On May 6, 2020 Exact Sciences Corp. (Nasdaq: EXAS) reported that the company generated revenue of $347.8 million for the first quarter ended Mar. 31, 2020, compared to $162.0 million for the same period of 2019 (Press release, Exact Sciences, MAY 6, 2020, View Source [SID1234557131]).

"Even in unprecedented times, cancer doesn’t stop. The coronavirus pandemic highlights and increases the need for novel ways to screen for cancer, detect it early, and guide treatment. Our Cologuard, Oncotype DX, and Paradigm tests meet that need," said Kevin Conroy, chairman and CEO. "In a world that is trying to avoid unnecessary physician office visits, invasive procedures, and treatments, our tests and deep pipeline are more valuable now than ever. We plan to play an even greater role in cancer screening and guiding therapy decisions after the coronavirus pandemic abates."

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First Quarter 2020 Financial Results

For the three-month period ended March 31, 2020, as compared to the same period of 2019 (where applicable):

Total revenue was $347.8 million, compared to $162.0 million

Screening revenue was $219.5 million, an increase of 35 percent

Precision Oncology revenue was $128.4 million

Gross margin including amortization of acquired intangible assets was 71 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 77 percent

Net loss was $105.7 million, or $0.71 per share, compared to a net loss of $82.9 million, or $0.66 per share

EBITDA was $(51.0) million and adjusted EBITDA was $(8.2) million

Non-cash interest expense related to convertible debt was $22.5 million, which included a non-cash loss on extinguishment of debt of $8.0 million, compared to $19.6 million, which included a non-cash loss on extinguishment of debt of $10.6 million

Cash, cash equivalents, and marketable securities were $1.2 billion at the end of the quarter, including $975.5 million, net, raised in relation to the issuance of new convertible notes and the repayment of a portion of previously issued convertible notes

Screening includes laboratory service revenue from Cologuard and revenue from Biomatrica products. Precision Oncology includes laboratory service revenue from global Oncotype DX products and the recent Paradigm acquisition.

Non-GAAP Disclosure
In addition to the company’s financial results determined in accordance with U.S. GAAP, the company provides non-GAAP measures that it determines to be useful in evaluating its operating performance. The company presents EBITDA, adjusted EBITDA, as well as non-GAAP gross margin and non-GAAP gross profit. EBITDA and adjusted EBITDA consist of net loss after adjustment for those items shown in the table below. The company defines non-GAAP gross profit and non-GAAP gross margin as GAAP gross profit and GAAP gross margin, respectively, excluding amortization of acquired intangible assets. The amortization of acquisition-related intangible assets used in the calculation of non-GAAP gross profit and non-GAAP gross margin pertain only to the amortization associated with developed technology acquired and recorded through purchase accounting transactions. The amortization of these intangible assets will recur in future periods until such intangible assets have

been fully amortized. The company believes that these non-GAAP measures are useful in evaluating the company’s operating performance. The company uses this non-GAAP financial information to evaluate ongoing operations and for internal planning and forecasting purposes. Non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental information purposes only, has limitations as an analytical tool and should not be considered in isolation or as a substitute for financial information presented in accordance with U.S. GAAP. For example, non-GAAP gross margin and non-GAAP gross profit exclude the amortization of acquired intangible assets although such measures include the revenue associated with the acquisitions. For a reconciliation of these non-GAAP measures to GAAP, see below "EBITDA and Adjusted EBITDA Reconciliations."

First Quarter Conference Call & Webcast
Company management will host a conference call and webcast on Wednesday, May 6, 2020, at 5 p.m. ET to discuss first quarter 2020 results. The webcast will be available at www.exactsciences.com. Domestic callers should dial 833-235-7650 and international callers should dial +1-647-689-4171.

An archive of the webcast will be available at www.exactsciences.com. A replay of the conference call will be available by calling 800-585-8367 domestically or 416-621-4642 internationally. The access code for the replay of the call is 7874149. The webcast, conference call and replay are open to all interested parties.

About Cologuard
Cologuard was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. Cologuard is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2016) and National Comprehensive Cancer Network (2016). Cologuard is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use Cologuard if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. Cologuard is not a replacement for colonoscopy in high risk patients. Cologuard performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. Cologuard performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again.

Medicare and most major insurers cover Cologuard. For more information about Cologuard, visit www.cologuardtest.com. Rx only.

About Oncotype DX
The Oncotype DX portfolio of breast, colon and prostate cancer tests applies advanced genomic science to reveal the unique biology of a tumor in order to optimize cancer treatment decisions. In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS. In prostate cancer, the Oncotype DX Genomic Prostate Score test predicts disease aggressiveness and further clarifies the current and future risk of the cancer prior to treatment intervention, and the Oncotype DX AR-V7 Nucleus Detect test helps determine which patients with metastatic castration-resistant prostate cancer (mCRPC) are resistant to androgen receptor (AR)-targeted therapies. The Oncotype DX AR-V7 Nucleus Detect test is performed by Epic Sciences at its centralized, CLIA-certified laboratory in San Diego and offered exclusively by Exact Sciences. With more than 1 million patients tested in more than 90 countries, the Oncotype DX tests have redefined personalized medicine by making genomics a critical part of cancer diagnosis and treatment. To learn more about Oncotype DX tests, visit www.OncotypeIQ.com, www.MyBreastCancerTreatment.org or www.MyProstateCancerTreatment.org.

