On November 7, 2019 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported its interim financial results in line with guidance for the first nine months of 2019 and business progress for the third quarter of 2019 (Press release, Bavarian Nordic, NOV 7, 2019, View Source [SID1234550647]).

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Paul Chaplin, President & Chief Executive Officer of Bavarian Nordic said: "We set out the year with a clear ambition and strategy to expand our commercial footprint, and with the recently announced acquisition of two commercial vaccines from GlaxoSmithKline (GSK), we turn to the next chapter for Bavarian Nordic to become an independent vaccine company with sustainable profitability. The company is fully focused on the integration of manufacturing of the two new vaccines and establishing the commercial operations to make this a great success. We will move ahead with our planned Phase 3 trial for RSV in 2021 that allows sufficient time to establish an improved commercial scale production and formulation to meet the demands of this blockbuster indication. The recent approval of JYNNEOS was also a tremendous milestone for the Company, offering exciting new business opportunities beyond our long-term partnership with the U.S. government to supply the national stockpile and offering clear synergies with the two acquired commercial vaccines."

TRANSFORMATIVE ACQUISITION OF TWO COMMERCIAL VACCINES FROM GLAXOSMITHKLINE
Announced on October 21, the acquisition of Rabipur/RabAvert and Encepur from GlaxoSmithKline (GSK) is a transformative transaction that will establish a leading infectious disease franchise and make Bavarian Nordic an independent and profitable vaccine company. Specifically, the transition:

Accelerates our vision by 3 years to become a leading and profitable vaccine company
Exploits significant manufacturing synergies between highly complementary technologies and builds on our expertise
Ensures strong cash flow generation, allowing us to continue progressing our promising innovative pipeline
Full details on the acquisition were published in company announcement no. 19/2019.

Closing of the acquisition is expected by December 31, 2019, conditional on antitrust approval as well as on approval of the rights issue by the Company’s shareholders. To resolve on the rights issue, an extraordinary general meeting has been convened on November 27, 2019. Details are available at www.bavarian-nordic.com/egm.

OPERATIONAL HIGHLIGHTS

Delivering our strategy
We set out for the year with a 5-year vision and four strategic imperatives that collectively build on our heritage in vaccine development and manufacturing, aiming to secure and reinforce a sustainable foundation, while also expanding the commercial opportunities. With the acquisition of two commercial vaccines from GSK, the recent approval of JYNNEOS and the advancement of several clinical programs during the year, we are on track to accomplish these targets:

MAINTAIN global leadership of our smallpox vaccine business
EXPAND and rapidly ADVANCE the pipeline of infectious disease programs
ESTABLISH a broad and deep cancer immunotherapy portfolio
EXPAND the commercial footprint and capabilities
Smallpox

In September, the U.S. Food and Drug Administration (FDA) approved JYNNEOS (MVA-BN) for protection against smallpox and monkeypox. The approval was the result of our successful, fifteen-year partnership with the U.S. government to develop a non-replicating smallpox vaccine, suitable for the entire population, presenting significant opportunities for our future business with the U.S. government beyond emergency stockpiling.
As expected, we were awarded a Priority Review Voucher along with the approval.
The additional indication for monkeypox provides new commercial opportunities to be exploited in the coming years as we expand the commercial infrastructure to accommodate sales and distribution of the vaccines acquired from GSK.
A Phase 3 lot-consistency trial, fully funded by BARDA, of the freeze-dried formulation of MVA-BN is ongoing. A prior Phase 2 study showed bioequivalence between the freeze-dried and liquid-frozen formulations of MVA-BN, and the lot-consistency trial was agreed with the FDA as the only Phase 3 study required to support licensure of the freeze-dried formulation, which is anticipated in 2022.
We are on track with the production of bulk vaccine for the current order for freeze-dried MVA-BN from the U.S. Government with the majority of the batches being invoiced in the fourth quarter of 2019 adding up to a total of USD 50 million included in our guided 2019 revenues.
Infectious diseases

