On May 5, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2022 and reaffirmed its financial guidance for the year (Press release, Ultragenyx Pharmaceutical, MAY 5, 2022, View Source [SID1234613752]).

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"Product based revenue continues to grow driven by increased traction for Crysvita in Latin America," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In the clinical portfolio, we are now dosing patients in three of our four concurrent pivotal programs and are on track for updated data from the Phase 1/2 for Angelman syndrome mid-year."

First Quarter 2022 Financial Results

Net Revenues
For the first quarter of 2022, Ultragenyx reported $79.9 million in total revenue. Ultragenyx recognized $54.6 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $45.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $9.4 million. Total royalty revenue related to European Crysvita sales was $4.8 million. Dojolvi (triheptanoin) product sales in the first quarter of 2022 were $12.4 million. Mepsevii (vestronidase alfa) product sales for the first quarter of 2022 were $4.9 million.

Total revenue for the first quarter of 2022 also includes $3.2 million related to technical assistance following the successful completion of technology transfer activities with Daiichi Sankyo. This compares to total revenue in the first quarter of 2021, which includes $42.8 million related to the technology transfer services which were ongoing at the time.

Operating Expenses
Total operating expenses for the first quarter of 2022 were $216.6 million, including non-cash stock-based compensation of $29.4 million.

Net Loss
For the first quarter of 2022, Ultragenyx reported net loss of $152.3 million, or $2.19 per share basic and diluted, compared with a net loss for the first quarter of 2021 of $136.1 million, or $2.03 per share, basic and diluted. Net cash used in operations for the quarter ended March 31, 2022 was $117.5 million.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were $813.8 million as of March 31, 2022.

2022 Financial Guidance

The company continues to expect 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.

First Quarter 2022 Revenue and Selected Financial Data Tables

Recent Updates

Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): European marketing authorization transitioned to Ultragenyx, reimbursement processes initiated
The European Commission has completed transfer of the marketing authorization of Evkeeza from Regeneron. Ultragenyx is preparing reimbursement dossiers for various national health authorities in Europe. Ultragenyx received rights to commercialize and distribute Evkeeza in countries outside the U.S. in January 2022.

UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing has been initiated for the Phase 2/3 Orbit study; Phase 2 study in children under age five planned for second half of 2022
Ultragenyx has begun dosing patients in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI ages five to <26 years. A dosing update on the Phase 2 portion of the Orbit study and transition to Phase 3 is expected in the second half of 2022.

In addition, Ultragenyx intends to initiate an additional study in children with OI under age 5 years in the second half of 2022 and will continue to evaluate adult patients who were previously treated in the ASTEROID study, a Phase 2b study conducted by our partner Mereo.

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 GlucoGene study dosing patients
Dosing and enrollment of the Phase 3 study of DTX401 is ongoing. The pivotal GlucoGene study has a 48-week primary efficacy analysis period and the company plans to enroll approximately 50 patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control.

UX701 for Wilson Disease: Cyprus2+ pivotal Phase 1/2/3 study dosing patients
The company is dosing patients in the Phase 1/2 stage of the seamless Phase 1/2/3 Cyprus2+ study of UX701. During the first stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary efficacy endpoints are change in 24-hour urinary copper concentration and percent reduction in standard of care medication by Week 52.

DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 eNH3ance study expected to initiate in mid-2022
Ultragenyx expects to initiate the Phase 3 eNH3ance study of DTX301 in patients with OTC in mid-2022. The 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

GTX-102 for Angelman Syndrome: Patients continue to be treated in the Phase 1/2 study in Canada and the U.K. and under a separate protocol in the U.S.
Dosing is ongoing in cohorts 4 and 5 of the Phase 1/2 study in the U.K. and Canada, as well as for four additional patients in the U.S. under a separate protocol. To date, no treatment-related serious adverse events or lower extremity weakness adverse events have been observed in these patients. An interim update on the safety and efficacy of GTX-102 is planned for mid-2022.

UX053 for Glycogen Storage Disease Type III (GSDIII) Debrancher Deficiency: Phase 1/2 study currently dosing patients; Preliminary data from first part of study and initiation of second part of study anticipated in second half of 2022
Ultragenyx has begun to dose patients in the two-part Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of UX053 in adults age 18 years and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. Preliminary data from the Part 1 single ascending dose phase of the study is expected in the second half of the year.

