On August 8, 2019 Pfenex Inc. (NYSE American: PFNX) is a clinical-stage development and licensing biotechnology company focused on leveraging its Pfēnex Expression Technology to develop and improve protein therapies for unmet patient needs (Press release, Pfenex, AUG 8, 2019, View Source [SID1234538435]). Using the patented Pfēnex Expression Technology platform, the Company has created an advanced pipeline of therapeutic equivalents, biologics, vaccines and biosimilars. Pfenex Inc. reported financial results for the second quarter ended June 30, 2019 and provided a business update.

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"During the past several months, there was significant progress made toward our key programs and partnerships, including PF708, our Jazz partnership, and our CRM197 partnerships with Merck and Serum Institute of India, which we believe are each on a path towards achieving pivotal clinical or regulatory milestones," said Eef Schimmelpennink, Chief Executive Officer of Pfenex. "Our lead program, PF708, continues to advance and the FDA has set a PDUFA date of October 7, 2019. We are pleased to announce that the FDA completed its mid-cycle review of our NDA for PF708 in May and did not identify any issues that require an advisory committee meeting. In anticipation of the FDA’s decision on our NDA for PF708, we continue to make progress on the launch readiness planning with our commercial partner Alvogen, which includes producing commercial materials and finalizing overall commercial strategy plans."

"We are very pleased with the progress by Jazz on PF743 (JZP-458). Jazz announced in their second quarter 2019 earnings that they completed a successful Phase 1 study and expect to initiate a single arm pivotal Phase 2/3 study later this year," mentioned Schimmelpennink, "similarly, we are progressing well with our development of PF745 and look forward to future updates on this program."

"We are moving ahead with our long-term strategy that further leverages our Pfēnex Expression Technology platform and expands and evolves our pipeline with new products and partnership opportunities. Our recently announced collaboration with Arcellx to advance proprietary sparX proteins that activate, silence and reprogram Antigen-Receptor Complex T cell-based therapies fits that strategy well. It provides us with another opportunity create value and enables us to further evolve the company towards novel technologies."

"This is an exciting time for Pfenex, as we harness near- and long-term opportunities to drive value for our shareholders. A combination of the progress made with our existing programs and new focus on R&D activities has attracted several new notable members of the scientific community to join our company’s executive team, board of directors and scientific advisory board. Our goal is to continue to build on this momentum and generate greater awareness around our platform technology and its broad potential for new programs and development partnerships," concluded Schimmelpennink.

Business Review and Update

PF708 therapeutic equivalent to Forteo (teriparatide)

PF708 is being developed as a therapeutic equivalent candidate to Forteo, which is approved and marketed by Eli Lilly & Co. for the treatment of osteoporosis in certain patients with a high risk of fracture and achieved $1.6 billion in global product sales in 2018.

The PF708 new drug application (NDA) has reached the Food and Drug Administration (FDA) mid-cycle review milestone. The FDA has set a PDUFA date of October 7, 2019 for this NDA Pfenex believes PF708 is on track for a potential commercial launch in the United States as early as the fourth quarter of 2019, subject to FDA approval, final commercial strategic decisions made by Pfenex’s partner Alvogen, and other factors.

Pfenex is also seeking an "A" therapeutic equivalence designation for PF708 to Forteo, which may permit PF708 to be automatically substituted for Forteo. Pfenex submitted the protocol for the comparative human factors study for FDA feedback, but the Agency recently informed Pfenex of its view that it might be premature to comment on the protocol until the details of the PF708 labeling are agreed to, which may be near the PF708 PDUFA date of October 7, 2019. Pfenex has engaged in a discussion with the FDA around this position. Depending on the outcome, it may delay the start of the study, submitting the study results to the FDA, and potentially obtaining a therapeutic equivalence rating.

Pfenex’s development and licensing partner for PF708, Alvogen, has assumed responsibility to manufacture and commercialize PF708 in the United States, assuming FDA approval, at Alvogen’s own cost and expense. Pfenex and Alvogen are working together on implementing initial stages of the commercialization strategy for PF708 in preparation for a U.S. launch following the PDUFA date.

In May, the European Medicines Agency (EMA) accepted the Marketing Authorization Application for PF708, which was submitted by Pfenex’s partner Alvogen as a biosimilar to Forsteo in the treatment of osteoporosis. Alvogen expects to receive initial comments on the application in the third quarter of 2019. If EMA approval is received, PF708 will be authorized for marketing in all 28 member states of the EU. Under the licensing agreement with Pfenex and Alvogen, Alvogen’s European distribution partner, Theramex, a leading global specialty pharmaceutical company dedicated to women’s health, will initiate sales of PF708, if the EMA approval is received.