The design of the Phase 3 efficacy trial, for our highly promising RSV vaccine candidate for the elderly (≥60 years old), has been finalized with the FDA. The randomized, placebo-controlled trial has an adaptive design and will enroll a total of 12,000 to 14,000 subjects over two seasons. The total number of subjects will depend on the independent analysis performed from the first 6,000 subjects enrolled for the first season. The estimated costs to determine futility after the season one will be USD 40 million and if positive, the second season will cost an additional USD 50-70 million, although after passing the first season threshold there would be approximately a 75% chance of successfully reaching the efficacy endpoint of the trial. The trial is planned to be initiated in 2021 prior to the RSV season, with the initial read out in 2022 and a potential approval in 2024.
In October, we initiated a Phase 1 clinical trial of MVA-BN WEV, a trivalent prophylactic vaccine against three strains of the equine encephalitis virus: Western, Eastern (also referred to as triple E) and Venezuelan. The current program is funded by the U.S. Department of Defense. Equine encephalitis virus is an alpha virus that is spread by mosquitos from infected birds to horses and people that can result in the rare condition of encephalitis in about 5% of the people that become infected. Since October, 31 cases, including 9 deaths have been reported by the US CDC in what is the largest ever recorded outbreak of triple E. Successful Phase 1 results could lead to additional funding of clinical development towards licensure of this important emerging infectious disease.
Our Janssen partnership provides an opportunity to explore and commercialize our MVA-BN platform in three infectious disease indications; HPV, HIV and HBV. A Phase 1/2a trial of the therapeutic HPV vaccine was initiated earlier this year and the first clinical trial of the therapeutic HIV vaccine is anticipated to begin in 2020. Collectively, these programs, along with our Ebola collaboration, represent USD 1 billion in potential future milestone payments, in addition to royalties on future sales.
Ebola continues to rage in the Democratic Republic of Congo, and Janssen recently announced the donation of up to 500,000 doses of the Ad26.ZEBOV/MVA-BN Filo vaccine regimen, which will be introduced to help counter the outbreak. Neighboring countries like Rwanda and Uganda have also expressed their intent to use the vaccine as Ebola cases have been reported around the border. In Uganda, a large trial of the vaccine was initiated in healthcare workers in August. The vaccine regimen has to-date been evaluated in more than 6,500 individuals in Phase 1, 2 and 3 clinical studies, and Janssen is planning to submit a Marketing Authorization Application to the European Medicines Agency this year.
Cancer immunotherapy

In October, we updated on the development of CV301, and the study in bladder cancer, which did not meet the efficacy threshold to expanded enrollment for the second stage. While we continue to follow and support two ongoing, investigator-sponsored Phase 2 trials evaluating CV301 in combination with checkpoint inhibitors in colorectal and pancreatic cancer, the Company’s focus moves to BN-Brachyury and the intravenous and intra-tumoral applications.
BN-Brachyury is currently being evaluated in a Phase 2 trial in patients with advanced chordoma. This trial, which also uses a two-stage design, indicated clinical activity in the first stage, allowing for expansion of enrollment, which was recently completed. Conclusive data from this study are expected within 12 months.
A planned Phase 1 dose escalation study at the National Cancer Institute (NCI) investigating the safety of MVA-BN Brachyury administered intravenously into patients with brachyury positive tumors will initiate shortly.
Commercial manufacturing

Construction of our fill and finish facility is progressing according to plan, and we expect the building to be completed by end of 2019, which is the peak investment year with approximately DKK 270 million in total investments.
The addition of a large-scale fill and finish line to our existing bulk production will significantly expand our manufacturing capabilities, allowing for integration of the products from GSK, while also enabling us to continuously support our partnerships, including fulfilling our smallpox vaccine contract with the U.S. government.
To support simultaneous manufacturing of multiple products, we will invest in a new, independent clean room suite at our existing facility to be initiated in 2020.
FINANCIALS AND OUTLOOK

Financial results for the first nine months in line with expectations

Revenue generated for the nine months ending September 30, 2019 was DKK 372 million/USD 54 million (DKK 319 million/USD 47 million in the first nine months of 2018).
The income before interest and tax (EBIT) was a loss of DKK 298 million/USD 43 million (loss of DKK 261 million/USD 38 million in the first nine months of 2018).
As of September 30, 2019, the Group’s cash preparedness was DKK 1,740 million/USD 254 million (DKK 2,314 million/USD 337 million as of December 31, 2018), including unutilized credit lines of DKK 244 million/USD 36 million (DKK 244 million/USD 36 million as of December 31, 2018).
Danish kroner (DKK) is the Company’s reporting currency. The USD figures provided above are based upon an assumed exchange rate of DKK 6.86 per 1.00 USD, which was the exchange rate as of September 30, 2019.