1: 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.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 5, 2022, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2022 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 7951356. The replay of the call will be available for one year.

On May 5, 2022 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases, reported first quarter 2022 financial results and provided a business update (Press release, NextCure, MAY 5, 2022, View Source [SID1234613819]).

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"In the first quarter, we continued to advance our pipeline of four product candidates towards key clinical milestones," said Michael Richman, NextCure’s President and Chief Executive Officer. "In the second half of 2022, we anticipate clinical trial updates for NC318, NC410, and NC762, as well as an IND filing for NC525. We also expect our quarter-end cash position of $201.3 million will fund our operations into the first quarter of 2024."

Expected Milestones

We believe we are on track to meet the following milestones during the second half of 2022:

NC318 Phase 2 update: fourth quarter of 2022 (Amended Phase 2 with patient selection and increased dosing).
NC318 anti-PD-1 combo initial data second half of 2022 (Yale University Investigator-Initiated trial).
NC410 Phase 1 update: second half of 2022.
NC762 Phase 1 initial data: second half of 2022.
NC525 Investigational New Drug Application (IND) filing: fourth quarter of 2022.
Financial Guidance

Based on its current research and development plans, NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditures into the first quarter of 2024.

Financial Results for First Quarter March 31, 2022

Cash, cash equivalents, and marketable securities, excluding restricted cash, as of March 31, 2022, were $201.3 million as compared with $219.6 million as of December 31, 2021. The decrease of $18.3 million primarily reflects cash used to fund operations.
Research and development expenses were $15.0 million for the quarter ended March 31, 2022 as compared to $12.4 million for the quarter ended March 31, 2021. The increase of $2.6 million was driven primarily by additional clinical and lab-related costs.
General and administrative expenses were $5.8 million for the quarter ended March 31, 2022 as compared with $4.9 million for the quarter ended March 31, 2021. The increase of $0.9 million was primarily related to additional personnel-related costs.
Net loss was $20.6 million for the quarter ended March 31, 2022 as compared with $16.5 million for the quarter ended March 31, 2021. The changes in net loss from the previous year’s quarter were primarily due to increased research and development expenses.

On May 4, 2022 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL inhibitors for severe unmet medical needs, reported an update on its business strategy (Press release, BerGenBio, MAY 4, 2022, View Source [SID1234613465]). BerGenBio will now focus on two key indications; 1st line non-small cell lung cancer (NSCLC) and COVID-19, which the Company believes offer the optimal path towards translating BerGenBio’s strong scientific foundation into significant value generation from marketed products to address unmet medical needs.

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The opportunity

BerGenBio has for many years pioneered research into AXL inhibition, with its lead development candidate bemcentinib showing clinical potential in oncology and infectious disease. With over 600 patients dosed (approx. 400 in oncology and approx. 200 in COVID-19), the Company has built a large dataset demonstrating biological and clinical activity while simultaneously defining dosing regimens to identify an appropriate balance of safety and efficacy.

The updated business strategy announced today builds on bemcentinib’s mode of action and data gathered from a broad clinical exploration to progress two distinct opportunities with the potential to significantly improve the lives of patients: NSCLC and COVID-19. Both indications show strong evidence of bemcentinib activity, with the advantage of accumulation in target organs, options for accelerated registration, compelling competitive advantages, and both represent high unmet medical needs.

Martin Olin, Chief Executive Officer of BerGenBio, commented: "BerGenBio has been at the forefront of understanding of AXL biology and, having pioneered this area, we remain confident that selective AXL inhibition holds significant potential as a transformative treatment modality for several serious diseases. A rapidly evolving treatment landscape, with improved standards of care in areas such as oncology requires BerGenBio to be nimble and identify specific opportunities where we can address unmet needs in a competitive manner.

With this in mind we believe that by introducing a laser focus and rightsizing the organization on two key areas where we see our pipeline has the greatest impact, we are efficiently advancing BerGenBio’s potential; building on our strong scientific foundation to deliver new drugs to market resulting in better outcomes for patients and the generation of significant value for our shareholders."

