On November 5, 2019 Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company focused on developing novel therapies for immune mediated diseases, reported financial results for the third quarter ended September 30, 2019 (Press release, Principia Biopharma, NOV 5, 2019, View Source [SID1234550337]).

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"With our focus on execution, we made significant progress on our clinical programs during the third quarter. We also recently reported preliminary positive data from our ongoing Phase 2 pemphigus and Phase 1/2 immune thrombocytopenia (ITP) clinical trials with PRN1008. Following these announcements, we were able to execute a successful follow-on offering of common stock with gross proceeds of $241.5 million," said Martin Babler, president and chief executive officer of Principia Biopharma. "We are also pleased industry veteran Shawn Tomasello joined our Board of Directors as she brings an extensive track record in commercializing novel pharmaceutical products."

Third Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $161.2 million as of September 30, 2019, compared to $180.6 million as of December 31, 2018. In addition, in October 2019, we completed a follow-on public offering which resulted in net proceeds of $226.5 million after deducting underwriting discounts and commissions and estimated offering expenses.

Revenues: We did not recognize any collaboration revenue for the three months ended September 30, 2019, compared to $18.6 million for the same period in 2018. The $18.6 million recognized for the three months ended September 30, 2018 consisted of a portion of upfront fees and milestone payments from our Sanofi and AbbVie collaborations.

R&D Expenses: Total research and development expenses were $18.4 million for the three months ended September 30, 2019, including stock-based compensation expense of $1.8 million, compared to $9.2 million for the same period in 2018, including stock-based compensation expense of $0.3 million. The increase in total research and development expenses was mainly driven by an increase in personnel-related expenses as we build out our R&D team, and an increase in PRN1008 program costs, due to the initiation of a global Phase 3 trial in patients with pemphigus in November 2018 and certain manufacturing campaigns to supply drug products for our PRN1008 clinical trials.

G&A Expenses: General and administrative expenses were $5.0 million for the three months ended September 30, 2019, including stock-based compensation expense of $1.5 million, compared to $2.9 million for the same period in 2018, including stock-based compensation expense of $0.4 million. The increase in total general and administrative expenses was primarily driven by increased personnel-related expenses and headcount costs related to operating as a public company.

Net Income (Loss): For the three months ended September 30, 2019, net loss was $22.3 million compared to a net income of $6.7 million for the same period in 2018, as we did not recognize any collaboration revenue for the three months ended September 30, 2019.

On November 5, 2019 Poseida Therapeutics Inc., a clinical-stage biopharmaceutical company leveraging proprietary non-viral gene engineering technologies to create life-saving therapeutics, reported it will present preclinical research findings during the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting on its lead allogeneic product candidate, P-BMCA-ALLO1, in multiple myeloma (Press release, Poseida Therapeutics, NOV 5, 2019, View Source [SID1234550336]).

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At SITC (Free SITC Whitepaper) 2019, preclinical results will highlight the potential of Poseida’s gene engineering technologies in addressing current challenges with earlier generation autologous CAR-T therapies. Poseida leverages its proprietary piggyBac DNA Modification System in combination with Cas-CLOVER gene editing technology to create P-BCMA-ALLO1, an off-the-shelf allogeneic CAR-T cell product candidate. These technologies enable the development of allogeneic CAR-T therapies with a variety of benefits to patients and the medical community including greater safety and duration of response, as well as manufacturing and patient cost savings.

Poseida will present the following research at SITC (Free SITC Whitepaper) 2019:

P-BCMA-ALLO1 – a Non-viral Allogeneic Anti-BCMA CAR-T Therapy with Potent Antitumor Function for the Treatment of Multiple Myeloma (Oral Presentation [O7, Annual Meeting Session 100]; November 7, 4:15 PM ET; Potomac Ballroom CD) which shows potent effector function in preclinical research indicating the need for rapid advancement into the clinic for the treatment of multiple myeloma.
"Broad adoption of earlier generation CAR-T therapies have been curtailed by serious safety concerns, limited duration of response and difficulty supporting access within the current healthcare system," said Eric Ostertag, M.D., Ph.D., chief executive officer of Poseida. "We are actively problem-solving to address these challenges and our new findings indicate that we are making progress with our allogeneic approach powered by our piggyBac DNA Modification System and Cas-CLOVER gene editing technology."

About P-BCMA-ALLO1

P-BCMA-ALLO1 is an allogeneic CAR-T therapy being developed by Poseida for multiple myeloma. It is designed to have the benefits of scale and administration efficiency that come from an allogeneic product. Poseida expects to file an IND for P-BCMA-ALLO1 in 2020. Approximately 32,110 people were diagnosed with multiple myeloma and 12,960 died from the condition in the United States in 2019.

On November 5, 2019 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended September 30, 2019, and provided a corporate update (Press release, Portola Pharmaceuticals, NOV 5, 2019, View Source [SID1234550335]).