Jazz Collaboration Agreement

Through its collaboration with Jazz Pharmaceuticals, Pfenex has completed the process development of PF743, a recombinant crisantaspase, and the development is ongoing for PF745, a recombinant crisantaspase with half-life extension technology. Pfenex believes its success on these programs further demonstrate the unique capabilities of Pfenex’s platform technology.

Jazz announced that they successfully completed a Phase 1 study for PF743 (JZP-458) and expect to initiate a single arm pivotal Phase 2/3 study later this year. In parallel we continue to advance the development of PF745 and are pleased with the progress we are making.

Pfenex believes these programs could be eligible to achieve certain payments in 2019 of the $29.5 million in development milestone payments available under its agreement with Jazz Pharmaceuticals. Under the agreement, Pfenex is eligible to receive an aggregate total of $224.5 million in development and sales milestone fees, of which $188.5 million is still eligible to be received. Of this $188.5 million, $29.5 million are development milestones, $34.0 million are regulatory milestones and $125.0 million are sales milestones. Pfenex may also be eligible to receive tiered royalties on worldwide sales of any products resulting from the collaboration.

CRM197

CRM197 is a non-toxic mutant of diphtheria toxin. It is a well characterized protein and functions as a carrier for polysaccharides and haptens making them immunogenic. CRM197 is currently being used by Pfenex’s vaccine development focused pharmaceutical partners, including in multiple Phase 3 clinical studies by Merck and the Serum Institute of India Private Ltd (SIIPL) for such diseases as pneumococcal and meningitis bacterial infections.

Arcellx – sparX Protein Development Agreement

Pfenex has entered into a development, evaluation and license agreement with Arcellx which provides access to the Pfēnex Expression Technology platform to advance Arcellx’ s proprietary sparX proteins that activate, silence and reprogram Antigen- Receptor Complex T cell-based therapies. Under the terms of the agreement, Pfenex is eligible to receive development funding in addition to development, regulatory and commercial milestones ranging from $2.6 million to $18 million for each product incorporating a sparX protein expressed using the Pfēnex Expression Technology, as well as royalties on worldwide sales of any such products.

Pfenex expects revenue in the near term to be primarily related to monetizing its protein production platform through CRM197 product sales, commercial license agreements and service agreements, which may provide for various types of payments, including upfront payments, royalties on sales, milestone payments, intellectual property access fees and licensing fees.

Board of Directors

Pfenex recently announced the appointment of Lorianne Masuoka, MD to its Board of Directors. In addition to being a board-certified neurologist, she brings to the Board more than 20 years of experience building and expanding high value pipelines in the biopharmaceutical industry that have resulted in drug approvals and strategic alliances. Dr. Masuoka has successfully created and overseen high performing teams to lead the clinical development of new medicines, many with a focus in neurology, CAN and pain. As the company is evolving Sigurdur Olafsson has transitioned off the Board of Directors. Pfenex’s Board wants to thank Siggi for his valuable contributions to Pfenex over the last few years and welcomes Lorianne as a valuable counselor to the Pfenex executive team as they further leverage the Pfēnex Expression Technology platform to build out the Company’s development pipeline.

Scientific Advisory Board

In April Pfenex announced that Dr. Robert Peach joined Pfenex’s Scientific Advisory Board. Dr. Peach has over 25 years of drug discovery and development experience in the pharmaceutical and biotechnology industry. In 2009, he co-founded Receptos, becoming Chief Scientific Officer and raised approximately $59 million in venture capital and approximately $800 million in an IPO and three subsequent follow-on offerings. In August 2015, Receptos was acquired by Celgene for $7.8 billion. Dr. Peach has also held senior executive and scientific positions in other companies, including Apoptos, Biogen Idec, IDEC and Bristol-Myers Squibb. His extensive drug discovery and development experience in autoimmune and inflammatory diseases and cancer has resulted in multiple drugs entering clinical trials and three registered drugs.

Financial Highlights for the Second Quarter 2019

Total Revenue decreased by $1.4 million, or 33%, to $2.8 million in the three-month period ended June 30, 2019, compared to $4.2 million in the same period in 2018. The decrease in revenue was primarily due to completion of revenue amortization for Pfenex’s Jazz collaboration agreement, as well as a decrease in revenue related to its BARDA program. This was partially offset by increased service revenue and sales of Pfenex’s CRM197 product.

Cost of Revenue increased by approximately $0.2 million, or 21%, to $1.1 million in the three-month period ended June 30, 2019, compared to $0.9 million in the same period in 2018. The increase was primarily due to greater sales of Pfenex’s CRM197 product, partially offset by decreased activity related to its BARDA program.