On May 6, 2020 MorphoSys (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported the results for the first quarter of 2020 (Press release, MorphoSys, MAY 6, 2020, View Source [SID1234557130]).

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Financial Highlights

Group revenue in the first quarter of 2020 increased to €251.2 million (Q1 2019: €13.5 million), mainly driven by the upfront payment from the Incyte collaboration.
EBIT increased to €213.6 million (Q1 2019: €-23.6 million).
At the end of the first quarter of 2020, MorphoSys has a liquidity position of €1.1 billion (December 31, 2019: €357.4 million).
Financial guidance for 2020 re-affirmed (Group revenues €280 to €290 million, R&D expenses €130 to €140 million, EBIT €-15 to €5 million).

Corporate and Program Updates

Tafasitamab (MOR208);
Collaboration and licensing agreement with Incyte to further develop and commercialize tafasitamab globally
Priority Review for BLA submission granted by U.S. FDA for tafasitamab in combination with lenalidomide for relapsed/refractory diffuse large B cell lymphoma (r/r DLBCL)
Expanded Access Program (EAP) for tafasitamab in the U.S. initiated
MOR202: First phase 3 patient in mainland China treated in multiple myeloma
Roland Wandeler, Ph.D., appointed Chief Commercial Officer and member of the MorphoSys AG Management Board to lead global commercial operations and oversee the Company’s U.S. operations with its planned launch of tafasitamab
In April 2020, MorphoSys decided not to exercise its exclusive option granted under the agreement with Vivoryon Therapeutics, signed in July 2019, to license Vivoryon’s small molecule QPCTL inhibitors in the field of oncology. This decision was based on the thorough analysis of data from preclinical validation studies. MorphoSys owns a minority stake in Vivoryon Therapeutics that is based on an equity investment in 2019

Update on Impact of the Global COVID-19 Pandemic:

MorphoSys acknowledges the impact of the global COVID-19 pandemic on health systems and broader society worldwide and the resulting potential impact on preclinical and clinical programs and in particular on clinical trials. Beyond the previously communicated measures to mitigate the impact of the pandemic on MorphoSys’ employees, patients and the broader community, additional measures may need to be implemented in the future, including potential adjustments to clinical trials due to factors including restrictions of visits to healthcare facilities, increased demands on the health services and changes to trial staff availability. Consequently, MorphoSys is continuously monitoring the situation and deciding how to proceed in its clinical trials on a "trial-by-trial" and "country-by-country" basis to ensure patient and site personnel safety and data integrity
Despite the uncertainty caused by the COVID-19 pandemic in the US, launch preparations continue including through digital channels to ensure launch readiness of MorphoSys and Incyte should tafasitamab be approved on or before its PDUFA date of August 30, 2020
Enrollment of patients in all tafasitamab related clinical trials is currently expected to continue as planned, but the above-mentioned restrictions might lead to future delays in timelines
Enrollment/screening of patients in the M-PLACE study for MOR202 has been temporarily paused
"Despite the ongoing global COVID-19 pandemic, we have made significant progress during the first quarter of 2020. MorphoSys had another strong quarter of execution across the entire organization. We completed hiring and onboarding of our sales force, switched to virtual interactions with the medical community to overcome travel restrictions and have been able to further raise the awareness of our company," commented Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "With our partnership with Incyte and having been granted Priority Review by the FDA, we are continuously working towards bringing our lead product candidate tafasitamab to patients needing new therapeutic options. We are excited that Dr. Roland Wandeler has joined our leadership team as Chief Commercial Officer with a strong track record of growing businesses internationally. During these challenging times, the safety and well-being of our employees and patients continue to be our top priority and we remain committed to taking every necessary precaution to help reduce the spread of the virus and to break the cycle of infection."

"The Company is continuously gearing up for the upcoming launch of tafasitamab, if approved by the FDA. With a strong financial position and together with our partner Incyte, we are well positioned for the next chapter of the Company’s development," added Jens Holstein, Chief Financial Officer of MorphoSys. "The first quarter was positively impacted by the upfront payment from our partnership with Incyte, which was cleared by the antitrust authorities. In addition, as part of the collaboration agreement, Incyte also purchased ~2.8% of MorphoSys registered share capital in American Depositary Shares (ADSs) at a price of $41.32 per ADS."

Financial Review for Q1 2020 (IFRS)

In Q1 2020, MorphoSys continued to focus on applying its proprietary technology and expertise to the research and development of innovative drug candidates, both for partners and for its own account. Group revenues for the first quarter of 2020 rose to €251.2 million (2019: €13.5 million). The increase was primarily driven by the collaboration and licensing agreement with Incyte to further develop and commercialize tafasitamab globally. Revenues for the first quarter of 2020 include success-based payments of €10.3 million, primarily from Janssen (Q1 2019 success-based payments: €11.0 million).

