On March 12, 2020 Sutro Biopharma, Inc. (NASDAQ: STRO), a clinical-stage drug discovery, development and manufacturing company focused on the application of precise protein engineering and rational design to create next-generation oncology therapeutics, reported that Merck, known as MSD outside the U.S. and Canada, has extended the research term of the collaboration’s first cytokine-derivative program by one year, which includes a payment to Sutro of an undisclosed amount (Press release, Sutro Biopharma, MAR 12, 2020, View Source [SID1234555455]). Sutro’s collaboration with Merck was announced in July 2018 to develop therapeutics for cancer and autoimmune disorders.

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"At Sutro, our proprietary and integrated cell-free protein synthesis and site-specific conjugation platform XpressCF+ enables us to design and develop next-generation targeted product candidates by innovating beyond the constraints of cell-based systems," said Sutro CEO, Bill Newell. "The advancement of our novel cytokine-derivative product candidate towards IND-enabling studies marks another important step forward in our collective goal of improving the outcomes for cancer patients."

"In all three of our current collaborations, and in three distinct therapeutic protein formats, we have progressed product candidates either into clinical development or to the late stages of preclinical development," said Sutro Chief Scientific Officer, Trevor Hallam, PhD. "These successes with Merck and with each of our other collaborators demonstrate the power and agility of our rapid and precise engineering platform."

On March 12, 2020 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the full year ended December 31, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 12, 2020, View Source [SID1234555484]).

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"The year is off to a strong start with the initiation of the Phase 1 trial of SRK-181 in immuno-oncology and the completion of enrollment in the SRK-015 TOPAZ Phase 2 trial in SMA," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "We now have two product candidates in the clinic with data expected from both programs later this year that will offer initial insights on their potential to treat patients with SMA and solid tumors, respectively. Scholar Rock is well-positioned to continue to execute and build momentum across its portfolio of product candidates."

Company Highlights and Upcoming Milestones

SRK-015 Program for Spinal Muscular Atrophy (SMA):

Published Structural Insights into the Mechanism by which SRK-015 Targets the Precursor or Latent Form of the Myostatin Growth Factor. In February 2020, the Journal of Biological Chemistry(1) published "Structural basis for specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015" detailing the integrated structural and biochemical approaches used to elucidate the unique molecular mechanism of SRK-015, a highly specific inhibitor of the activation of latent myostatin. The publication offers the first structural insights into the approach of targeting precursor forms to modulate growth factor activation. This approach could be applied to the discovery of specific modulators across members of the TGFβ superfamily or, even more broadly, to other signaling proteins that require precursor processing for activation.

On Track to Report Interim Safety and Efficacy Data from TOPAZ Phase 2 Trial in Mid-2020. In January 2020, Scholar Rock announced the completion of enrollment in the TOPAZ proof-of-concept trial for SRK-015 with a total of 58 patients with Type 2 or Type 3 SMA enrolled across 19 sites in the U.S. and Europe. Scholar Rock plans to report interim safety and efficacy results for all patients enrolled across the three cohorts of the trial following six months of treatment exposure in mid-2020. Top-line data for the full 12-month treatment period are expected beginning in the fourth quarter of 2020 and into the first quarter of 2021.

Presented Preliminary Data Highlighting Patient Demographics and Baseline Characteristics from the TOPAZ Trial at SMA Europe 2nd International Scientific Congress. In February 2020, Scholar Rock presented two posters at the SMA Europe 2nd International Scientific Congress highlighting key preclinical and Phase 1 data, in addition to preliminary pharmacokinetic (PK)/pharmacodynamic (PD) and baseline characteristics from the TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA. Preliminary demographic and baseline characteristic data for patients in the TOPAZ trial are in-line with previously published studies in SMA, including the evaluation of patients treated with nusinersen.

Announced Preliminary Pharmacokinetic and Pharmacodynamic Results from TOPAZ Trial. In November 2019, Scholar Rock announced results from a planned preliminary PK/PD analysis of 29 patients across all three cohorts of the TOPAZ trial. The analysis showed dose-proportional drug exposure and dose-dependent increases of up to 100-fold in serum latent myostatin levels following treatment with SRK-015. These biomarker results confirmed the presence of latent myostatin in patients with SMA and provides the first evidence of successful pharmacologic engagement of a latent growth factor in a human disease setting.