Research and development expenses decreased by approximately $5.9 million, or 55%, to $4.8 million in the three-month period ended June 30, 2019, compared to $10.7 million in same period in 2018. The decrease is primarily due to expenses incurred in the second quarter of 2018 for Pfenex’s Phase 3 clinical trial and regulatory activities related to its lead drug candidate PF708. Additionally, in the second quarter of 2019, R&D expenses were offset by $1.2 million for certain costs Alvogen agreed to reimburse the Company for in connection with quality, manufacturing and supply chain activities for the Company’s PF708 collaboration.

Selling, general and administrative expenses increased by approximately $1.0 million, or 25%, to $4.6 million in the three-month period ended June 30, 2019, compared to $3.6 million in the same period in 2018. The increases were primarily due to an increase in expenses related to IP legal, consulting, and the expansion of business development efforts.

Cash and cash equivalents as of June 30, 2019, were $41.6 million. Pfenex believes that its existing cash and cash equivalents and cash inflow from operations will be sufficient to meet Pfenex’s anticipated cash needs for at least the next 12 months, including all necessary activities leading up to and including potential approval of PF708 in the United States in the fourth quarter of 2019, subject to FDA approval, final commercial strategic decisions made by Pfenex’s partner Alvogen and other factors.

Conference Call Information

The Pfenex management will host a conference call and webcast today at 4:30 PM Eastern Time. Participants may access the call by dialing 866-376-8058 (Domestic) or 412-542-4131 (International). The call will also be webcast and can be accessed from the Investors section of the Company’s website at www.pfenex.com or View Source

A replay of the call will also be available through August 16th. Participants may access the replay of the call by dialing 877-344-7529 (Domestic) or 412-317-0088 (International) and providing the conference ID number: 10133889.

On August 8, 2019 Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, reported financial results for the second quarter ended June 30, 2019, and provided an update on the Company’s business and product development programs (Press release, Reata Pharmaceuticals, AUG 8, 2019, View Source [SID1234538451]).

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Product Development Updates

Phase 2/3 CARDINAL Trial of Bardoxolone in Alport Syndrome

The Phase 3 portion of CARDINAL is an international, multi-center, randomized, double-blind, placebo-controlled trial studying the safety and efficacy of bardoxolone methyl (bardoxolone) in patients with chronic kidney disease caused by Alport syndrome. Enrollment in the pivotal Phase 3 portion of CARDINAL was completed last year with 157 patients. We expect to announce one-year, top-line results from CARDINAL in the second half of 2019. The U.S. Food and Drug Administration (FDA) has provided guidance that an improvement in retained estimated glomerular filtration rate (eGFR) versus placebo after 48 weeks of treatment and a four-week drug withdrawal period may support accelerated approval under subpart H. Data demonstrating an improvement versus placebo in retained eGFR after two years may support full approval. No safety concerns have been reported by the data monitoring committee.

MOXIe Trial of Omaveloxolone in Friedreich’s Ataxia

MOXIe is a two-part, international, multi-center, randomized, double-blind, placebo-controlled registrational trial studying the safety and efficacy of omaveloxolone in patients with Friedreich’s ataxia (FA). Enrollment in the pivotal part 2 of MOXIe was completed last year with 103 patients, and we expect to announce top-line data in the second half of 2019. The FDA has provided guidance that an analysis of modified Friedreich’s Ataxia Rating Scale (mFARS) scores demonstrating an improvement versus placebo after 48 weeks of omaveloxolone treatment may support submission of a New Drug Application for omaveloxolone for the treatment of FA. No safety concerns have been reported by the data monitoring committee.

Phase 3 FALCON Trial of Bardoxolone in Autosomal Dominant Polycystic Kidney Disease

We announced in May 2019 that the first patient was enrolled in a registrational Phase 3 trial called FALCON, an international, multi-center, randomized, double-blind, placebo-controlled trial studying the safety and efficacy of bardoxolone in approximately 300 patients with autosomal dominant polycystic kidney disease. The FDA has provided guidance that an improvement in retained eGFR versus placebo at one year may support accelerated approval under subpart H, and that data demonstrating an improvement versus placebo in retained eGFR after two years may support full approval.

Phase 3 CATALYST Trial of Bardoxolone in Connective Tissue Disease-Associated Pulmonary Arterial Hypertension

We are conducting the pivotal Phase 3 CATALYST trial of bardoxolone in patients with pulmonary arterial hypertension associated with connective tissue disease, an often-fatal manifestation of many types of autoimmune disease, including systemic sclerosis (scleroderma) and systemic lupus erythematosus. The trial will enroll approximately 200 patients, with top-line data expected in the first half of 2020.