In the Proprietary Development segment, MorphoSys focuses on research and clinical development of its own drug candidates in the fields of cancer and inflammation. In Q1 2020, this segment recorded revenues of €240.4 million (Q1 2019: €5.8 million), with the increase primarily driven by the collaboration and licensing agreement with Incyte.

In the Partnered Discovery segment, MorphoSys applies its proprietary technology to discover new drug candidates for pharmaceutical companies, benefiting from its partners’ development advancements through R&D funding, licensing fees, success-based milestone payments and royalties. Revenues in the Partnered Discovery segment increased from €7.8 million in Q1 2019 to €10.8 million in Q1 2020.

Total operating expenses in Q1 2020 increased to €-47.7 million from €-37.3 million in Q1 2019, based on the ramp-up of preparations for a potential tafasitamab U.S. commercialization as well as the build-up of MorphoSys US Inc. In the first quarter of 2020, cost of sales amounted to €3.3 million (Q1 2019: €5.0 million). In Q1 2020, research and development expenses amounted to €21.5 million, as compared to €24.7 million in Q1 2019. Selling expenses increased to €12.8 million (Q1 2019: €1.7 million), and general and administrative expenses increased from €5.9 million in Q1 2019 to €10.1 million in the first quarter of 2020, both primarily as a result of higher personnel expenses and expenses for external services.

Earnings before interest and taxes (EBIT) amounted to €213.6 million in Q1 2020 (Q1 2019: €-23.6 million), with the increase primarily driven by the collaboration and licensing agreement with Incyte. The Proprietary Development segment reported an EBIT of €210.8 million (Q1 2019: €-25.0 million). EBIT in the Partnered Discovery segment was €8.5 million (Q1 2019: €5.5 million). In 2020, the consolidated net profit amounted to €195.5 million (consolidated net loss Q1 2019: €-22.7 million). Basic earnings per share for 2020 was €6.12 (loss per share Q1 2019: €-0.72).

On March 31, 2020, the Group’s liquidity position amounted to €1,132.1 million, reported on the balance sheet under the line items "cash and cash equivalents"; "financial assets at fair value through profit or loss"; and current and non-current "other financial assets at amortized cost". The number of shares issued totaled 32,890,046 at the end of Q1 2020 (year-end 2019: 31,957,958).

Financial Guidance and Operational Outlook for 2020

For the financial year 2020, MorphoSys confirmed its financial guidance. Management continues to expect Group revenues in the range of €280 to €290 million. R&D expenses are anticipated in a corridor of €130 to €140 million. The Company expects earnings before interest and taxes (EBIT) of €-15 to €5 million. This guidance is based on constant currency exchange rates and does not include any contributions from tafasitamab revenues and any effects from potential in-licensing or co-development deals for new development candidates. The operational and financial guidance might potentially be impacted by the ongoing global COVID-19 crisis on MorphoSys’ business operations including but not limited to the Company’s supply chain, clinical trial conduct, as well as timelines for regulatory and commercial execution. While MorphoSys is maintaining its previously communicated guidance on its clinical trials, these could potentially be affected in terms of patient enrollment and data collection timelines, among other factors.

In its Proprietary Development segment, MorphoSys expects the following events and activities in 2020:

Tafasitamab (MOR208)

MorphoSys and Incyte continue launch preparations including through digital channels to ensure launch readiness should tafasitamab be approved on or before its PDUFA date of August 30, 2020; Despite the uncertainty caused by the COVID-19 pandemic in the U.S., to date there has not been a change of the PDUFA date of August 30, 2020 for tafasitamab
Support of Incyte for the submission of a marketing authorization application for tafasitamab in combination with lenalidomide for r/r DLBCL to the European Medicines Agency (EMA) by mid-2020; Incyte has exclusive commercialization rights outside of the U.S.; MorphoSys is not currently aware of the COVID-19 pandemic negatively impacting the expected achievement of a regulatory filing for tafasitamab with the EMA
Continue phase 1b study First-MIND in previously untreated DLBCL
Continue pivotal phase 3 trial evaluating tafasitamab plus bendamustine in r/r DLBCL in comparison to rituximab and bendamustine (B-MIND trial)
Continue phase 2 COSMOS trial of tafasitamab with idelalisib and venetoclax in CLL/SLL
Expansion of tafasitamab’s clinical development beyond DLBCL under the collaboration and licensing agreement with Incyte
Continue expansion of the commercial infrastructure and strategic presence in the U.S. to ensure tafasitamab launch readiness, matched by the resources provided by Incyte

MOR202

I-Mab: Continue pivotal development program with MOR202 in multiple myeloma in the Chinese region
MorphoSys: Continue clinical development of MOR202 in membranous nephropathy as well as other autoimmune indications; enrollment/screening of patients in the M-PLACE study temporarily paused due to COVID-19. This could lead to delays in previously communicated timelines
Otilimab (MOR103/GSK3196165): GSK to continue clinical phase 3 development program with otilimab in rheumatoid arthritis (recruitment currently proactively paused by GSK due to COVID-19).

MOR107: Continue preclinical investigation of MOR107 with a focus on oncology indications.

