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 Perrigo Company plc (NYSE; TASE: PRGO) reported financial results for the second quarter ended June 29, 2019 (Press release, Perrigo Company, AUG 8, 2019, View Source [SID1234538431]).

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President and CEO Murray S. Kessler commented, "The Perrigo transformation to a consumer self-care company, discussed in depth on our Investor Day on May 9, is now in the critically important execution phase. While we are in the early stages, significant progress was made during the second quarter, as evidenced by robust store-brand OTC sales in the USA, solid branded new product sales internationally, a return to strong customer service levels in the USA and the finalization of the Project Momentum $100 million cost-savings program road map. The company also closed on the Ranir (market leading private label oral self-care company) acquisition and the Perrigo Animal Health Division divestiture shortly after quarter-end."

Kessler continued, "This marks the third consecutive quarter Perrigo has delivered on our stated financial goals, which we believe is integral to the re-establishment of management credibility. Transformation activities will help Perrigo return to year-over-year growth for the balance of the year."

Refer to Tables I – V at the end of this press release for a reconciliation of non-GAAP adjustments to the current year and prior year periods and additional non-GAAP information. The Company’s reported results are included in the attached Condensed Consolidated Statements of Operations, Balance Sheets and Statements of Cash Flows.

Reported net sales for the second quarter of calendar year 2019 were approximately $1.15 billion.

Adjusted net sales increased 1% to $1.13 billion, excluding unfavorable currency movements of $24 million, the held-for-sale animal health business of $32 million and $10 million from the exited infant foods business. New product sales of $65 million and increased demand-driven sales in the Consumer Self-Care Americas and RX businesses were partially offset by lower sales in the Consumer Self-Care International business. Discontinued products of $27 million, included $10 million from the exited infant foods business.

Reported net income was $9 million, or $0.07 per diluted share, versus $36 million, or $0.26 per diluted share, in the prior year period. Excluding certain charges as outlined in Table I, second quarter 2019 adjusted net income was $117 million, or $0.86 per diluted share versus $169 million, or $1.22 per diluted share, for the same period last year.

Consumer Self-Care Americas reported net sales of $582 million were 2.5% lower than the prior year period.

Adjusted net sales of $560 million were approximately 1% higher than the prior year period, excluding the held-for-sale animal health business and exited infant foods business. Within CSCA, net sales for the core OTC business, which represent approximately 80% of CSCA net sales, increased more than 4% compared to the prior year period driven by an extended cough/cold season, a good start to the allergy season, as well as increased sales in the gastrointestinal and smoking cessation categories. CSCA also benefited from new product sales of $8 million in the quarter, led by the store brand equivalent of Imodium Multi-Symptom Relief and cherry ice mini lozenge.

Sales growth was partially offset by 1) lower net sales in the infant nutrition category primarily related to lower infant formula contract manufacturing sales as a number of branded customers made the strategic decision to exit the category, 2) discontinued products of $11 million primarily related to the exit of the infant foods business, and 3) an infant formula recall at a leading customer.

Based on the most recent 52-weeks U.S. MULO data, total OTC retail market dollars grew 1.1% compared to the same 52-weeks a year ago. For the same period, store brand retail dollars grew 1.7%, outpacing national brand growth of 0.8%, resulting in one point of share gain for store brands. Over the most recent 13 weeks, total OTC retail market dollar growth accelerated to 3.5% versus a year ago, led by total store brand retail dollar growth of 3.8%. This acceleration was driven by the extended cough/cold season and good start to the allergy season.

Second quarter reported gross profit margin was 33.8%. Adjusted gross profit margin was 34.0%, or 170 basis points lower than the prior year period, due primarily to the held-for-sale animal health business, which had a relatively higher gross margin, lower net sales from infant formula contract manufacturing and greater operating inefficiencies versus last year. Sequentially, versus first quarter, adjusted gross profit margin increased 150 basis points due primarily to favorable product mix and higher OTC sales volumes.

Reported operating margin was 18.5%. Adjusted operating margin was 20.3%, or 130 basis points lower than the prior year period, due primarily to gross margin flow-through and higher R&D investments to fuel growth. Sequentially, adjusted operating margin increased 200 basis points due to gross profit flow-through and lower SG&A expenses.