Selected Clinical Milestones in 2019

Pivotal CARDINAL data in the second half of 2019
Pivotal MOXIe data in the second half of 2019
Financial Highlights

The Company incurred total expenses of $41.5 million for the quarter ended June 30, 2019, with research and development accounting for $29.6 million. This compares to total expenses of $34.2 million for the same period of the year prior, when research and development accounted for $23.4 million. We reported a net loss of $34.4 million or $1.14 per share for the quarter ended June 30, 2019. This compares to a net loss of $28.2 million or $1.08 per share in the same period of the year prior.

The net loss for the three-month period compared to the year prior is primarily driven by an increase in expenses while revenue remained consistent to the year prior. Higher expenses were driven by an increase in research and development expenses due to clinical, manufacturing, and medical affairs activities, and an increase in personnel expenses to support growth of our development activities.

We incurred total expenses of $77.8 million for the six month period ended June 30, 2019, with research and development accounting for $55.7 million. This compares to total expenses of $62.4 million for the same period of the year prior, when research and development accounted for $44.8 million. We reported a net loss of $63.5 million or $2.12 per share for the six month period ended June 30, 2019. This compares to a net loss of $24.1 million or $0.92 per share in the same period of the year prior.

The increase in net loss for the six month period is driven primarily by both an increase in expenses and a decrease in revenue. Higher expenses were driven by an increase in research and development expenses due to clinical, manufacturing, and medical affairs activities, and an increase in personnel expenses to support expanded development activities. Revenue to date has primarily been related to license and collaboration agreements entered into during 2009, 2010, and 2011. The decrease in revenue was primarily due to an increase in revenue recognized in the first quarter of 2018 from the portion of a $30 million milestone from Kyowa Kirin Company that was included in the transaction price for which we did not have a similar event during 2019.

Our cash-based operating expenses, a non-GAAP measure, were $36.8 million and $68.7 million for the three and six months ended June 30, 2019, respectively. This compares to $31.6 million and $57.1 million for the same period of the year prior. Cash-based operating expenses for the quarters ended June 30 and March 31, 2019, were $36.8 million and $31.9 million, respectively. The increase in cash-based operating expenses for the three months ended June 30, 2019, were driven by increased manufacturing and clinical activities, as well as increased personnel costs to support growth in our development activities. We expect our cash-based operating expenses to continue to increase in the future as we advance bardoxolone and omaveloxolone through ongoing and future clinical trials, scale manufacturing for registrational and validation purposes, advance other product candidates into mid- and later-stage clinical trials, expand our product candidate portfolio, increase both our research and development and administrative personnel, and plan for commercialization of our product candidates.

At June 30, 2019, we had $280.4 million in cash and cash equivalents. We expect our current cash to fund our operations through data readouts for CARDINAL, MOXIe, and CATALYST.

Non-GAAP Financial Measures

In addition to the U.S. generally accepted accounting principles (GAAP) financial highlights, this earnings release includes cash-based operating expenses, a non-GAAP financial measure, which the Company defines as total expenses excluding stock-based compensation expense and depreciation expense. A reconciliation of this non-GAAP financial measure to its most directly comparable GAAP financial measure is presented in the table below in this earnings release.

We believe that this non-GAAP financial measure, in addition to GAAP financial measures, provides a meaningful measure of our ongoing business and operating performance by allowing investors to analyze our financial results similarly to how management analyzes our financial results by viewing period expense totals more indicative of effort directly expended to advance the business and our product candidates. Non-GAAP financial measures should be considered in addition to, not in isolation or as a substitute for, GAAP financial measures. In addition, our non-GAAP financial measure may differ from similarly named measures used by other companies.

CONFERENCE CALL INFORMATION

Date: Wednesday, August 8, 2019
Time: 8:00 a.m. ET
Audience Dial-in (toll-free): (844) 348-3946
Audience Dial-in (international): (213) 358-0892
Conference ID: 7587139
Webcast Link: View Source

On August 8, 2019 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported financial results and business highlights for the second quarter ended June 30, 2019 (Press release, Tocagen, AUG 8, 2019, View Source [SID1234538469]).