Outlook for 2019 maintained
The majority of our 2019 revenues are related to the production and release of bulk smallpox vaccine for the U.S. Government, most of which will occur in the fourth quarter of 2019. Hence, we maintain our financial expectations for 2019 as announced on March 21, 2019 with revenues of approximately DKK 600 million/USD 92 million for the full year and a loss before interest and tax (EBIT) of approximately DKK 360 million/USD 55 million. The expected cash preparedness at year-end was revised in October from approximately DKK 1,600 million/USD 246 million to approximately DKK 1,000 million/USD 154 million as a result of the announced acquisition of two vaccines from GlaxoSmithKline.

A detailed guidance for 2020 will be provided in connection with the publication of the Company’s annual report for 2019. However, pending a successful closing of the transaction with GSK, the Company is able to confirm that it will be profitable on an EBITDA basis already in 2020, exclusive of the sale of the Priority Review Voucher, which was awarded the Company by FDA upon the approval of JYNNEOS in September.

The financial expectations are based on an exchange rate of DKK 6.50 per 1.00 USD. For further details regarding assumptions behind the guidance see the Annual Report 2018.

Financial calendar 2019/2020
The date for announcement of the 2019 annual report has been changed to February 20, 2020.

November 27, 2019 Extraordinary General Meeting *
February 20, 2020 2019 Annual Report
April 21, 2020 Annual General Meeting **
May 14, 2020 First quarterly report (Q1) for the three-month period ended March 31, 2020
August 26, 2020 Half-year report (Q2) for the six-month period ended 30 June 2020
November 11, 2020 Third quarterly report (Q3) for the nine-month period ended 30 September 2020
* An extraordinary general meeting has been convened to seek shareholder approval to increase the Company’s share capital through a planned rights issue in the first half of 2020 with pre-emptive rights for the existing shareholders in order to obtain financing of costs associated with the acquisition of Rabipur/RabAvert and Encepur. See www.bavarian-nordic.com/egm for additional information.

** Pursuant to Article 12 of the Articles of Association, shareholders who wish to submit a request for proposals for consideration at the annual general meeting must lodge this with the Company no later than Wednesday, March 11, 2020.

OTHER DEVELOPMENTS

Incentive programs for employees and executive management in Bavarian Nordic
The board of directors has today decided to issue warrants to executive management and certain employees in the Bavarian Nordic Group. The decision is made in accordance with the shareholder authorization for the board of directors adopted as Article 5b of the Articles of Association and the Company’s guidelines regarding incentive programs.

The warrant program entails the issuance of 564,585 warrants in total which entitle the warrant holders to subscribe for up to 564,585 shares in total with a nominal value of DKK 10 each at an exercise price of DKK 185.4 per share. The warrants may be exercised wholly or partly during eight fixed subscription periods during 2023 and 2024.

The value of each warrant equals DKK 45.5 and is calculated on the Black-Scholes model with a risk-free interest rate of -0.69 per cent and on the historical volatility of the shares. The calculation is based on a share price of DKK 154.05.

Furthermore, the Company introduces a three-year incentive program in January 2020 for all employees in the Bavarian Nordic Group, with the exception of employees receiving warrants. The program is a cash bonus program based on the development in the Company’s share price. The incentive program will not have a dilutive effect on the shareholders.

Each employee participating in the program is awarded so-called phantom shares every month of employment until 31 December 2022. The exercise price is DKK 185.4. The phantom shares may be exercised in January 2023, only if the Company’s share price by then exceeds the exercise price by at least DKK 5. In that case, each phantom share will yield a cash bonus equivalent to DKK 1 per point the share price exceeds the exercise price.