Identification of Second Indication for SRK-015 Planned for 2020. Scholar Rock continues to evaluate multiple potential opportunities beyond SMA, for which the selective inhibition of the activation of myostatin with SRK-015 may offer therapeutic benefit.
SRK-181 Program for Immuno-Oncology:

Initial Clinical Data from SRK-181 Phase 1 Dose Escalation and Dose Expansion Trial in Solid Tumors Expected in the Second Half of 2020. In the first quarter of 2020, Scholar Rock initiated a Phase 1 proof-of-concept trial for SRK-181 in patients with locally advanced or metastatic solid tumors. The two-part trial will consist of a dose escalation portion (Part A) for SRK-181 as both a single-agent and in combination with an approved anti-PD-(L)1 antibody, followed by a dose expansion portion (Part B) evaluating SRK-181 in combination with an approved anti-PD-(L)1 antibody in multiple tumor-specific cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Key objectives of the study include evaluating the safety and pharmacokinetics of SRK-181 and the efficacy of SRK-181 in combination with anti-PD-(L)1 therapy in the treatment of solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Initial clinical results, including biomarker data, from Part A of the Phase 1 trial are expected in the second half of 2020 with clinical response and safety data from Part B anticipated throughout 2021.
RGMc Program for Iron-Restricted Anemias:

Nomination of a Product Candidate from the RGMc Program Planned in 2020. Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody as its third product candidate in 2020. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Corporate Highlight:

Achieved First Milestone of $25 Million in Strategic Fibrosis Collaboration with Gilead Sciences. In December 2019, Scholar Rock achieved a $25 million preclinical milestone under the strategic fibrosis-focused collaboration with Gilead Sciences, Inc. for the successful demonstration of efficacy in preclinical in vivo proof-of-concept studies. This initial milestone advances the collaboration to develop potent and selective inhibitors of latent TGFβ activation for the potential treatment of patients suffering from fibrotic diseases.
Full Year 2019 Financial Results

For the year ended December 31, 2019, net loss was $51.0 million or $1.85 per share compared to a net loss of $49.3 million or $3.15 per share for the year ended December 31, 2018.

Revenue was $20.5 million for the year ended December 31, 2019 and was related to the Gilead Collaboration Agreement that was executed in December 2018.

Research and development expense was $54.2 million for the year ended December 31, 2019 compared to $36.3 million for the year ended December 31, 2018. The increase year-over-year primarily reflects preclinical and manufacturing costs for SRK-181 and higher personnel-related costs, slightly offset by lower manufacturing costs for SRK-015 and early development costs.

General and administrative expense was $20.8 million for the year ended December 31, 2019 compared to $14.4 million for the year ended December 31, 2018. The increase year-over-year was primarily attributable to increased headcount and professional services.
As of December 31, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $157.4 million, compared to $175.6 million as of December 31, 2018. A $25 million payment was received in January 2020 from Gilead for the achievement of the preclinical milestone under the strategic fibrosis-focused collaboration.

Dagbay, K., Treece, E., Streich Jr, F., Carven, G., et al. Structural basis for specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015, Journal of Biological Chemistry, View Source

On March 12, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the fourth quarter and year ended December 31, 2019 and provided a corporate update (Press release, Regulus, MAR 12, 2020, View Source [SID1234555516]).

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"We have made significant progress over the past few months, including receiving notification from FDA of their decision to lift the partial clinical hold on our Phase 1 multiple ascending dose clinical study for RGLS4326, enabling the initiation of dosing of the second cohort of that study for the treatment of autosomal dominant polycystic kidney disease this past February," said Jay Hagan, CEO of Regulus. "This important milestone, coupled with the closing of the second tranche of financing, enables us to advance the program toward key data read-outs."

Corporate Highlights

Closed $26 Million Second Tranche of Private Financing: In December 2019, following the announcement of the Company’s plan to recommence the Phase 1 Multiple Ascending Dose ("MAD") clinical study of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease ("ADPKD") in the first quarter of 2020, the Company completed a second and final closing under the May 2019 securities purchase agreement, pursuant to which the Company sold and issued 3,288,390 shares of non-voting Class A-2 convertible preferred stock, in lieu of shares of common stock, at a price of $6.66 per share, and accompanying warrants to purchase an aggregate of 32,883,900 shares of common stock at a price of $0.125 for each share of common stock underlying such warrants. Each share of the non-voting Class A-2 convertible preferred stock is convertible into 10 shares of common stock, subject to certain beneficial ownership conversion limitations. The warrants are exercisable for a period of five years following the date of issuance and have an exercise price of $0.666 per share, pursuant to proportional adjustments in the event of stock splits or combinations or similar events. Together with the first tranche, which closed in May 2019, the Company raised a total of $42.7 million from the private financing, which the Company expects will provide cash resources to fund planned activities into mid-2021.