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"We are making excellent progress towards achieving our corporate objectives for the year. We are focused on providing the final analysis of our pivotal Phase 3 Toca 5 trial and preparing for a potential rolling BLA filing with FDA. The Toca 5 trial involving 403 patients with recurrent brain cancer will generate an extensive data set and holds promise to shape the treatment of these patients with a novel, multi-modality cancer-selective gene therapy in combination with surgery," said Marty Duvall, chief executive officer of Tocagen. "We also continue to execute our life cycle strategy for Toca 511 & Toca FC with the submission to FDA of a draft protocol for the planned Phase 2/3 trial of Toca 511 & Toca FC in patients with newly diagnosed glioblastoma sponsored by NRG Oncology, which remains on track to activate by the end of 2019. In addition, we recently advanced plans to evaluate Toca 511 & Toca FC in non-CNS tumors with a trial for patients with non-muscle invasive bladder cancer, which is also expected to initiate by the end of the year."

Second Quarter 2019 and Recent Highlights
2019 Corporate Milestones

Toca 5 second interim analysis completed: On May 21, Tocagen announced the pivotal Toca 5 Phase 3 clinical trial evaluating Toca 511 & Toca FC in patients with recurrent high grade glioma (HGG) would continue without modification following a planned second interim analysis conducted by an Independent Data Monitoring Committee. Tocagen expects to report the final analysis of the Toca 5 trial this year.
Progress related to Toca 511 & Toca FC BLA and commercialization: With Breakthrough Therapy Designation, Tocagen is preparing for the initiation of a rolling biologics license application, pending positive data from Toca 5. The company’s launch readiness activities span disease state awareness, branding activities, distribution, channel and pricing strategies, payor and key opinion leader engagements and beginning to build the commercial and medical affairs infrastructure and team.
NRG Oncology trial in newly diagnosed glioblastoma (ndGBM) proceeding: The Phase 2/3 trial to be conducted by NRG Oncology (NRG-BN006) evaluating Toca 511 & Toca FC in combination with standard of care (SOC) versus SOC alone in patients with ndGBM is proceeding towards initiation by year-end 2019.
Planned reporting of updated Toca 6 data: Tocagen remains on track to provide updated data including immune modulation data from this Phase 1b trial in advanced solid tumors at an upcoming scientific congress this year.
Activation of Toca 8 clinical program in bladder cancer: As part of Tocagen’s life cycle plans for Toca 511 & Toca FC and based on encouraging preclinical data and results from the Toca 6 trial, the company plans to initiate a Phase 1b trial, Toca 8, in non-muscle invasive bladder cancer (NMIBC) in the second half of this year.
Preclinical research publication: Tocagen’s most recent preclinical research paper described the use of Tocagen’s retroviral replicating platform to successfully deliver a single chain monoclonal antibody fragment against PD-L1 resulting in durable and highly selective anti-tumor activity compared to systemically administered anti-PD-L1 or anti-PD-1 monoclonal antibodies.
Second Quarter 2019 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $12.0 million for the quarter ended June 30, 2019, compared to $12.8 million for the quarter ended June 30, 2018. The R&D expenses in both periods were primarily driven by costs to support the Toca 5 trial and manufacturing of drug product. The decrease quarter over quarter is primarily due to reduced clinical trial costs in 2019 compared to 2018 as the Toca 5 trial nears completion.

General and Administrative (G&A) Expenses: G&A expenses were $4.9 million for the quarter ended June 30, 2019, compared to $2.6 million for the quarter ended June 30, 2018. The increase in G&A expenses was primarily due to higher personnel and related costs, including stock-based compensation, due to additional headcount and increased contracted services to support commercial readiness activities.

Net Loss: Net loss was $17.1 million, or $0.72 per common share (basic and diluted), for the quarter ended June 30, 2019, compared to a net loss of $16.1 million, or $0.81 per common share (basic and diluted), for the quarter ended June 30, 2018. The 2019 calculation is based on 23.7 million average common shares outstanding for the second quarter of 2019, compared to 19.9 million average common shares outstanding for the second quarter of 2018.

2019 Six-Month Results

R&D Expenses: R&D expenses were $24.4 million for the six months ended June 30, 2019 compared to $23.2 million for the six months ended June 30, 2018. The increase in R&D expenses for the six months ended June 30, 2019 primarily reflect increased personnel and related costs to support the Toca 5 clinical trial as well as manufacturing and other activities to support the potential commencement of a regulatory filing later this year following the completion of the study.

G&A Expenses: G&A expenses were $9.3 million for the six months ended June 30, 2019 compared to $5.0 million for the first six months ended June 30, 2018, with the increase primarily driven by higher personnel and related costs, including stock-based compensation, due to additional headcount and increased contracted services to support commercial readiness activities.

Net Loss: Net loss for the first six months ended June 30, 2019 was $34.2 million, or $1.46 per common share (basic and diluted), compared to a net loss of $29.0 million, or $1.45 per common share (basic and diluted), for the six months ended June 30, 2018. This calculation is based on 23.4 million average common shares outstanding for the six months ended June 30, 2019, compared to 19.9 million average shares outstanding for the same period in 2018.