In its Partnered Discovery segment, MorphoSys expects the following events in 2020:

Tremfya(R) (guselkumab):

Janssen is currently conducting a series of clinical studies with Tremfya(R) (guselkumab) in various indications that could generate data during 2020. In 2019, Janssen submitted marketing authorization applications to the U.S. FDA and EMA for Tremfya(R) for the treatment of psoriatic arthritis. Decisions on these applications could potentially be made in 2020.

Other partnered programs: Publication of clinical data and achievement of regulatory milestones from other partnered programs may occur during 2020. Whether, when and to what extent news will be published following the primary completion of trials in the Partnered Discovery segment is at the full discretion of MorphoSys’ partners.

MorphoSys will continue to support its proprietary development activities by evaluating potential in-licensing, co-development, and/or acquisition opportunities or the potential initiation of new proprietary development programs with the goal of maintaining and expanding the Company’s position in its current therapeutic and technological fields of activities.

* Percentage point

** Including MOR107, which concluded a phase 1 study in 2017 and is currently in preclinical investigation with a focus on oncology indications. Tremfya(R) is still considered as a clinical program due to ongoing studies in various indications

*** Including otilimab (MOR103/GSK3196165), which is fully out-licensed to GSK

MorphoSys will hold its conference call and webcast tomorrow, May 7, 2020, to present the first quarter financial results 2020 and the further outlook for 2020.

Dial-in number for the analyst conference call (in English) at 2:00pm CEST; 1:00pm BST; 8:00am EDT:

Germany: +49 69 201 744 220
For UK residents: +44 203 009 2470
For US residents: +1 877 423 0830
(all numbers reachable from any geography)

Participant PIN: 14809293#

Please dial in 10 minutes before the beginning of the conference.

A live webcast and slides will be made available at www.morphosys.com.

Approximately two hours after the call, a slide-synchronized audio replay of the conference and a transcript of the prepared remarks will be available on View Source

The interim statement for the first quarter of 2020 (IFRS) is available online:
View Source

About tafasitamab
Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. In January 2020, MorphoSys and Incyte Corporation entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. In the U.S., MorphoSys and Incyte will co-commercialize tafasitamab, outside the U.S., Incyte will have exclusive commercialization rights. Tafasitamab is being clinically investigated as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL) who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). The ongoing phase 3 study B-MIND assesses the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL. In addition, tafasitamab is currently being investigated in patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On May 6, 2020 Immunomedics, Inc., (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates (ADC), reported financial results for the first quarter of 2020 (Press release, Immunomedics, MAY 6, 2020, View Source [SID1234557129]). Please refer to the Company’s Quarterly Report on Form 10-Q for more details on the Company’s financial results.

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As the world responds to the outbreak of COVID-19, the thoughts of the Immunomedics team are with those affected, the staff who are providing essential services and healthcare workers fighting at the front lines of this pandemic. We also thank our colleagues, who are working tirelessly under the constraints of the current environment, to support the Company’s mission of delivering breakthrough treatments to those in need.

"We entered 2020 with strong momentum across our operations," stated Dr. Behzad Aghazadeh, Executive Chairman of Immunomedics. "Following a successful biologics license application resubmission in late 2019, we were pleased to receive accelerated approval for Trodelvy in late April and accomplished our goal of bringing this therapy to patients with metastatic triple-negative breast cancer (mTNBC). Trodelvy was launched within days of receiving FDA approval and transformed Immunomedics into a fully-integrated biopharmaceutical company in the process. We also announced the early stoppage of our confirmatory Phase 3 ASCENT study due to compelling evidence of efficacy and look forward to reporting topline study results in mid-2020. These data are expected to establish Trodelvy as a new standard of care in mTNBC and help improve the lives of people with hard-to-treat cancers worldwide. Finally, we significantly strengthened our balance sheet with an over-subscribed follow-on offering that will allow us to build on our momentum during the growth period ahead."

Recent Highlights and New Developments

The Phase 3 confirmatory ASCENT study of Trodelvy in third-line mTNBC was halted early due to compelling evidence of efficacy across multiple endpoints, based on the unanimous recommendation by the independent Data Safety Monitoring Committee during its recent routine review of the study. Topline readout is expected around mid-2020.
Trodelvy received accelerated approval from the FDA for the treatment of adult patients with mTNBC who have received at least two prior therapies for metastatic disease. Trodelvy is the first ADC approved by the FDA specifically for previously-treated mTNBC and is also the first FDA-approved anti-Trop-2 ADC.

Commercial launch of Trodelvy is underway, with the first patient treated within days of FDA approval and a National Drug Rebate Agreement with The Centers for Medicare & Medicaid Services fully executed.

The Company entered into a clinical collaboration with Dana-Farber Cancer Institute to conduct two Phase 2 studies to evaluate the safety and efficacy of combining Trodelvy with pembrolizumab (Keytruda), Merck’s anti-programmed cell death protein 1 (PD-1) antibody, in patients with (i) mTNBC and (ii) hormone receptor positive (HR+)/human epidermal growth factor receptor 2-negative (HER2–) metastatic breast cancer (mBC). In the mTNBC study, approximately 110 newly-diagnosed patients, who are programmed cell death ligand 1 (PD-L1)-negative, will be randomized to receive the combination of Trodelvy and pembrolizumab or Trodelvy alone. The same combination of Trodelvy and pembrolizumab will be used in the second randomized study in approximately 110 hormonal treatment- and chemotherapy-refractory patients with PD-L1-positive, HR+/HER2– mBC. Primary endpoint for both studies is progression-free survival. Other clinical outcome measures, including overall survival, objective response rate by RECIST 1.1, duration of response, and clinical benefit rate, will be used as secondary endpoints.