Consumer Self-Care International reported net sales were $328 million, or 8.5% lower than the prior year period. On a constant currency basis, net sales were lower by 1.8% compared to the prior year period. New product sales of $30 million were driven primarily by the successful launch of XLS Forte 5, a next generation weight loss product within the lifestyle category, and strong performance by the ACO brand in the dermatological category. Net sales were higher versus last year in the distribution business and analgesics portfolio.

These were more than offset by 1) unfavorable currency movements of $24 million, 2) lower net sales in France due primarily to a restructuring of the sales force to improve efficiency and long-term profitability, 3) lower net sales in the cough/cold and anti-parasites categories, due primarily to relatively weaker seasonal effects across Europe, and 4) discontinued products of $6 million.

According to IRI/InQvia (IMS) data, the markets in which the Company competes have produced low to mid-single-digit growth over the last 12 months, with CSCI maintaining its overall market share.

Reported and adjusted gross margins decreased 100 basis points compared to the prior year period as new product innovations were more than offset by unfavorable product mix, due primarily to the sales force restructuring in France.

Reported operating margin was (0.9)% while the adjusted operating margin was 15.3%. Operating expenses were lower compared to the prior year period due primarily to timing of advertising and promotional investments.

RX reported net sales were $239 million in the quarter, or 3.4% higher than the prior year period, due primarily to new product sales of $27 million, improved customer service levels and moderating pricing pressure in the generics industry. Discontinued products were $9 million.

Reported gross margin was 32.8%. Adjusted gross margin was 41.7%, or 830 basis points lower than the prior year period, due primarily to pricing pressure and higher sales volumes of relatively lower margin authorized generic products.

Reported operating margin was 6.1%. Adjusted operating margin was 27.4%, or 670 basis points lower than the prior year period, due primarily to adjusted gross margin flow-through partially offset by lower administrative expenses.

Outlook

The Company reaffirms its adjusted diluted EPS to be in the range of to $3.75 to $4.05 per share. Guidance includes the Ranir acquisition and Animal Health divestiture.
Potential upside to this adjusted EPS range includes contributions from a launch of the generic version of ProAir (+ $0.00 – $0.10 cents) and/or incremental cost savings from Project Momentum of up to $0.05.
The Company expects an acceleration of net sales growth in the second half of the year driven by Ranir (excluding animal health, exited infant foods and impact of currency).

On August 8, 2019 Pacira BioSciences, Inc. (Nasdaq: PCRX), a leading provider of innovative non-opioid pain management options, reported financial results for the second quarter of 2019 (Press release, Pacira Pharmaceuticals, AUG 8, 2019, View Source;991.htm [SID1234538430]).

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"During the second quarter, we made meaningful progress across key areas including commercial, clinical and corporate. We delivered yet another quarter of outstanding results highlighted by continued strong top-line growth, the addition of the novel ioverao system to our commercial offering, and the enhancement of our leadership team with the addition of Max Reinhardt as our newly appointed President," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "We continue to see robust growth for EXPAREL (bupivacaine liposome injectable suspension) with growing penetration across a wide-range of soft tissue and orthopedic procedures and the shifting of inpatient procedures to the ambulatory setting through the expanding utilization of EXPAREL-based opioid-sparing protocols."

"Looking ahead, EXPAREL continues to be well positioned for long-term market leadership as the only opioid-free, long-acting, local and regional analgesic approved for infiltration, field blocks and interscalene brachial plexus nerve block. We are also pleased with the progress we have made integrating ioverao into our commercial offering, which is expected to deliver accelerating accretion beginning in the second half of 2020." added Mr. Stack.

Second Quarter 2019 Financial Results

Total revenues were $102.6 million in the second quarter of 2019, a 22 percent increase over the $84.1 million reported for the second quarter of 2018.

Total net product sales were $101.8 million in the second quarter of 2019, a 26 percent increase over the $80.7 million reported for the second quarter of 2018.

Net product sales of EXPAREL/bupivacaine liposome injectable suspension were $99.8 million in the second quarter of 2019, a 24 percent increase over the $80.7 million reported for the second quarter of 2018.