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"We delivered another quarter of strong Andexxa revenue in the U.S. and expanded our growth potential with our first sales of Ondexxya in Europe. The use of Factor Xa inhibitors in both markets continues to grow, driving the underlying market opportunity for Andexxa/Ondexxya and long-term value of Portola," said Scott Garland, Portola’s president and chief executive officer. "Looking forward, we will continue to focus on exceptional launch execution, leveraging external support from health authorities and favorable society guidelines, and building the clinical evidence and awareness of Andexxa. In addition, we remain on track to initiate a registration trial for cerdulatinib in early 2020 and will present updated data at the ASH (Free ASH Whitepaper) meeting in December."
Product Sales:

Total global revenues for the third quarter of 2019 were $36.8 million compared with $14.2 million for the third quarter of 2018. This includes $35.7 million in net product revenues from sales of Andexxa/Ondexxya [coagulation factor Xa (recombinant), inactivated-zhzo], $17,000 in revenues from Bevyxxa (betrixaban) sales and $1.1 million in collaboration and license revenues.

Net loss attributable to Portola, according to generally accepted accounting principles in the U.S. (GAAP) was $49.6 million, or $0.68 net loss per share for the third quarter of 2019, compared with a net loss of $71.3 million, or $1.08 net loss per share, for the same period in 2018.
Operating Expenses:
For the third quarter of 2019, compared to the same period in 2018:

Research and development (R&D) expenses decreased by $14.6 million, or 36.3%, primarily due to the manufacturing costs for Andexxa Gen 2 being capitalized and no longer flowing through R&D.

Selling, general and administrative (SG&A) expenses increased by $13.3 million, or 34.2%, due to commercial costs to support the Andexxa launch including the expansion of the field sales teams and launch preparations in Europe.

Cost of Sales (COS) were $2.7 million for the third quarter of 2019, compared to $4.3 million for the same period in 2018. This decrease was primarily due to transition from our Gen 1 to our Gen 2 Andexxa product.

These amounts on a GAAP and non-GAAP basis are reflected in the table below. A table reflecting the reconciliation of GAAP to non-GAAP amounts is included at the end of this release.

Cash, Cash Equivalents and Investments:

Cash, cash equivalents and investments at September 30, 2019, totaled $476.8 million, compared with $317.0 million as of December 31, 2018. During the third quarter, the Company added net proceeds of $245 million in connection with the follow on offering of the Company’s common stock.
Recent Achievements and Events

Ondexxya launched with $2.7 million of European net sales in the third quarter of 2019.

Andexxa added to the Veteran’s Health Administration national formulary, making access to 170 VA hospitals across the U.S. possible.

Andexxa recommended as first line therapy by The American Academy of Family Physicians (AAFP).

Centers for Medicare and Medicaid Services (CMS) New Technology Add-on Payment (NTAP) reimbursement for Andexxa increased from 50% to 65% effective on October 1, 2019.

Presented sub-analysis of ANNEXA-4 study at the American College of Gastroenterology annual meeting, highlighting the efficacy and safety of Andexxa in Factor Xa patients with acute gastrointestinal bleeding, which demonstrated excellent or good hemostasis achieved in 82% of evaluable patients.

Presented in vitro data at International Society of Thrombosis and Hemostasis annual meeting, demonstrating that four-factor prothrombin complex concentrate (4F-PCC) does not appear to have an effect on the inhibition of thrombin generation by apixaban or rivaroxaban unless the Factor Xa inhibitor concentration was less than 75 ng/mL. In contrast, data from the same thrombin generation assay demonstrated that Andexxa fully corrected the inhibition of thrombin generation by apixaban and rivaroxaban across a broad range of inhibitor concentrations.

Net proceeds of $245 million raised in a public offering of the Company’s common stock.
Planned Upcoming Milestones

Continue launch of Ondexxya in a select group of high-potential European countries with significant usage of Factor Xa inhibitors and supportive access and reimbursement.

Plan to initiate an urgent surgery study for Andexxa by year end or in early 2020.

Plan to launch a cerdulatinib registration study in peripheral T-cell lymphoma (PTCL) in early 2020.

Multiple abstracts related to Andexxa and cerdulatinib accepted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December.
Analyst and Investor Meeting
Portola will host an analyst and investor meeting focused on Andexxa on Thursday, November 14, 2019, from 8:00 to 10:30 a.m. ET in New York. Seating is limited to those who RSVP. Please contact [email protected] for an invitation. The event will also be webcast live and can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for one year following the date of the event.

Conference Call Details
Portola will host a conference call today, Tuesday, November 5, 2019, at 4:30 p.m. ET, during which time management will discuss the third quarter 2019 financial results, updates on the U.S. and European launch of Andexxa/Ondexxya, and its operations. The live call can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 2980632. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.
Use of Non-GAAP Financial Measures
This press release and the reconciliation table included herein include non-GAAP R&D expenses. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When viewed in conjunction with GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those that the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation of GAAP to non-GAAP financial measures is provided in the accompanying table entitled "Reconciliation of GAAP to Non-GAAP Financial Informati

On November 5, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported that the Company will present phase 1 dose-escalation monotherapy data for PRS-343, a 4-1BB/HER2 bispecific for the treatment of HER2-positive solid tumors, in a late-breaking oral presentation session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in National Harbor, Maryland (Press release, Pieris Pharmaceuticals, NOV 5, 2019, View Source [SID1234550334]).