An abstract containing early results from cohort 2 of TROPHY U-01 study of Trodelvy in metastatic urothelial cancer patients who are platinum-ineligible and progressed after prior checkpoint inhibitor therapy has been accepted for poster presentation at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) virtual meeting. Topline data from the full first cohort of 100 patients with prior platinum-based and PD-1 or PD-L1 inhibitor therapies are expected to be available in the second half of 2020.

Additionally, an abstract from a Phase 1/2 study of Trodelvy in patients with previously-treated metastatic endometrial cancer has also been accepted for poster presentation at the 2020 ASCO (Free ASCO Whitepaper) virtual meeting.

In adhering to federal and local guidelines on COVID-19, in March 2020 the Company proactively suspended activation of new study sites and slowed the enrollment of new patients for certain clinical studies. The Company continues to work closely with investigators and the FDA to ensure the safety of patients considering the health risks posed by the pandemic, while preserving the integrity of the studies.

Enrollment into the Phase 3 TROPiCS-02 study of Trodelvy in HR+/HER2– mBC is expected to resume in late May 2020 at select sites that have been carefully vetted for their ability to ensure normal clinical trial operations and patient safety.

The Company’s leadership team has been strengthened by the appointments of Harout Semerjian as President and Chief Executive Officer, and Dr. Loretta M. Itri as Chief Medical Officer. Dr. Behzad Aghazadeh remains as Executive Chairman while Scott Canute has resumed his role as a member of the Board of Directors.

John Stubenrauch has been appointed Senior Vice President, Global Head of Manufacturing. Mr. Stubenrauch has decades of experience in global commercial manufacturing across organizations, including AstraZeneca and Merck. Dr. Morris Rosenberg, who helped support the manufacturing operations leading up to Trodelvy’s commercial launch, will be stepping down from the management team, effective May 15, 2020, to pursue the next chapter in his career.

The Company raised approximately $464.6 million in net proceeds through a public offering of common stock.
Financial Results for the First Quarter of 2020

Total costs and expenses were $82.0 million for the three months ended March 31, 2020, compared to $79.6 million for the comparable quarter ended March 31, 2019. The increase was primarily due to a $4.3 million increase in research and development expenses and a $0.2 million increase in sales and marketing expenses, partially offset by a $2.1 million decrease in general and administrative expenses.

Interest expense was $13.5 million for the three months ended March 31, 2020, compared to $10.0 million for the comparable quarter March 31, 2019. The increase was due primarily to changes in the fair value of our debt balances as a result of the agreement with RPI Finance Trust.

Net loss attributable to stockholders was $93.0 million, or $0.44 per share, for the three months ended March 31, 2020, compared to $87.3 million, or $0.46 per share, for the comparable quarter ended March 31, 2019.

As of March 31, 2020, the Company had $540.6 million in cash, cash equivalents, and marketable securities. On May 1, 2020, the Company closed on its previously announced underwritten public offering of common stock with net proceeds of approximately $464.6 million. The Company believes its projected financial resources are adequate to support commercial launch of Trodelvy in the United States in mTNBC; continue to expand the clinical development programs for Trodelvy; invest in the broader clinical development of the ADC platform (including IMMU-130 and IMMU-140); continued scale up of manufacturing and manufacturing process improvements; and general working capital requirements.

Conference Call

The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Time to discuss first quarter 2020 financial results and provide a corporate update. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 9146008. The conference call will be webcast via the Investors page on the Company’s website at View Source Approximately two hours following the live event, a webcast replay of the conference call will be available on the Company’s website for approximately 30 days.

On May 6, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance (Press release, Ultragenyx Pharmaceutical, MAY 6, 2020, View Source [SID1234557128]).

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"The global coronavirus pandemic has created unprecedented challenges for the world at large, and I am proud of the company’s continued efforts to assure an uninterrupted supply of our therapies" said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We delivered strong financial results in the first quarter, and our commercial business so far has been manageably impacted and resilient, though the global crisis continues. The FDA reviews for Crysvita for TIO and UX007 for LC-FAOD are on track to be completed in the coming months. Our gene therapy franchise has recently been bolstered by our recent manufacturing collaboration with Daiichi Sankyo, and our two clinical-stage gene therapy programs continue to proceed."

COVID-19 Update
As the COVID-19 pandemic continues to evolve, the top priority for Ultragenyx is the health and safety of its extended community, including patients, caregivers, healthcare providers, partners, and employees. To date, the company has been able to maintain an uninterrupted supply of medicine to patients around the world as no significant disruptions to manufacturing or distribution activities have been experienced. Even if there were an interruption in the supply chain, the company does not anticipate a shortage of medicine in the near term due to ample levels of inventory currently available.