EXPAREL net product sales were $98.9 million in the second quarter of 2019, compared to $80.4 million in the second quarter of 2018. Sales of bupivacaine liposome injectable

suspension to a third-party licensee for use in animals were $0.9 million in the second quarter of 2019, compared to $0.3 million in the second quarter of 2018.

iovera° net product sales during the second quarter of 2019 were $2.0 million. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.

Total operating expenses were $97.3 million in the second quarter of 2019, compared to $77.6 million in the second quarter of 2018.

GAAP net income was $2.7 million, or $0.06 per diluted share, in the second quarter of 2019, compared to $2.6 million, or $0.06 per diluted share, in the second quarter of 2018.

Non-GAAP net income was $17.5 million, or $0.41 per diluted share, in the second quarter of 2019, compared to $9.9 million, or $0.24 per diluted share, in the second quarter of 2018.

Pacira ended the second quarter of 2019 with cash, cash equivalents, short-term and long-term investments ("cash") of $317.6 million. Cash provided by operations was $22.8 million in the second quarter of 2019, compared to $13.7 million in the second quarter of 2018.

See "Non-GAAP Financial Information" and "Reconciliation of GAAP to Non-GAAP 2019 Financial Guidance" below.

Recent Business Highlights

Validation of EXPAREL Marketing Authorization Application from European Medicines Agency. In June, Pacira announced that the company’s Marketing Authorization Application (MAA) for EXPAREL for postsurgical analgesia was validated by the European Medicines Agency (EMA). With this validation, the Pacira application is complete and the EMA Committee for Medicinal Products for Human Use will now begin the review procedure with an opinion expected in the second half of 2020.

Appointment of former Johnson & Johnson executive, Max Reinhardt, as President. In June, Pacira announced the appointment of Max Reinhardt as the company’s president. Mr. Reinhardt will report to Dave Stack, chairman and chief executive officer of Pacira, and be responsible for overseeing all commercial and medical affairs functions at Pacira. Mr. Stack will maintain leadership of the overall Pacira corporate strategy.

New analysis shows use of EXPAREL associated with improved clinical and economic outcomes following hip replacement surgery. In June, Pacira announced new data on the use of EXPAREL following total hip arthroplasty (THA). The findings show that patients receiving EXPAREL had a significant reduction in opioid use, hospital length of stay and total hospitalization costs compared to THA patients who did not receive the product. The results were published in The Journal of Medical Economics.

Publication of pivotal study of EXPAREL as a single-dose interscalene brachial plexus nerve block in patients undergoing shoulder surgery. In June, Pacira announced the publication of its multinational Phase 3 study supporting the efficacy and safety of EXPAREL as a single-injection interscalene plexus nerve block in patients undergoing total shoulder arthroplasty or rotator cuff repair. The data, which provided the basis for FDA approval for this indication, were published in Pain Medicine.

Phase 4 study demonstrates superiority of EXPAREL plus bupivacaine versus bupivacaine alone in Cesarean Section procedures. In May 2019, Pacira announced full results from its Phase 4 study of EXPAREL administered via TAP field block in patients undergoing C-Section. EXPAREL achieved its primary endpoint with a statistically significant reduction in total postsurgical opioid consumption through 72 hours. EXPAREL also achieved statistical significance for reduction in pain intensity scores through 72 hours.

2019 Financial Guidance

Pacira updated its guidance for selling, general and administrative (SG&A) expense and reiterated its remaining guidance. For the full year 2019, the company currently expects:

EXPAREL net product sales in the range of $400 million to $410 million.

iovera° net product sales in the range of $8 million to $10 million.

Non-GAAP gross margins in the range of 75% to 76%.

Non-GAAP research and development (R&D) expense in the range of $60 million to $70 million.

Non-GAAP SG&A expense in the range of $180 million to $190 million versus the company’s previously guided range of $165 million to $175 million. Non-GAAP SG&A guidance was increased primarily due to the inclusion of commercial infrastructure costs for iovera°.

Stock-based compensation in the range of $30 million to $35 million.