Based on the current number of employees in the Group eligible for participating in the program, the program will comprise up to 72,288 phantom shares. The average value of each phantom share granted equals DKK 27.7 calculated on the basis of the Black-Scholes model with a risk-free interest rate of -0.69 per cent and on the historical volatility of the shares. The calculation is based on a share price of DKK 154.05.

Conference call and webcast
The management of Bavarian Nordic will host a conference call today at 2 pm CET (8 am ET) to present the interim results followed by a Q&A session. A listen-only version of the call can be accessed via View Source To join the Q&A session, use one of the following dial-in numbers: Denmark: +45 32 72 80 42, UK: +44 (0) 844 571 8892, USA: +1 631-510-7495. Participant code is 8772778.

On November 7, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended September 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 7, 2019, View Source [SID1234550663]).

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"In the third quarter, we made meaningful progress towards our clinical and regulatory milestones," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We received IMPD and IND clearance for our global Phase 1 trial for PKD, treated our first patient for LAD-I and continued enrollment of our Phase 1 trial of RP-A501 for Danon disease. We also announced promising long-term data from our Phase 1/2 trial of first-generation RP-L102 for Fanconi Anemia supporting sustained clinical improvement with robust engraftment exceeding the 10% threshold agreed to by regulators for the upcoming global Phase 2 trial. We hope to further optimize these responses with our ‘Process B’ of RP-L102 designed to enable consistent results with commercial-grade product without cytotoxic conditioning. We look forward to data from ‘Process B’ later this year, along with initial Phase 1 data from our RP-L201 trial for LAD-I."

Dr. Shah continued, "We now have four gene therapy candidates in the clinic, meeting one of our 2019 corporate goals and reinforcing our multi-platform strategy. As we advance our first candidate into late-stage development, we remain dedicated to our pursuit of potentially curative treatments for patients contending with rare genetic diseases."

Recent Pipeline Developments

Commencement of registration-enabling Phase 2 study for Fanconi Anemia (FA). Patient enrollment is ongoing in the global Phase 2 trial. The study initiation follows recent alignment from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) on the trial design and the primary endpoint. Resistance to mitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum time point of one year will serve as the primary endpoint and potentially as a surrogate endpoint for accelerated approval. Patient dosing is anticipated to begin in the fourth quarter with preliminary Phase 2 data anticipated in 2020.
Long-term Phase 1/2 clinical data of RP-L102 for FA "Process A" presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress. Long-term patient follow-up data demonstrate evidence of increasing and durable engraftment leading to bone marrow restoration exceeding the 10% threshold agreed to by the FDA and EMA for the ongoing registration-enabling Phase 2 trial. In patient 02002, who received what we consider adequate drug product, hemoglobin levels are now similar to those in the first year after birth, suggesting hematologic correction over the long term. Preliminary results from the first two patients receiving "Process B" of RP-L102 are anticipated in the fourth quarter.
Global Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD) initiates enrollment. Rocket received Investigational Medicinal Product Dossier (IMPD) clearance and Investigational New Drug (IND) application approval of RP-L301 during the quarter. The open-label, single-arm, clinical trial will enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301. Preliminary data are anticipated in 2020.
Patient dosing continues in Phase 1/2 registrational trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. The Phase 2 portion of the trial will evaluate overall survival. Preliminary Phase 1 data will be presented in the fourth quarter.
Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates reporting Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
Anticipated Milestones

FA (RP-L102)
Global Phase 2 trial first patient treatment (4Q19)
Initial Phase 1 data for "Process B" (4Q19)
Additional Phase 1 "Process B" data (1H20)
Preliminary Phase 2 data (2H20)
Danon Disease (RP-A501)
Phase 1 data (2020)
LAD-I (RP-L201)
Initial Phase 1 data (4Q19)
Phase 1 data update (1H20)
Commence enrollment of Phase 2 study (2H20)
PKD (RP-L301)
Preliminary Phase 1 data (2H20)
IMO (RP-L401)
Initiation of clinical study (2H20)
Upcoming Investor Conferences