Program Highlights

Initiated Dosing of the Second Cohort in RGLS4326 Phase 1 for ADPKD: In February 2020, the Company initiated dosing of the second cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is also planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity. The re-initiation of the MAD study was allowed following a satisfactory complete response provided to FDA in November 2019 to address the partial clinical hold placed on the clinical program in July 2019. A partial clinical hold on studies of RGLS4326 dosed for extended durations remains in effect until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

Financial Results

Cash Position: As of December 31, 2019, Regulus had $34.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $2.1 million and $12.3 million for the quarter and year ended December 31, 2019, respectively, compared to $5.3 million and $34.0 million for the same periods in 2018. The decreases were largely driven by decreases in external development expenses, primarily attributable to the pause of the RGLS4326 MAD study in the third quarter of 2018 and transfer of the RG-012 program to Sanofi beginning in the fourth quarter of 2018. Additionally, the decreases were driven by reductions in personnel and internal expenses, primarily attributable to a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million and $11.3 million for the quarter and year ended December 31, 2019, respectively, compared to $2.7 million and $12.9 million for the same periods in 2018. The decreases were mostly driven by a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million and $6.8 million for the quarter and year ended December 31, 2019, respectively, compared to less than $0.1 million and $0.1 million for the same periods in 2018. The increase for the year ended December 31, 2019 was attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $4.9 million and $18.6 million for the quarter and year ended December 31, 2019, respectively, compared to a net loss of $8.6 million and $48.7 million for the same periods in 2018. Basic and diluted net loss per share was $0.23 and $1.08 for the quarter and year ended December 31, 2019, respectively, compared to $0.98 and $5.59 for the same periods in 2018.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration.

On March 12, 2020 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the European Commission has approved Venclyxto (venetoclax) in combination with Gazyvaro (obinutuzumab) for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL) (Press release, Hoffmann-La Roche, MAR 12, 2020, View Source [SID1234555456]).

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"Venclyxto plus Gazyvaro is the first fixed-duration, chemotherapy-free treatment option that has been shown to help patients with untreated chronic lymphocytic leukaemia live longer without their disease progressing," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. "This is an important step forward for adults with this disease in the EU, who will now have an effective treatment option that enables them to end treatment after one year."

The approval is based on results from the primary analysis of the pivotal phase III CLL14 study, which evaluated the combination of 12-month, fixed-duration Venclyxto plus Gazyvaro compared to Gazyvaro plus chlorambucil in adults with previously untreated CLL who had co-existing medical conditions. Results from the primary analysis showed that the combination of Venclyxto plus Gazyvaro led to a 65% reduction in the risk of disease worsening or death (progression-free survival [PFS], as assessed by investigators) compared to Gazyvaro plus chlorambucil, a current standard-of-care for CLL (HR=0.35; 95% CI 0.23-0.53; p<0.0001). When PFS was assessed by an independent review committee, this finding was confirmed. Venclyxto plus Gazyvaro also showed higher response rates (ORR), doubled the complete response rates (CR/CRi) and demonstrated higher rates of minimal residual disease (MRD)-negativity, meaning that no cancer can be detected using a specific and highly sensitive test. In peripheral blood, MRD-negativity rates were 76% for Venclyxto plus Gazyvaro versus 35% for Gazyvaro plus chlorambucil, and in bone marrow MRD-negativity rates were 57% versus 17%, respectively. Results from an updated efficacy analysis (with a median follow-up of 40 months) were consistent with the primary data, and showed that the combination reduced the risk of disease worsening or death by 69% compared to Gazyvaro plus chlorambucil (HR=0.31; 95% CI 0.22-0.44). Safety for Venclyxto plus Gazyvaro appeared consistent with the known safety profile of the individual medicines, and no new safety signals were identified with the combination. The most commonly reported adverse events in people treated with Venclyxto plus Gazyvaro were low white blood cell count (neutropenia), diarrhoea and upper respiratory tract infection.

Results from the CLL14 study further demonstrate Venclyxto’s potential across multiple lines (in the R/R and the frontline settings) in CLL. Previously, the European Commission has approved Venclyxto in combination with MabThera (rituximab) for the treatment of adults with CLL who have received at least one prior therapy, and as a monotherapy for the treatment of CLL in the presence or absence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor. The US Food and Drug Administration (FDA) approved Venclexta in combination with Gazyva for the treatment of people with previously untreated CLL with co-existing medical conditions in May 2019. This followed rapid review and approval of the supplemental New Drug Application under the FDA’s Real-Time Oncology Review (RTOR) and Assessment Aid pilot programmes. Additional submissions of the data to health authorities around the world are still ongoing, with the goal of bringing this new treatment option to more patients as soon as possible.