On August 8, 2019 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation, reported an update on the cytisinicline clinical development program and second quarter 2019 financial results (Press release, OncoGenex Pharmaceuticals, AUG 8, 2019, View Source [SID1234538429]).

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Q2 2019 Highlights

Reported positive results from the Phase 2b ORCA-1 dose-selection trial evaluating cytisinicline in 254 smokers. Cytisinicline demonstrated a statistically significant improvement in quit rates for a simplified 3.0 mg, three times daily dose. Cytisinicline was well-tolerated with no serious adverse events reported.

Announced ORCA-1 data has been accepted for an oral presentation at the Society for Research on Nicotine & Tobacco Europe (SRNT-E) 19th Annual Conference to be held in Oslo, Sept. 12-14, 2019

Patent granted in the U.S. for new cytisinicline succinate salt formulation designed to enhance product stability and long-term potency

Received $4.2 million in proceeds from exercise of warrants

"It has been an exciting quarter with the release of the positive ORCA-1 Phase 2b trial results that demonstrated impressive smoking cessation efficacy, safety, and cytisinicline treatment adherence," commented Rick Stewart, Chairman and Chief Executive Officer of Achieve. "The data exceeded our expectations and provide a clear path forward for our future Phase 3 program."

ORCA-1 Positive Study Results

The Phase 2b ORCA-1 trial of cytisinicline in U.S. smokers demonstrated a statistically significant improvement in quit rates for the 3.0 mg, three times daily dosing (TID) schedule. In the 3.0 mg TID arm, a 54% abstinence rate at week 4, compared to 16% for placebo (p < 0.0001) was observed. Continuous abstinence at weeks 5 through 8 was 30% for cytisinicline compared to 8% for placebo (p= 0.005). Adherence to study treatment was 98% in the 3.0 mg TID arm and cytisinicline was well-tolerated with no serious adverse events reported.

Cytisinicline Data Accepted for Presentation

Data from the Phase 2b ORCA-1 trial has been accepted for oral presentation at the SRNT-E Annual Conference, to be held in Oslo, September 12-14, 2019. The abstract "A Multicenter, Double-blind, Randomized, Placebo-controlled Phase 2b Trial of Cytisinicline in Adult Smokers" and two, oral presentations will include updated cytisinicline data from the recently completed trial.

Patent Granted in the U.S. for Novel Formulation of Cytisinicline

The U.S. Patent and Trademark Office has granted Achieve a patent on succinate salt of cytisinicline and use thereof. The patent covers a novel salt form of cytisinicline, namely cytisinicline succinate, and pharmaceutical compositions and dosage forms. Achieve has been pursuing cytisinicline

succinate salt as a novel new drug product formulation that may further enhance cytisinicline product stability and long-term potency.

Received $4.2 Million from Exercise of Warrants

Achieve announced that it had entered into an agreement with a single investor to exercise outstanding warrants that provided an aggregate of $4.2 million in total proceeds. In exchange for the warrant exercise, Achieve issued to the investor a new warrant, exercisable for six years, to purchase up to 1,200,000 shares of Common Stock at an exercise price of $4.50 per share. The Company filed an S-1 registration statement on July 24, 2019 covering the resale of the shares issuable upon exercise of the new warrant.

Financial Results

As of June 30, 2019, the company’s cash, cash equivalents, and restricted cash was $10.5 million. Total operating expenses for the three and six months ended June 30, 2019 were $3.7 million and $9.6 million, respectively. Total net loss for the three and six months ended June 30, 2019 was $3.6 million and $9.5 million, respectively.

As of August 8, 2019 Achieve had 8,097,763 shares outstanding.

Conference Call Details

Achieve will host a conference call at 4:30 p.m. Eastern time today, Thursday, August 8, 2019. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 6587726. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

On August 8, 2019 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2019 (Press release, Omeros, AUG 8, 2019, View Source [SID1234538428]).

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Second Quarter 2019 Financial Highlights

OMIDRIA revenues for 2Q 2019 were a record high at $26.8 million. This compares to $21.8 million in 1Q 2019. The increase of $5.0 million, or 23 percent, over the prior quarter reflects both an expanded number of purchasing accounts and deeper penetration across Ambulatory Surgery Centers (ASCs), hospitals and the Veterans Administration and other government systems.