Cash Position
Cash, cash equivalents and marketable securities were $68.3 million at June 30, 2019 compared to $96.1 million at December 31, 2018.

About Toca 511 & Toca FC
Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprising an investigational biologic, Toca 511 (vocimagene amiretrorepvec), and an investigational small molecule, Toca FC (flucytosine, extended-release). Toca 511 is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells resulting in anti-cancer immune activation and subsequent tumor killing.

On August 8, 2019 Oncternal Therapeutics, Inc., (Nasdaq: ONCT) a clinical-stage biotechnology company developing potential first-in-class product candidates for cancers with critical unmet medical needs, reported financial results for the second quarter, which ended June 30, 2019, and provided a business update (Press release, Oncternal Therapeutics, AUG 8, 2019, View Source [SID1234538501]).

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"Oncternal is thrilled to provide its first quarterly results as a Nasdaq-listed Company. We are making great progress developing our clinical and preclinical programs as we head into the second half of 2019," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are very encouraged by initial results from our lead clinical program’s Phase 1/2 study of cirmtuzumab in combination with ibrutinib in patients with chronic lymphocytic leukemia, and excited to be opening the randomized Phase 2 portion of the study. The early results for cirmtuzumab in combination with ibrutinib in patients with mantle cell lymphoma are also encouraging, and we expect to report data in this patient population before the end of the year. With respect to our other pipeline programs, our clinical study of TK216 in combination with vincristine in patients with Ewing sarcoma is advancing as planned, and we expect to begin enrolling patients in an expansion cohort of this study soon. Finally, we anticipate selecting a construct for IND-enabling studies of our ROR1 CAR-T program before the end of this year."

Recent Corporate Highlights

In August 2019, Oncternal announced it has opened for enrollment its randomized Phase 2 study of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib in patients with chronic lymphocytic leukemia (CLL). The decision to open the Phase 2 portion of the Company’s ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was triggered by favorable outcomes from the Part 1 dose-finding and Part 2 dose-confirming cohorts of the clinical trial, including the recently announced interim objective response rate (ORR) of 100% for the first nine CLL patients with evaluable data receiving the recommended dosing regimen who have completed 12 weeks of cirmtuzumab plus ibrutinib treatment in Part 2. The Company continues to see a well-tolerated safety profile consistent with that seen with ibrutinib treatment alone.

In June 2019, Oncternal announced that the reverse merger between GTx, Inc., GTx’s merger subsidiary and privately-held Oncternal Therapeutics, Inc., had closed and the combined company was renamed Oncternal Therapeutics, Inc. Trading on the Nasdaq stock exchange under the ticker symbol "ONCT" began on June 10, 2019. The closing of the merger was a transformative event that the Company believes will allow it to pursue its next level of corporate growth and continue to advance its oncology drug candidates in multiple cancer indications.

In June 2019, Oncternal presented interim data from its ongoing Phase 1/2 study of cirmtuzumab in combination with ibrutinib at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting – the largest oncology conference of the year. Results from the first 12 patients with CLL treated in the Part 1 dose-finding portion of the Phase 1 study showed an interim ORR of 91.7% for the combination of cirmtuzumab plus ibrutinib, including three patients with clinical or confirmed complete responses, and a well-tolerated safety profile consistent with that seen for ibrutinib treatment alone.

Oncternal also disclosed at the ASCO (Free ASCO Whitepaper) meeting that six patients with mantle cell lymphoma (MCL), had been treated in a separate cohort of the CIRLL study. One patient with MCL who had relapsed following an allogeneic stem cell transplant experienced a confirmed complete response (CR) after 3 months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be durable, and has been confirmed after 6, 9 and 11 months of cirmtuzumab plus ibrutinib treatment.

Expected Upcoming Milestones

Cirmtuzumab Program

Oncternal anticipates reporting additional data from its Phase 1/2 study of cirmtuzumab in combination with ibrutinib from patients with CLL at a scientific conference in the fourth quarter of 2019

Oncternal anticipates reporting additional data from its Phase 1/2 study of cirmtuzumab in combination with ibrutinib from patients with MCL at a scientific conference in the fourth quarter of 2019

Oncternal anticipates reporting data from its Phase 1 study of cirmtuzumab in combination with paclitaxel from patients with breast cancer at a scientific conference in the fourth quarter of 2019

TK216 Program

Oncternal anticipates completing the dose finding portion of its Phase 1 study of TK216 for patients with Ewing sarcoma and opening the expansion cohort in the third quarter of 2019