First Quarter 2020 Financial Results

Net Revenues
For the first quarter of 2020, Ultragenyx reported $36.3 million in total revenue. Ultragenyx recognized $28.8 million in total Crysvita revenue for Ultragenyx territories, which includes $27.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $1.6 million. Non-cash royalty revenue related to European Crysvita royalties were $2.6 million. Mepsevii (vestronidase alfa) product revenue for the first quarter of 2020 was $3.4 million, and UX007 named patient revenue was $1.4 million.

Operating Expenses
Total operating expenses for the first quarter of 2020 were $157.0 million, which includes $25.0 million to maintain the option to acquire GeneTx after the acceptance of the investigational new drug application (IND) for GTX-102, $7.0 million for an exclusive, sublicensable, worldwide license to NAV AAV8 and AAV9 Vectors from REGENXBIO, Inc and non-cash stock-based compensation of $20.2 million. The cost of sales for the three months ended March 31, 2020 was negative $3.5 million due to a credit with an estimated value of $4.6 million that was agreed to during the quarter with a contract manufacturer in exchange for certain Mepsevii inventory batches that did not meet specified quality standards in prior quarters. Total operating expenses in the first quarter of 2019 were $117.4 million, which includes non-cash stock-based compensation of $20.2 million. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

For the first quarter of 2020, Ultragenyx reported a net loss of $119.0 million, or $2.05 per share, basic and diluted, compared with a net loss for the first quarter of 2019 of $96.8 million, or $1.82 per share, basic and diluted. The loss for the first quarter of 2020 includes unrealized gains of $7.7 million from the fair value adjustments on the investment in Arcturus equity securities and non-cash interest expense on the liability related to the sale of future royalties of $8.1 million. The net loss for the first quarter of 2020 reflected cash used in operations of $95.2 million compared to $95.8 million for the same period in 2019.

Cash, Cash Equivalents and Investments
Cash, cash equivalents, and investments were $705.0 million as of March 31, 2020. This includes $75.0 million from the Daiichi Sankyo common stock purchase which closed in March 2020, but excludes the $125.0 million upfront license payment from the Daiichi Sankyo strategic manufacturing partnership which was received in April 2020.

2020 Guidance

Crysvita Guidance in Ultragenyx Territories
The company currently maintains the guidance range for 2020 Crysvita revenue in the Ultragenyx territories to be between $125.0 million and $140.0 million, although it continues to monitor the COVID-19 pandemic situation. Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

2020 Expected Net Cash Burn Rate
The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with a significant anticipated growth in revenue.

Corporate Update

Gene Therapy Manufacturing: Strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo

Ultragenyx granted Daiichi Sankyo a non-exclusive license on March 31, 2020 to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. Daiichi Sankyo made an upfront payment in April 2020 of $125.0 million pursuant to the license agreement and in March 2020 closed on the purchase of 1,243,913 shares of Ultragenyx common stock in exchange for $75.0 million, or an effective purchase price of approximately $60 per share. Daiichi Sankyo will pay an additional $25.0 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms, as well as single-digit royalties on net sales of products manufactured in either system.
Program Updates and Upcoming Milestones

UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): New Drug Application (NDA) under review by U.S. FDA

The U.S. FDA is currently reviewing the NDA and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The review process is currently on track for a decision by the PDUFA date. The anticipated clinical and manufacturing inspections for UX007 have been successfully completed and, at this time, the FDA has not indicated that any additional inspections would be required for completion of their review.
Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) under review by U.S. FDA

The U.S. FDA accepted for review the sBLA and has set a PDUFA date of June 18, 2020. The review process is currently on track for a decision by the PDUFA date and the FDA has not indicated, at this time, that any inspections would be required for completion of their review.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose cohort of Phase 1/2 study; prophylactic steroid cohort planned and Phase 3 study planning underway

Positive data from this study indicated two confirmed responders and a potential third responder out of three total patients in Cohort 3, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses. An update with currently available data from the first three cohorts of the study will be presented at the virtual American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) on May 13.
A fourth cohort of three patients at the same 1.0 × 10^13 GC/kg dose is planned, using prophylactic steroids. Dosing in this cohort is currently on hold, but data are still expected in the second half of 2020, barring significant further delays related to COVID-19. Ultragenyx is simultaneously planning the Phase 3 study and is currently planning to hold an end-of-phase 2 meeting with the FDA based on the first three cohorts. The Phase 3 study is currently expected to begin enrollment in the first half of 2021.
DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in second quarter 2020

Enrollment is complete in the confirmatory cohort of three patients at the second dose level (6.0 x 10^12 GC/kg). An update with currently available data from the confirmatory cohort will be presented at the ASGCT (Free ASGCT Whitepaper) meeting on May 15. Certain data may not be available for later patient site visits due to COVID-19 related delays. Depending on the results of the confirmatory cohort, a Phase 3 study could begin in late 2020.
GTX-102 for Angelman Syndrome (AS): Partnered program with GeneTx; First two patients enrolled

The two patients in the first cohort of the Phase 1/2 study of GTX-102 have been enrolled and continue through dose escalation. Preliminary data are expected in first half of 2021.
In February 2020, Ultragenyx paid $25.0 million after the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.
In May 2020, the U.S. FDA granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome.
UX701 for Wilson Disease: IND anticipated by the end of the year

An IND application for UX701 gene therapy for Wilson disease, a larger rare metabolic disease, is expected by the end of 2020 barring any delays in nonclinical or manufacturing activities. UX701 will be the second clinical program to utilize the company’s HeLa manufacturing system, in addition to the hemophilia A program being developed by our collaborative partner, Bayer.
Conference Call and Webcast Information

Ultragenyx will host a conference call today, Wednesday, May 6, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss first quarter 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 9455159. The replay of the call will be available for one year.