Net loss in 2Q 2019 was $14.5 million, or $0.29 per share, which included non-cash expenses of $6.3 million, or $0.13 per share. This compares to a net loss $24.3 million, or $0.50 per share, in 1Q 2019.

At June 30, 2019, Omeros had cash, cash equivalents and short-term investments available for operations of $31.8 million and an accounts receivable balance of $28.5 million.

In August 2019, the company entered into a $50-million revolving line of credit facility with Silicon Valley Bank. Borrowing availability is based on eligible accounts receivable, subject to applicable reserves.

Recent Business Highlights

Reached agreement with FDA on the primary endpoint criteria for the pivotal trial to support the biologics license application (BLA) for narsoplimab to treat hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

As part of a successful pre-BLA meeting directed to chemistry, manufacturing and controls (CMC), the company discussed with FDA the CMC requirements for the narsoplimab BLA for HSCT-TMA and is confident in its ability to meet them.

Executed a long-term commercial manufacturing agreement with Lonza in preparation for market launch of narsoplimab.

Received a product-specific permanent J-code for OMIDRIA. The J-code will become effective October 1, 2019.

"OMIDRIA revenues continue to set new quarterly records as our customer base continues to broaden throughout all channels and our per-account capture of cataract procedures grows," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "Indications are that sales will continue to grow, helped in part by our new permanent J-code, broadening Med Advantage and commercial payer reimbursement, and additional strong clinical data that we believe places OMIDRIA squarely within CMS’ criteria for separate payment. As OMIDRIA ramps toward fully funding our pipeline, our assets increasingly declare their value – narsoplimab is moving toward anticipated approval and launch, OMS527 for addiction has successfully completed the Phase 1 clinical trial, OMS906 targeting the alternative complement pathway and our follow-on MASP-2 inhibitors are slated to enter the clinic beginning next year, and GPR174 inhibition appears to play a key role in cancer immunotherapy. Each of these unique and cutting-edge programs is focused on significantly improving – or saving – patients’ lives."

Other Business Updates and Developments

Recent developments regarding OMIDRIA include the following:

o In July, CMS awarded a product-specific permanent J-code for OMIDRIA, which will become effective October 1, 2019. J-codes standardize the submission and payment of insurance claims across Medicare, Medicare Advantage, Medicaid and commercial insurance plans. The J-code should allow many commercial and Medicare Advantage insurers that currently do not reimburse under the existing C-code for OMIDRIA to reimburse under the permanent J-code. Omeros’ commercial team has already initiated efforts to ensure that customers and payers are prepared to implement the new J-code.

o Two independent studies in a total of approximately 2,800 cataract surgery patients demonstrate that OMIDRIA, compared to steroids, significantly reduces the incidence of cystoid macular edema (CME) by three to twelve-fold and breakthrough iritis as well as pain/photophobia, each by approximately three-fold. The studies are expected to be submitted later this month for peer-reviewed journal publication.

o An independent investigator study shows that OMIDRIA, with statistical significance, reduces cataract surgery patients’ requirement for perioperative opioids by nearly 80 percent while reducing visual analog scale (VAS) pain scores by more than 50 percent. The study results have been selected for presentation at the meetings of the American Academy of Ophthalmology in October and a manuscript is expected to be submitted later this month for publication in a peer-reviewed journal

Recent developments regarding narsoplimab, Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in Phase 3 clinical programs for the treatment of HSCT-TMA, Immunoglobulin A (IgA) nephropathy, and atypical hemolytic uremic syndrome (aHUS), include the following:

oAll criteria for the response-based primary endpoint in Omeros’ pivotal trial to support the BLA for narsoplimab to treat HSCT-TMA have now been finalized following agreement with FDA. The endpoint criteria are comprised of (1) laboratory markers reflecting disease progression and (2) organ function as a measure of clinical response. No additional patients are necessary for submission of the BLA. The majority of the data that comprise the BLA’s clinical module are in-house and collection of the remaining data is underway. As part of a successful pre-BLA CMC meeting the company reached agreement with FDA on the narsoplimab BLA for HSCT-TMA with respect to chemistry, manufacturing and controls. Omeros has submitted to FDA its proposed schedule for a rolling BLA and is targeting submission of the BLA’s first module by mid-next quarter.