Venclexta/Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US, under the brand name Venclexta, and commercialised by AbbVie outside of the US.

About the CLL14 study1
CLL14 (NCT02242942) is a randomised phase III study evaluating the combination of fixed-duration Venclyxto plus Gazyvaro compared to Gazyvaro plus chlorambucil in adult patients with previously untreated chronic lymphocytic leukaemia (CLL) and co-existing medical conditions. 432 patients with previously untreated CLL were randomly assigned to receive either a 12-month duration of Venclyxto alongside six-month duration of Gazyvaro (Arm A) or six-month duration of Gazyvaro alongside 12-month duration of chlorambucil (Arm B). Arm A started with an initial dosing of Gazyvaro followed by a five-week Venclyxto dose ramp-up to help reduce the risk of tumour lysis syndrome. The primary endpoint of the study is investigator-assessed (INV) progression-free survival (PFS). Secondary endpoints include PFS assessed by independent review committee (IRC), minimal residual disease (MRD) status, overall response rate (ORR), complete response rate (CR, with or without complete blood count recovery), overall survival, duration of response, event-free survival, time to next CLL treatment, and safety. MRD-negativity means no cancer can be detected using a specific, highly sensitive test, and was defined as less than one cancer cell in 10,000 leukocytes. The CLL14 study is being conducted in cooperation with the German CLL Study Group, headed by Michael Hallek, MD, University of Cologne.

CLL14 study primary analysis results2
Treatment arm Venclyxto + Gazyvaro
(n=216) Gazyvaro + chlorambucil
(n=216)
INV-assessed PFS (primary endpoint)
Median PFS Not reached Not reached
Hazard ratio 0.35 (95% CI 0.23-0.53), p<0.0001
Additional efficacy results (secondary endpoints)
IRC-assessed PFSa
Median PFS Not reached Not reached
Hazard ratio 0.33 (95% CI 0.22-0.51), p<0.0001
Response rates
ORR 85% 71%
CR + complete remission with incomplete bone marrow recovery (CRi) 50% 23%
MRDb
MRD-negative, bone marrow, 57% 17%
p-value p<0.0001
MRD-negative, peripheral blood, 76% 35%
p-value p<0.0001
Safety
Adverse events The most common adverse reactions (≥20%) with Venclyxto plus Gazyvaro were low white blood cell count (neutropenia), diarrhoea and upper respiratory tract infection. The most common serious adverse events in people treated with Venclyxto plus Gazyvaro were febrile neutropenia, pneumonia, infusion-related infection and pyrexia.
a Data at median follow-up of 28 months
b Data at three months following end of combination treatment, in the intention-to-treat population

About Venclyxto (venetoclax)
Venclyxto is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumours, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclyxto blocks the BCL-2 protein and works to restore the process of apoptosis.

Venclexta/Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US, under the brand name Venclexta, and commercialised by AbbVie outside of the US. Together, the companies are committed to research with Venclexta/Venclyxto, which is currently being studied in clinical trials across several types of blood and other cancers.

In the US, Venclexta has been granted five Breakthrough Therapy Designations by the US Food and Drug Administration: one for previously untreated chronic lymphocytic leukaemia (CLL), two for relapsed or refractory CLL and two for previously untreated acute myeloid leukaemia.

About Gazyvaro (obinutuzumab)
Gazyvaro is an engineered monoclonal antibody designed to attach to CD20, a protein expressed on certain B-cells, but not on stem cells or plasma cells. Gazyvaro is designed to attack and destroy targeted B-cells both directly and together with the body’s immune system. Gazyvaro is marketed as Gazyva in the US.

Gazyva/Gazyvaro is currently approved in more than 90 countries in combination with chlorambucil for people with previously untreated chronic lymphocytic leukaemia, in more than 80 countries in combination with bendamustine for people with certain types of previously treated follicular lymphoma and in more than 70 countries in combination with chemotherapy for previously untreated follicular lymphoma.

Additional combination studies investigating Gazyvaro with other approved or investigational medicines, including cancer immunotherapies and small molecule inhibitors, are underway across a range of blood cancers.