On May 6, 2020 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2020 (Press release, Celldex Therapeutics, MAY 6, 2020, View Source [SID1234557127]).

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"Despite the ongoing challenges associated with the COVID-19 pandemic, Celldex continued to make considerable progress advancing our pipeline in the first quarter of 2020," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "Notably, the Company completed enrollment and treatment of all subjects in the Phase 1 healthy volunteer study of our KIT inhibitor, CDX-0159, which we intend to study in mast cell driven disorders. Results from this study have been accepted as a late-breaking poster presentation with voice over at the EAACI Annual Congress 2020 and will be presented in early June by Dr. Marcus Maurer, a leading medical expert in urticaria. We are excited about the promising data observed to date and, based on these results, have expanded development of CDX-0159. We are planning to initiate studies in chronic urticaria later this year.

"We are also completing preparations to advance CDX-527, the first candidate from our bispecific platform, into a Phase 1 study in refractory, advanced cancers and look forward to initiating this study in the second half of 2020. Finally, we continue to enroll patients in the ongoing studies of CDX-1140 and CDX-3379 and plan to report data updates from these programs later this year," concluded Marucci.

Recent Pipeline Highlights:

CDX-0159—a monoclonal antibody that specifically binds the KIT receptor and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells. In certain inflammatory diseases, such as chronic urticarias, mast cell degranulation plays a central role in the onset and progression of the disease.

Enrollment and treatment was recently completed in the ongoing Phase 1 randomized, double-blind, placebo-controlled, single ascending dose escalation study of CDX-0159 in healthy subjects. Subjects received a single intravenous infusion of CDX-0159 at 0.3, 1, 3, or 9 mg/kg or placebo. This study is designed to evaluate the safety profile, pharmacokinetics and pharmacodynamics of CDX-0159 and to select a dose for further study in mast cell driven diseases. The Phase 1 study also evaluates plasma tryptase levels in healthy subjects. Tryptase is an enzyme synthesized and secreted by mast cells and decreases in plasma tryptase levels reflect a systemic reduction in mast cell burden, even in healthy volunteers. If CDX-0159 is able to decrease systemic mast cell load in healthy volunteers, Celldex believes the drug candidate could have significant potential in mast cell driven diseases. Based on promising results observed to date, Celldex is expanding development of CDX-0159 into both chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CINDU).

— Results from the Phase 1 study of CDX-0159 have been accepted as a late-breaking poster presentation with voice over at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2020, which this year will be held digitally June 6-8, 2020. Following the press release issued this morning announcing the presentation, Celldex became aware that the full abstracts had been temporarily published on the EAACI website ahead of the lifting of the embargo. Information included in the published abstract was current as of the time of submission in March and did not include the full results that will be presented in June. Results published in the abstract outlined that mild infusion reactions were the most common adverse event occurring in 50% of subjects and did not appear dose related. The infusion reactions spontaneously resolved shortly after the infusions were completed. Transient, mild decreases in hemoglobin and white blood cell count were observed, which spontaneously resolved without intervention. Remarkably, patients who received a single 0.3 mg/kg dose of CDX-0159 decreased serum tryptase—a specific marker of mast cell load—by approximately 50% from baseline for a week, while a single dose at 1 or 3 mg/kg rapidly reduced serum tryptase below the levels of assay detection for at least 3 weeks and 6 weeks, respectively. The poster presentation with voice over in June will include complete, unblinded results from the study, including the 9 mg/kg cohort and pharmacokinetic, immunogenicity and stem cell factor data. The study will be presented by Dr. Marcus Maurer, Professor of Dermatology and Allergy and Director of Research at the Department of Dermatology and Allergy at the Allergie-Centrum-Charité of the Charité – Universitätsmedizin in Berlin. Dr. Maurer is also head of the Specialty Clinics for Urticaria, Mastocytosis, Pruritus and Angioedema and the Dermatological Allergology Lab. Dr. Maurer´s research focuses on the physiological and pathological functions of mast cells.