Barclays Gene Editing & Gene Therapy Summit—November 13, 2019 in New York, N.Y.
Evercore ISI 2nd Annual HealthCONx Conference—December 3, 2019 in Boston, M.A.
Piper Jaffray’s 31st Annual Healthcare Conference—December 5, 2019 in New York, N.Y.
Third Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2019, were $240.6 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $14.8 million for the three months ended September 30, 2019, compared to $13.1 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in clinical trial costs of $1.3 million.
G&A expenses. General and administrative expenses were $4.3 million for the three months ended September 30, 2019, compared to $2.3 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in non-cash stock compensation expense of $1.4 million and an increase in compensation and benefits expense of $0.3 million as a result of increased headcount.
Net loss. Net loss was $19.3 million or $0.38 per share (basic and diluted) for the three months ended September 30, 2019, compared to $16.1 million or $0.40 per share (basic and diluted) for the three months ended September 30, 2018.
Shares outstanding. 50,376,030 shares of common stock were outstanding as of September 30, 2019.
Financial Guidance

Cash position. As of September 30, 2019, we had cash, cash equivalents and investments of $240.6 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.

On November 7, 2019 MedX Health Corp. ("MedX" or the "Company") (TSX-V:MDX) reported that, pursuant to a non-brokered Private Placement Offering (the "Placement Offering"), it has completed an initial Closing of 1,251,000 Units effective November 4, 2019, to raise $150,120 from accredited investors pursuant to a Private Placement Offering announced on November 1, 2019 (Press release, MedX Health, NOV 7, 2019, View Source [SID1234550679]). Each Unit, priced at $0.12, comprises one fully paid common share and one share purchase warrant; each warrant entitles the holder to purchase one additional share at a price of $0.20 during the period up to two years from the closing date of the placement. The securities issued as a result of the Placement Offering will be restricted from trading for four months following each Closing. The Placement Offering is still open to subscribers, and further subscriptions may be accepted for further Closings. This Closing, and any further Closing of the Placement Offering, is subject to all relevant regulatory and other consents and approvals, including acceptance by the TSX Venture Exchange. In connection with the first Closing, a cash commission of $5,136 was paid and 42,800 Agent’s Warrants were issued.

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Following this Closing, MedX has 143,169,762 common shares issued and outstanding.

On November 7, 2019 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), reported that data related to TAVALISSE (fostamatinib disodium hexahydrate) tablets has been accepted for two poster presentations at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition to be held December 7-10, 2019, in Orlando, FL (Press release, Rigel, NOV 7, 2019, View Source;exposition-300953564.html [SID1234550696]).

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Rigel conducted a post-hoc data analysis from a Phase 3 clinical program of TAVALISSE in adult patients with immune thrombocytopenia (ITP). In this analysis, 32 patients received fostamatinib as a second-line therapy, and 78% (25/32) achieved ≥1 platelet count of ≥50,000/µL (without rescue therapy). Adverse events were manageable and consistent with those previously reported with fostamatinib.

"These data show a high response rate in the early-line treatment of adult ITP," said Raul Rodriguez, Rigel’s President and CEO. "We believe that these data, coupled with TAVALISSE’s differentiated and targeted mechanism of action, provide an attractive treatment approach for early-line patients. These patients represent the vast majority of the adult ITP population."

Additionally, in a Phase 2 open-label study of fostamatinib in patients with warm antibody autoimmune hemolytic anemia (wAIHA), data showed that 44% (11/25) of evaluable patients met the primary efficacy endpoint of a hemoglobin (Hgb) level >10 g/dL with an increase of ≥2 g/dL from baseline by week 24. Including one late responder at week 30, the overall response rate was 48% (12/25). Adverse events were manageable and consistent with those previously reported with fostamatinib.

Fostamatinib disodium hexahydrate is an oral drug designed to inhibit spleen tyrosine kinase (SYK), a key signaling component of the body’s immune process that leads to platelet destruction in ITP and proposed red blood cell destruction in AIHA. Fostamatinib is commercially available in the U.S. under the brand name TAVALISSE (fostamatinib disodium hexahydrate) tablets and is the first and only SYK inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment. TAVALISSE is currently being investigated in a Phase 3 trial for wAIHA, a rare, serious blood disorder for which there are no approved therapies.