Oncternal anticipates reporting data from its Phase 1 study of TK216 from patients with Ewing sarcoma at a scientific conference in the fourth quarter of 2019

ROR1 CAR-T Program

Oncternal anticipates selecting a candidate CAR-T construct for IND-enabling studies in hematologic cancers in the second half of 2019, and opening clinical trials for hematological cancers in 2020

Oncternal anticipates selecting a candidate CAR-T construct for IND-enabling studies in solid tumors in 2020

Financial Results

On June 7, 2019, the former privately-held Oncternal Therapeutics, Inc. ("Private Oncternal"), completed a reverse merger transaction with GTx, Inc. and its merger subsidiary. Under the merger agreement, a wholly-owned subsidiary of GTx, Inc. merged with and into Private Oncternal, with Private Oncternal surviving as a wholly-owned subsidiary of the merged parent company. The surviving parent entity changed its corporate name from GTx, Inc. to Oncternal Therapeutics, Inc., and commenced trading on the Nasdaq stock exchange under the ticker symbol "ONCT."

The transaction was accounted for as a reverse asset acquisition in accordance with generally accepted accounting principles. Under this method of accounting, Private Oncternal was deemed to be the accounting acquirer for financial reporting purposes. As a result, effective as of the closing date of the merger, the reported historical operating results prior to the merger closing date will be those of Private Oncternal. Information regarding the reverse merger transaction and our financial results is also included on form 10-Q to be filed with the SEC.

Grant revenue was $0.7 million for the quarter ended June 30, 2019. Our grant revenue is derived from a California Institute for Regenerative Medicine (CIRM) grant subaward with the University of California, San Diego. The grant was awarded to advance our lead program in a Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with B-cell lymphoid malignancies CLL and MCL.

Total operating expenses for the second quarter ended June 30, 2019 were $22.3 million, which included in-process research and development expenses of $18.1 million that was recorded in connection with the closing of our merger transaction in June 2019.

Research and development expenses for the quarter totaled $2.6 million.

General and administrative expenses for the quarter totaled $1.6 million.

Including the one-time merger charge, net loss for the second quarter was $22.8 million, or a loss of $3.38 per share, basic and diluted.

As of June 30, 2019, Oncternal has $28.5 million in cash and cash equivalents. The Company believes these funds will be sufficient to fund its operations into the second quarter of 2020. As of June 30, 2019, we had 15.4 million shares of common stock outstanding.

Oncternal Management Hosting Conference Call and Live Webcast

Oncternal will host a conference call today, August 8, 2019, at 2:00 p.m. PDT (5:00 p.m. EDT) to review quarterly results and provide an update on clinical and preclinical development programs. A live webcast of the call will be available online and may be accessed from the "Investors" page of the company website at View Source A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

On August 8, 2019 Veru Inc. (NASDAQ: VERU), an oncology and urology biopharmaceutical company developing novel medicines for prostate cancer treatment and prostate cancer supportive care, reported its financial results for its fiscal 2019 third quarter ended June 30, 2019 (Press release, Veru, AUG 8, 2019, View Source [SID1234538384]).

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Business and Operational Highlights

VERU-111. VERU-111 is an oral, first-in-class, alpha and beta antitubulin being evaluated in a Phase 1b/2 clinical trial in men who have metastatic prostate cancer and whose disease is resistant to both castration and novel androgen blocking agents (abiraterone or enzalutamide). The objective of this clinical trial is to determine the dose limiting toxicity of VERU-111. At this point we have dosed 6 cohorts (18 men total) with escalating doses of VERU-111. In clinical observations, VERU-111 is well-tolerated with a favorable safety profile and no dose limiting toxicity has been observed. In some of the men whose PSA blood level was rapidly rising at enrollment, a marker of cancer progression, we have observed that VERU-111 treatment resulted in PSA stabilizations and reductions consistent with early promising signals of anticancer efficacy. Once we have reached a dose level that has dose limiting toxicity, we will be able to select the dose that will be evaluated in the Phase 2 clinical study. There are no drugs that are FDA approved to treat men who have both castration and novel androgen blocking agent resistant prostate cancer and which are also prechemotherapy (chemotherapy naïve), representing an estimated $4.5 billion annual global market.

Zuclomiphene Citrate. Zuclomiphene Citrate is a novel, proprietary, oral, nonsteroidal, estrogen receptor agonist being evaluated as a treatment for hot flashes caused by androgen deprivation therapy for men with advanced prostate cancer. Hot flashes are one of the main reasons why men want to stop androgen deprivation therapy. Approximately 100 men will be randomized in the Phase 2 clinical trial evaluating 2 doses (10mg and 50mg) of oral zuclomiphene versus placebo. Top line results expected late Summer/early Fall 2019. Based on an independent market analysis sponsored by the Company, expected U.S. sales potential for zuclomiphene citrate is estimated to exceed $600 million annually.

