per share.

Financial Results

·Cash, cash equivalents and marketable securities as of June 30, 2020, were $208.7 million. Protagonist estimates sufficient financial resources from its cash, cash equivalents, marketable securities and access to its debt facility to fund its currently planned operating and capital expenditures through mid-2023.
·License and collaboration revenues were $6.2 million and $9.9 million for the second quarter and six months ended 2020, respectively, in comparison to $(8.2) million and $(6.6) million reported for the same periods of 2019. The increase in revenue was due mainly to the previously reported 2019 one-time cumulative adjustment related to the application of revenue recognition principles following the May 2019 amendment of the Janssen Biotech collaboration agreement that had reduced revenue by $9.4 million for the three and six months ended June 30, 2019.
·Research and Development ("R&D") expenses for the three and six months ended June 30, 2020, were $20.3 million and $39.0 million, respectively, as compared to $19.4 million and $31.8 million for the same periods of 2019. These variances were primarily due to increased activities in advancing our ongoing clinical trial for polycythemia vera with PTG-300, preparedness for PN-943 Phase 2 study in ulcerative colitis, and our IL-23 receptor antagonist research collaboration activities with Janssen Biotech.

·General and Administrative ("G&A") expenses for the three and six months ended June 30, 2020, were $4.2 million and $8.8 million, respectively, as compared to $3.9 million and $7.6 million for the same periods of 2019. The increases were primarily due to increases in salaries, insurance expense and professional services to support the growth in our operations.
·Net loss for the three and six months ended June 30, 2020, was $19.4 million and $39.5 million or a net loss of $0.59 per share and $1.31 per share, respectively, as compared to a net loss of $29.2 million and $43.3 million, or a net loss of $1.18 per share and $1.77 per share, for the same periods of 2019.

On August 6, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2020. In addition, the Company provided a clinical and business update (Press release, Syndax, AUG 6, 2020, View Source [SID1234563058]).

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"We are very pleased that the FDA has agreed to several proposed changes to the Phase 1 portion of AUGMENT-101 which build on emerging clinical data and help us maximize SNDX-5613’s potential in as many appropriate patients as possible," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "This includes focusing enrollment exclusively on patients with MLL-r and NPM1 mutant acute leukemias, the ability to expand dose cohorts that demonstrate efficacy, and inclusion of pediatric patients as young as one month, which has long been a key component of our overall strategy for SNDX-5613. We remain on track to identify a recommended Phase 2 dose by the end of this year, with full Phase 1 data now anticipated in early 2021."

Dr. Morrison added, "Progress also continues with the Phase 1/2 trial of axatilimab, our anti-CSF-1R monoclonal antibody, in patients with cGVHD, with Phase 1 results expected in the fourth quarter. We are actively committed to helping people with cancer live longer and better than ever before and look forward to further advancing this mission throughout the balance of the year."

Pipeline Updates

SNDX-5613

Syndax reported that the U.S. Food and Drug Administration (FDA) has agreed to several enhancements to the Phase 1 portion of the AUGMENT-101 protocol. AUGMENT-101 is the Company’s Phase 1/2 open-label trial designed to evaluate the safety, tolerability, pharmacokinetics and efficacy of orally administered SNDX-5613, its potent, highly selective oral menin inhibitor, in patients with acute leukemias. As recently reported, the Phase 1 dose escalation portion of AUGMENT-101 was separated into two cohorts based on concomitant treatment with a strong CYP3A4 inhibitor. Arm A will enroll patients not receiving a strong CYP3A4 inhibitor, while Arm B will enroll patients receiving a strong CYP3A4 inhibitor.

Supported by initial clinical data, as well as new insights from emerging data in the pediatric compassionate use setting, the Company will enact the following enhancements to the Phase 1 portion of the trial: focusing enrollment exclusively on patients with mixed lineage leukemia rearranged (MLL-r) and nucleophosmin (NPM1) mutant acute leukemias; backfilling any dose escalation cohort up to a total of 12 patients in either Arm A or Arm B if efficacy has been observed at that dose level; and expansion of enrollment of pediatric patients over one month old. The Company continues to anticipate identifying a recommended Phase 2 dose by the end of 2020, with full data from the amended Phase 1 portion expected in early 2021. SNDX-5613 was recently granted Orphan Drug Designation for the treatment of adult and pediatric AML by the FDA.