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"We are pleased to have had our abstract accepted as one of the few late-breaking podium presentations at the conference, and we look forward to sharing an updated dataset for the program on November 9th, including safety, PK, biomarker, and clinical response data for patients enrolled in the highest dose cohorts," said Stephen S. Yoder, President and Chief Executive Officer of Pieris.

Presentation Details

Title: A Phase 1 Dose Escalation Study of PRS-343, a HER2/4-1BB Bispecific Molecule, in Patients with HER2-positive Malignancies
Session: 304
Date/Time: Saturday, November 9, 2019, 11:50 AM EST
Location: Potomac Ballroom, Gaylord National Hotel & Convention Center in National Harbor, Maryland

The presentation will be made available on the publications section of the Company’s website at www.pieris.com.

On November 5, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended September 30, 2019 (Press release, Opko Health, NOV 5, 2019, View Source [SID1234550333]).

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Business Highlights

Somatrogon meets primary and key secondary endpoints in global Phase 3 trial: On October 21, OPKO and Pfizer announced that the global Phase 3 trial evaluating somatrogon dosed once weekly in pre-pubertal children with growth hormone deficiency met its primary endpoint of non-inferiority to GENOTROPIN (somatropin) injected daily, as measured by annual height velocity at 12 months. Key secondary endpoints including change in height standard deviation scores at six and 12 months, and change in height velocity at six months, were also higher in the somatrogon group.

RAYALDEE total prescriptions reported by IQVIA increased 83% in 3Q 2019 compared with 3Q 2018: Total prescriptions for the three months ended September 30, 2019 increased to approximately 14,600, compared with approximately 8,000 during the comparable period of 2018.

BioReference Laboratories continued to establish new alliances: During the third quarter BioReference Laboratories formed a strategic collaboration with SOMOS, New York City’s largest multicultural physician-led network, and was named the preferred provider for the IPA Association of America in laboratory services and, also, to assist with data analytics for member patients. These wins build upon successes earlier in the year, including selection as part of the preferred lab network with UnitedHealthcare and new in-network status with Humana.

JAMA Oncology published data from a GeneDx study addressing cancer risk estimates for gastric cancers in patients with gene variants of CDH1: Results indicated that the lifetime risk of gastric cancer for individuals with pathogenic variants in the CDH1 gene is significantly lower than previously described; the lifetime risk of gastric cancer associated with pathogenic CDH1 variants was 42% in men and 33% in women, compared to published estimates of 40-70% in men and 56-83% in women. These results allow for a more personalized risk assessment and better informed decision making for patients at risk for hereditary gastric cancer.

Pharmaceutical pipeline continues to advance:

The open-label Phase 2 trial for RAYALDEE in hemodialysis patients is progressing and initial data are expected in 1Q 2020.

The somatrogon registration study in Japanese pediatric GHD patients to assess pharmacokinetics and compare efficacy of weekly somatrogon to daily GENOTROPIN is on track for data readout in 2020.

Financial Highlights

Consolidated revenues for the third quarter of 2019 were $228.8 million, compared with $249.8 million for the comparable period of 2018. Revenue from services in the third quarter was $181.1 million, revenue from products was $26.2 million, including RAYALDEE net revenue of $7.4 million, and revenue from licensing and intellectual property was $21.5 million.

Operating expenses for the third quarter of 2019 were $267.8 million. This included R&D expenses of $30.0 million, principally for the completion of the pediatric Phase 3 study for our long-acting human growth hormone product and other ongoing clinical trials.

Accounting rules require marking strategic investments to market at the end of each quarter, which had a negative impact on the net loss for the third quarter of 2019 of $13.6 million, or $0.02 per share. The net loss for the third quarter of 2019, after giving effect to the decrease in market value of our strategic investments, was $62.0 million, or $0.11 per share, compared with a net loss of $27.7 million, or $0.05 per share, for the comparable period of 2018. The 2018 period’s net loss benefited from an $11.6 million income tax benefit primarily resulting from a change in state income tax rates during that period.

Cash, cash equivalents and marketable securities were $64.7 million as of September 30, 2019. Subsequent to the close of the third quarter, the Company raised gross proceeds of $75.0 million from an underwritten public offering of common stock.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, Tuesday, November 5, 2019. The conference call dial-in and webcast information is as follows:
DOMESTIC DIAL-IN:

866-634-2258
INTERNATIONAL DIAL-IN:

330-863-3454
PASSCODE:

7270239
WEBCAST:

OPKO 3Q19 Results Conference Call
For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 7270239. The replay can be accessed for a period of time on OPKO’s website at OPKO 3Q19 Results Conference Call.