VERU-100. VERU-100 is a long-acting gonadotropin-releasing hormone (GnRH) antagonist for the treatment of hormone sensitive advanced prostate cancer. Currently, there are no GnRH antagonists commercially approved beyond 1 month, which would make VERU-100, if approved, the only commercially available GnRH antagonist 3-month depot. Recently, we added this proprietary internally developed peptide drug candidate to the Company’s late-stage drug development pipeline. Based on regulatory clarity obtained in the preIND meeting with FDA in May 2019, we plan to initiate a Phase 2 clinical study in early 2020 after GMP manufacturing of VERU-100. Androgen deprivation therapy for advanced prostate cancer is an established multi-billion-dollar global market.

TADFIN (Tadalafil and Finasteride Combination Capsule). TADFIN is being developed for BPH and would be the first combination of a PDE5 inhibitor and 5 alpha reductase inhibitor. The Company had a successful preNDA meeting with FDA in May 2019. After the Company has 12-month stability data on manufacturing batches, the Company will submit an NDA for TADFIN which is expected in Summer of 2020. BPH is an established multi-billion-dollar market.

"We are delivering on our strategy of providing multiple prostate cancer and prostate cancer supportive care medicines for the continuum of prostate cancer care," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru. "We are pleased with the clinical progress we are making in advancing our product development program and in adding a high value, late stage drug candidate that complements our existing portfolio."

Highlights Third-Quarter Financial Results: Fiscal 2019 vs Fiscal 2018

Net revenues increased 77% to $9.7 million from $5.5 million;

Gross profit more than doubled to $6.6 million, or 68% of net revenues, from $3.1 million, or 56% of net revenues;

FC2 US prescription net revenues increased more than tenfold to $4.4 million from $0.4 million;

Operating loss significantly narrowed to $1.8 million from $5.0 million; and

Net loss was $2.8 million, or $0.04 per share, compared with $7.9 million, or $0.15 per share.

Highlights Year-to-Date Financial Results: Fiscal 2019 vs Fiscal 2018

Net revenues rose 116% to $23.1 million from $10.7 million;

Gross profit climbed 183% to $15.8 million, or 69% of net revenues, from $5.6 million, or 52% of net revenues;

FC2 US prescription net revenues increased over tenfold to $9.4 million from $0.83 million;

FC2 public sector net revenues were $13.0 million, a 33% increase from $9.8 million;

Operating loss significantly narrowed to $5.0 million from $17.1 million (fiscal 2018 year to date included a $4.0 million loss for the settlement of Brazilian receivables); and

Net loss was $9.0 million, or $0.14 per share, compared with $16.0 million, or $0.30 per share.

"For the fiscal 2019 third quarter, we had impressive increases to net revenues and to gross profit which combined to substantially improve our bottom line," said Dr. Steiner. "Key contributors to the quarter’s performance included the continued significant ramp up of prescription sales of FC2 and the increased consumer demand for our PREBOOST/Roman Swipes. As our revenue base has grown to include increased FC2 prescription sales, our gross profit has risen substantially. The robust growth of the US FC2 prescription business remains noteworthy as it allows us to be less reliant on traditional intermittent ordering patterns typically seen in our FC2 public sector business. All of these factors combined to significantly narrow our operating loss for the quarter compared with the prior year period, which allows us to continue to invest in our multiple prostate cancer drug candidate clinical development efforts."

Financial Outlook

The Company reiterated its fiscal 2019 full year guidance of net revenues between $29 and 32 million, which represents a 95% increase over full year fiscal 2018, and gross margin of approximately 66%.

The Company does not expect to update the guidance for the full year fiscal 2019 provided above before the release announcing results for its fiscal 2019 year end. The Company notes that the statements of future performance made in this release, including the guidance for the full year fiscal 2019, are based upon current expectations and are subject to factors that could cause actual results to differ materially from those suggested here, including those factors set forth in the "Safe Harbor" Statement below.

Conference Call Event Details

Veru Inc. will host a conference call today at 8 a.m. ET to review the Company’s performance. Interested investors may access the call by dialing 800-341-1602 from the U.S. or 412-902-6706 from outside the U.S. and asking to be joined into the Veru Inc. call. In addition, investors may access a replay of the conference call the same day beginning at approximately noon Eastern Time by dialing 877-344-7529 for U.S. callers, or 412-317-0088 from outside the U.S., passcode 10133960. The replay will be available for one week, after which, the recording will be available via the Company’s website at View Source