oOmeros and Lonza, a premier global drug manufacturer, executed a long-term commercial manufacturing agreement. Omeros and Lonza have partnered since 2015 in connection with the development of narsoplimab. The new multi-year agreement secures commercial supply of narsoplimab for use following anticipated regulatory approvals.

oOmeros began meetings with the rapporteurs assigned by the European Medicines Agency to work with the company throughout the preparation and submission of the Marketing Authorization Application for narsoplimab in HSCT-TMA. Omeros plans to use the same clinical, manufacturing, and nonclinical data for approval in Europe.

oIn response to increasing physician demand, Omeros has expanded its compassionate use program for narsoplimab in HSCT-TMA. This expanded program provides patients and physicians with increased access to narsoplimab and will generate additional data useful in the drug’s planned market launch.

oOmeros’ Phase 3 trial evaluating narsoplimab for IgA nephropathy, referred to as ARTEMIS-IGAN, continues enrollment at sites in the U.S., Europe and Asia. A manuscript detailing the clinical data from the Phase 2 IgA nephropathy program has been prepared by Omeros’ Academic Leadership Committee and is being finalized for submission to a peer-reviewed journal.

o A case report will soon be submitted for publication detailing the impressive response to narsoplimab treatment by a patient who was quickly deteriorating due to IgA vasculitis-associated nephritis and rapidly progressive glomerulonephritis.

Updates regarding Omeros’ other development programs and platforms include the following:

oOmeros’ antibody against MASP-3, OMS906, continues to progress toward planned clinical entry in the first half of next year. Targeting subcutaneous dosing of once-monthly or longer, the initial focus in this program is paroxysmal nocturnal hemoglobinuria.

o As part of lifecycle planning for its MASP-2 program, Omeros is advancing development of a long-acting second-generation MASP-2 antibody targeting monthly subcutaneous delivery as well as an orally available small-molecule inhibitor of MASP-2. Both programs are slated for clinical entry by mid-2021.

o Dosing is complete in the Phase 1 trial for OMS527, the lead compound in Omeros’ phosphodiesterase 7 (PDE7) program, which targets treatment of addiction and compulsive disorders. The compound was generally well tolerated with no significant adverse events being reported. The pharmacokinetic data support once-daily dosing, with or without food. Data analysis is being finalized, and detailed study results will soon be released. Omeros plans to conduct a Phase 2a study targeting nicotine addiction.

Financial Results

2Q 2019 revenues were a record-high $26.8 million, all relating to sales of OMIDRIA. On a sequential quarter-over-quarter basis, OMIDRIA revenues increased by $5.0 million, or 23 percent, from the $21.8 million achieved in 1Q 2019. The increase is due to a growing number of ASCs and hospitals using OMIDRIA for cataract surgery as well as deeper penetration within individual accounts.

Inventory on hand at wholesalers at June 30, 2019 remained consistent with historical norms. Gross-to-net deductions increased slightly from 27 percent in 1Q 2019 to 28 percent in 2Q 2019, primarily due to increased chargebacks and rebates.

Total 2Q 2019 costs and expenses were $36.1 million compared to $41.0 million for 1Q 2019. The decrease reflected reduced narsoplimab manufacturing costs, which vary from quarter to quarter depending on the timing of manufacturing development activities, partially offset by an increase in selling, general and administrative expense.

For 2Q 2019, Omeros reported a net loss of $14.5 million, or $0.29 per share, which included non-cash expenses of $6.3 million ($0.13 per share). In comparison, for 1Q 2019 Omeros reported a net loss of $24.3 million, or $0.50 per share, which included non-cash expenses of $6.0 million ($0.12 per share).

As of June 30, 2019, the company had $31.8 million of cash, cash equivalents and short-term investments available for operations. In August 2019, the company entered into a loan and security agreement under which Omeros may borrow, on a revolving basis, up to $50 million, subject to applicable reserves and an available borrowing base of eligible accounts receivable.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 1697797. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 1697797.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at www.omeros.com and select "Events" under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.