The Company recently participated in the U.S. FDA’s June 2020 meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (pedsODAC) to discuss the clinical development plan for SNDX-5613 in pediatric patients. A replay of the pedsODAC meeting, which was intended to improve and encourage the development of oncology and hematology drugs for pediatric use, as well as a copy of the Company’s briefing package, can be found here.

At the 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I in April, Syndax announced initial clinical data from the AUGMENT-101 trial. Data presented serve as the first clinical evidence that inhibition of the menin-MLL1 interaction can induce response in patients with MLL-r acute leukemias. The presentation also highlighted preclinical findings, including data published in Cancer Cell and Science magazine, supporting the potential of single-agent menin-MLL inhibition to serve as an effective intervention for both MLL-r acute leukemias and NPM1 mutant AML. A copy of the presentation is available on Syndax’s website under Publications, Menin-MLLr Inhibitors.

Axatilimab

Enrollment continues across the Company’s Phase 1/2 trial evaluating axatilimab, its anti-CSF-1R monoclonal antibody, for the treatment of chronic graft versus host disease (cGVHD). The Phase 1 portion continues to explore alternate dose and schedules, while the Phase 2 expansion is evaluating the benefit of treatment at 1 mg/kg every two weeks. The Company expects to present additional results from the Phase 1 trial in the fourth quarter of 2020.

Data from the Phase 1 trials exploring axatilimab, both as a monotherapy and in combination with IMFINZI (durvalumab) in patients with locally-advanced or metastatic solid tumors, were summarized in two oral presentations at the AACR (Free AACR Whitepaper) Virtual Annual Meeting I. The data indicate that axatilimab is tolerated well in solid tumor patients and provide evidence of its ability to deplete circulating pro-inflammatory monocytes. A recommended Phase 2 dose of axatilimab for the treatment of patients with solid tumors was determined as monotherapy and in combination with IMFINZI (durvalumab). A copy of each presentation is available on Syndax’s website under Publications, Axatilimab.

Entinostat

In May 2020, the Company reported final results of E2112, the Phase 3 clinical trial conducted by ECOG-ACRIN Cancer Research Group and sponsored by the National Cancer Institute, that evaluated the investigational compound entinostat, Syndax’s class I HDAC inhibitor, plus exemestane in patients with advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer. The trial did not achieve the primary endpoint of demonstrating a statistically significant overall survival benefit over hormone therapy alone. The Company has decided to deprioritize the entinostat program to focus resources on advancing the remainder of its pipeline.

Financial Update and Guidance

As of June 30, 2020, Syndax had cash, cash equivalents and short-term investments of $186.8 million and 44.1 million shares and share equivalents issued and outstanding which included 38.5 million shares of common stock and pre-funded warrants to purchase 5.6 million shares of common stock.

In May 2020, Syndax closed an underwritten public offering whereby the Company sold 6.4 million shares of common stock at a price of $18.00 per share. The aggregate net proceeds received by the Company were $107.9 million, net of underwriting discounts and commissions and estimated offering expenses payable by the Company.

Second quarter 2020 research and development expenses decreased to $10.9 million from $12.3 million for the prior year period. The second quarter decrease was primarily due to the impact of a $4.0 million expense for achievement of a clinical milestone associated with SNDX-5613 which was recognized and recorded in the second quarter of 2019. Excluding this milestone, research and development expenses in the second quarter of 2020 increased compared to the prior year period primarily due to an increase in clinical activities related to SNDX-5613 and axatilimab, and professional fees, partially offset by a decrease in clinical activities related to entinostat.

General and administrative expenses for the second quarter 2020 increased to $6.0 million from $3.5 million for the prior year period. The increase was primarily due to increased pre-commercialization activities in advance of the phase 3 breast cancer results for entinostat and employee related expenses.

For the three months ended June 30, 2020, Syndax reported a net loss attributable to common stockholders of $17.1 million or $0.42 per share compared to $14.9 million or $0.47 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the third quarter and second half of 2020. For the third quarter and second half of 2020, research and development expenses are expected to be $14 to $16 million and $30 to $35 million, respectively, and total operating expenses are expected to be $19 to $21 million and $40 to $45 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Thursday, August 6, 2020.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 8998873
Domestic Dial-in Number: (855) 251-6663
International Dial-in Number: (281) 542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.