About chronic lymphocytic leukaemia
Chronic lymphocytic leukaemia (CLL) is the most common type of leukaemia in the Western world.3 CLL mainly affects men and the median age at diagnosis is about 70 years.4 Worldwide, the incidence of all leukaemias is estimated to be over 400,000, with an incidence of over 100,000 in Europe.5 CLL is estimated to affect around one-third of all people newly diagnosed with leukaemia.3

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for over 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera/Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), Polivy (polatuzumab vedotin), Venclexta/Venclyxto (venetoclax) in collaboration with AbbVie, and Hemlibra (emicizumab). Our pipeline of investigational haematology medicines includes idasanutlin, a small molecule which inhibits the interaction of MDM2 with p53; T-cell engaging bispecific antibodies targeting both CD20 and CD3, Tecentriq (atezolizumab), a monoclonal antibody designed to bind with PD-L1; and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.

On March 12, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for 2019 (Press release, Y-mAbs Therapeutics, MAR 12, 2020, View Source [SID1234555485]).

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"We are very pleased with our 2019 results, highlighted by notable progress in the preparation of our BLAs for naxitamab and omburtamab, as well as commercial ramp-up for the potential launch of both compounds. We start 2020 with $207 million in cash, and are excited to enter what we believe will be a truly transformational year for Y-mAbs," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "During 2019, we have worked hard to make sure that naxitamab and omburtamab advance towards BLA submissions. We submitted the first portion of the rolling BLA for naxitamab in November 2019, and expect to complete the submission later this month. For omburtamab, we expect the rolling BLA submission to be completed in May."

Fourth Quarter 2019 and Recent Corporate Developments

·After the close of the fourth quarter, on February 26, 2020, Y-mAbs announced a positive Pre-BLA meeting with FDA for omburtamab.

·On December 12, 2019, Y-mAbs announced that its GD2-GD3 Vaccine has been granted a Rare Pediatric Disease Designation by the FDA for the treatment of neuroblastoma.

·On December 11, 2019, Y-mAbs announced positive frontline data for naxitamab at the Company’s R&D event, which took place in New York City. Key opinion leaders discussed the current treatment landscape and unmet medical needs for high-risk neuroblastoma and other solid tumors.

·On December 5, 2019, the Company announced that the European Medicines Agency agreed to the Company’s proposed Pediatric Investigation Plan for omburtamab.

·On November 29, 2019, Y-mAbs announced that it had submitted to the FDA the first portion of its Biologics License Application for naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma under the FDA’s Rolling Review process.

·On November 15, 2019, Y-mAbs announced a clinical update on omburtamab for Desmoplastic Small Round Cell Tumor. The data was presented at the 2019 CTOS Annual Meeting in Tokyo, Japan.

·On November 1, 2019, Y-mAbs announced the pricing of a follow-on shelf public offering, resulting in gross proceeds to the Company of approximately $143.8 million.

·On October 28, 2019, Y-mAbs announced an update on omburtamab data, which was presented at the International Society of Pediatric Oncology conference.

·On October 25, 2019, Y-mAbs announced a naxitamab update, which was presented at the International Society of Pediatric Oncology conference.

Financial Results

Y-mAbs reported a net loss for the year ended December 31, 2019 of $81.0 million, or $2.30 per basic and diluted share, compared to a net loss of $43.3 million, or $1.50 per basic and diluted share, reported for the year ended December 31, 2018.

Operating Expenses

Research and Development

Research and development expenses were $63.5 million for the twelve months ended December 31, 2019, compared to $34.3 million for the twelve months ended December 31, 2018, an increase of $29.2 million. The increase in research and development expenses primarily reflects the following:

·$17.1 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;
·$5.4 million increase in outsourced research and supplies to support expanding development activities;
·$3.3 million increase in personnel costs; and
·$2.5 million increase in clinical trials expenses.

General and Administration

General and administrative expenses were $19.5 million for the twelve months ended December 31, 2019, compared to $9.0 million for the twelve months ended December 31, 2018, an increase of $10.5 million. Such increase in general and administrative expenses primarily reflects the following:

·$5.1 million increase in personnel costs; and
·$2.8 million increase in commercial infrastructure costs;

Cash and Cash Equivalents

The Company had approximately $207.1 million in cash and cash equivalents as of December 31, 2019, compared to $147.8 million as of December 31, 2018. The increase of $59.3 million was primarily attributable to the $134.7 million net proceeds from the secondary public offering, completed in November 2019, which was partially offset by the net loss of $81.0 million for the fiscal year 2019 due to the increased costs of operation as the Company prepares for its submission of rolling BLAs for naxitamab and omburtamab, build-up of the Company’s commercial infrastructure, and increased personnel costs.

Webcast and Conference Call

The Company will host a conference call today at 4:30 pm eastern time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the access code 13699294. A webcast will be available at: View Source