— The Company plans to initiate Phase 1b studies of CDX-0159 in CSU and CINDU, both mast cell-related diseases, by year end. CSU presents as itchy hives, angioedema or both for at least six weeks without a specific trigger; multiple episodes can play out over years or even decades. About 50% of patients with CSU achieve symptomatic control with antihistamines or leukotriene receptor antagonists. Omalizumab, an IgE inhibitor, provides relief for roughly half of the remaining antihistamine/leukotriene refractory patients. Consequently, there is a need for more effective later line therapies. CINDUs are forms of urticaria that have an attributable cause or trigger associated with them, typically resulting in hives or wheals. Celldex is exploring cold-induced and dermographism (scratch-induced) urticarias.
CDX-1140—a potent CD40 agonist that Celldex believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 dose-escalation study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas both the monotherapy and combination with CDX-301 dose escalation portions of the trial are complete with an identified maximum tolerated dose (MTD) and recommended Phase 2 dose of CDX-1140 at 1.5 mg/kg—one of the highest systemic dose levels in the CD40 agonist class. Expansion cohorts are actively recruiting including:

— CDX-1140 with KEYTRUDA (pembrolizumab) in patients who have progressed on checkpoint therapy; and,
— CDX-1140 with CDX-301 in patients with head and neck squamous cell carcinoma (HNSCC).

In addition, a combination of CDX-1140 with chemotherapy in first line metastatic pancreatic cancer is planned.
CDX-3379—a differentiated human monoclonal antibody designed to block the activity of ErbB3 (HER3). ErbB3 is expressed in many cancers, including HNSCC and is believed to be an important receptor regulating cancer cell growth and survival as well as resistance to targeted therapies.

Enrollment continues in the Phase 2 study of CDX-3379 in advanced HNSCC in combination with Erbitux (cetuximab) in Erbitux-resistant patients who have been previously treated with or are ineligible for checkpoint therapy. Data reported at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June suggested that observed antitumor activity with CDX-3379 might be associated with somatic mutations in the FAT1 and NOTCH1-3 genes—genes associated with tumor suppression. Based on these biomarker observations and the clinical activity observed in the ongoing Phase 2 study, the study was expanded (n= ~45 patients, including at least 15 patients with FAT1 mutations) to allow for an evaluation of the utility of biomarkers for patient selection.
Celldex continues to advance a robust preclinical portfolio and is preparing to advance the first candidate from the Company’s bispecific platform, CDX-527, into the clinic in the second half of 2020. CDX-527 uses Celldex’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway. The Phase 1 dose escalation study will enroll up to 90 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy, followed by tumor specific expansion cohorts to further evaluate the tolerability, biologic and anti-tumor effects of selected dose level(s) of CDX 527 in specific tumor types.

Recent Business Highlights:

In March 2020, Celldex announced a milestone payment related to an existing 2013 agreement with Rockefeller University under which Celldex performed manufacturing and development services for Rockefeller University’s portfolio of broadly neutralizing antibodies (bNAbs) against HIV, including two clinical-stage candidates 3BNC117 and 10-1074. These investigational agents have potential for use in HIV long-acting therapies for treatment and prevention, as well as cure strategies. This portfolio was licensed by Gilead Sciences in January of 2020 from Rockefeller University and pursuant to Celldex’s agreement with Rockefeller, Celldex received an upfront payment of $1.8 million as a result of this transaction and is eligible to receive additional payments from Rockefeller if this portfolio progresses through clinical and commercial development. Under the terms of the agreement, Celldex utilized internal capabilities in production cell line development, process development and GMP manufacturing to support the rapid and successful execution of Rockefeller University’s clinical development of 3BNC117 and 10-1074 and contributed to the IND and clinical studies by managing key IND-enabling studies and clinical sample testing for pharmacokinetics and anti-drug antibodies. The close collaboration between Celldex and Rockefeller University allowed for efficient program development and the ability to rapidly overcome challenges.
First Quarter 2020 Financial Highlights and 2020 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2020 were $53.7 million compared to $64.4 million as of December 31, 2019. The decrease was primarily driven by first quarter cash used in operating activities of $12.1 million, partially offset by $1.6 million in net proceeds from sales of common stock under the Cantor agreement. At March 31, 2020, Celldex had 17.7 million shares outstanding.

Revenues: Total revenue was $2.7 million in the first quarter of 2020 compared to $1.4 million for the comparable period in 2019. The increase in revenue was primarily due to the $1.8 million milestone payment from Rockefeller University related to our manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $11.7 million in the first quarter of 2020 compared to $11.2 million for the comparable period in 2019. The increase in R&D expenses was primarily due to higher laboratory supply and clinical trial costs, offset by lower contract research costs.

G&A Expenses: General and administrative (G&A) expenses were $3.7 million in the first quarter of 2020 compared to $4.9 million for the comparable period in 2019. The decrease in G&A expenses was primarily due to lower stock-based compensation expense and professional service costs.

Changes in Fair Value Remeasurement of Contingent Consideration: During the quarter ended March 31, 2020, the Company recorded a $0.2 million loss on fair value remeasurement of contingent consideration primarily due to the passage of time. During the quarter ended March 31, 2019, the Company recorded a $1.5 million loss on the fair value remeasurement of contingent consideration primarily due to changes in discount rates and the passage of time.

Net Loss: Net loss was $12.6 million, or ($0.73) per share, for the first quarter of 2020 compared to a net loss of $17.2 million, or ($1.40) per share, for the comparable period in 2019.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2020 are sufficient to meet estimated working capital requirements and fund planned operations into the second quarter of 2021. This guidance excludes anticipated proceeds from future sales of common stock under the Cantor agreement or other potential fundraising.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA. Erbitux is a registered trademark of Eli Lilly & Co.