Poster Presentations
Abstract #1069
Enhanced Responses to Fostamatinib as Second-Line Therapy and in Persistent Immune Thrombocytopenia (ITP) Patients
Session Name: 311. Disorders of Platelet Number or Function: Poster I
Presenter: Ralph Boccia
Date: Saturday, December 7, 2019
Presentation Time: 5:30 PM-7:30 PM EST
Location: Hall B (Orange County Convention Center)

Abstract #3518
Fostamatinib1, a Spleen Tyrosine Kinase (SYK) Inhibitor, for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA): Final Results of the Phase 2, Multicenter, Open-Label Study
Session Name: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Presenter: Kerry Rogers
Date: Monday, December 9, 2019
Presentation Time: 6:00 PM – 8:00 PM EST
Location: Hall B (Orange County Convention Center)

The conference abstracts can be accessed here.

To learn more about Rigel Pharmaceuticals and TAVALISSE visit booth #415 during ASH (Free ASH Whitepaper) 2019.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm antibody AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Please see www.TAVALISSE.com for full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE is a trademark of Rigel Pharmaceuticals, Inc.

On November 7, 2019 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage, immunotherapy and targeted oncology company, reported financial results and recent corporate highlights for the third quarter ended September 30, 2019 (Press release, Checkpoint Therapeutics, NOV 7, 2019, View Source [SID1234550712]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "We achieved a key inflection point in September with the presentation of positive interim clinical results for cosibelimab (formerly CK-301), our fully human anti-PD-L1 antibody, at the European Society for Medical Oncology ("ESMO") Congress 2019. These compelling clinical data demonstrated anti-tumor activity across multiple advanced cancers, with a 50% objective response rate observed in cutaneous squamous cell carcinoma ("CSCC"), a 40% objective response rate observed in non-small cell lung cancer ("NSCLC"), as well as a well-tolerated safety profile across all cohorts. These results highlight the potential of cosibelimab to be potentially differentiated from other drugs in its class, in addition to our intended commercial strategy to position cosibelimab as a lower-cost alternative to available anti-PD-1/L1 mAbs. Enrollment in this trial is ongoing and, with additional data, could support the submission of an initial Biologics License Application ("BLA") for cosibelimab in CSCC. We also look forward to soon reporting additional clinical data for CK-101, our novel, oral, third-generation epidermal growth factor receptor ("EGFR") inhibitor, in order to support the potential initiation of a Phase 3 clinical trial in first-line EGFR mutation-positive NSCLC."

Financial Results:

Cash Position: As of September 30, 2019, Checkpoint’s cash and cash equivalents totaled $13.1 million, compared to $13.2 million as of June 30, 2019, and $22.0 million as of December 31, 2018, a decrease of $0.1 million for the quarter and a decrease of $8.9 million year-to-date.
R&D Expenses: Research and development expenses for the third quarter of 2019 were $3.9 million, compared to $7.8 million for the third quarter of 2018, a decrease of $3.9 million. Research and development expenses for the third quarter of 2019 included $0.2 million of non-cash stock expenses, compared to $0.6 million in stock compensation expense for the third quarter of 2018. The Company expects that for the balance of 2019, research and development expenses will continue to remain lower than the comparable period in 2018.
G&A Expenses: General and administrative expenses for the third quarter of 2019 were $1.6 million, compared to $1.5 million for the third quarter of 2018, an increase of $0.1 million. General and administrative expenses for the third quarters of 2019 and 2018 each included $0.7 million of non-cash stock expenses.
Net Loss: Net loss attributable to common stockholders for the third quarter of 2019 was $5.2 million, or $0.15 per share, compared to a net loss of $9.3 million, or $0.32 per share, for the third quarter of 2018. The net loss for the third quarter of 2019 included $0.9 million of non-cash stock expenses, compared to $1.3 million for the third quarter of 2018.