NSCLC

Despite advances in treatment, lung cancer remains the leading cause of cancer-related deaths throughout the world. Approximately 85% of lung cancers are classified as NSCLC. BerGenBio is targeting 1st Line STK11 mutated Non-Squamous NSCLC patients, an extremely large patient population with very limited response to the standard of care treatments. STK11 is an important tumor suppressor gene reported to confer immunotherapy resistance in NSCLC and is present in up to 20% of NSCLC patients.

Pre-clinical and clinical studies have suggested a mechanism by which bemcentinib may restore response to immune checkpoint inhibitor therapy and enable the avoidance of chemoresistance in NSCLC patients harboring STK11 mutations, thus potentially offering a treatment option to those patients who respond poorly to existing therapies. Data from the subset of STK11mutated patients treated in the Company’s BGB008 study in 2nd line NSCLC also provides early indications of efficacy in this biomarker driven patient population.

The FDA has recognized that STK11 is currently a "non-actionable" mutation – one that confers poor outcome and has no specific therapeutic approaches today and have granted BerGenBio a Fast Track Designation for bemcentinib in this setting. To date, bemcentinib is to the Company’s knowledge the only selective AXL inhibitor in development for patients with STK11 mutation. BerGenBio has a strong proprietary position for treatment of this population and believes there may be a potential for an accelerated approval pathway in this patient sub-set.

COVID-19

Despite the success of vaccines, there is still a large number of hospitalized patients that remain in need of improved therapeutic options for COVID-19. Research into bemcentinib’s potential in hospitalized COVID-19 patients began in 2020, in response to the emergence of the pandemic, and based on the Company’s understanding of AXL’s role in mediating aggressive diseases.

Recently, BerGenBio announced results from the Phase II sub-protocol of the platform ACCORD2 study, which met its primary and key secondary endpoints, with demonstrable efficacy in patients on top of current standard-of-care treatments including remdesivir and corticosteroids. Further, bemcentinib has been selected to be studied under the EUSolidAct platform trial through a sub-protocol enrolling 500 patients across European sites. Given the ongoing need for new treatment options for hospitalized COVID-19 patients, the novel mechanism of action of bemcentinib (independent of the spike protein), along with potential to confirm the ACCORD2 data in the EUSolidAct trial, the Company believes that this could warrant Emergency Use Authorizations based on precedents.

The Company believes that the unique mechanism of action and properties of bemcentinib positions it well as a novel treatment modality within severe respiratory infections beyond COVID-19.

With a focused strategy and rightsized organization BerGenBio plans to unlock significant potential value related to the two indications selected and define the path to market.

On May 4, 2022 Amphista Therapeutics, a global leader in the discovery and development of next generation targeted protein degradation (TPD) therapeutics, reported a strategic collaboration and license agreement with Bristol Myers Squibb (Press release, Amphista Therapeutics, MAY 4, 2022, View Source [SID1234613481]).

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Under the terms of the agreement, Bristol Myers Squibb and Amphista will work collaboratively to discover and develop small molecule protein degraders. Bristol Myers Squibb will be granted a global exclusive license to the degraders developed and will be responsible for further development and commercialization activities. Amphista will receive a $30 million upfront payment, the potential for up to $1.25 billion in performance-based milestone payments and payment for a limited expansion of the collaboration, as well as royalties on global net sales of products. The closing of the transaction is subject to the parties obtaining regulatory clearances or approvals.

Nicola Thompson, CEO of Amphista, said, "Our collaboration with Bristol Myers Squibb is a powerful validation of our advances in TPD research and the capabilities of our Eclipsys next-generation TPD platform. Combining our expertise with Bristol Myers Squibb’s strong legacy and experience in the protein degradation space brings new promise to the potential of delivering more effective new treatment to patients seeking treatment options."

TPD therapies are designed to use physiological mechanisms to remove pathogenic protein from the body, offering the potential to access many disease targets previously considered "undruggable." Amphista’s technology is specifically designed to develop next generation TPD therapeutics based on advanced mechanistic insights and novel chemistry approaches that enable the development of novel protein degrading therapeutics.

"Bristol Myers Squibb continues to build its leadership and scientific expertise in the protein degradation space," said Rupert Vessey, M.A., B.M., B.Ch., FRCP, D.Phil., Executive Vice President, Research & Early Development, Bristol Myers Squibb. "We look forward to collaborating with Amphista and using its TPD platform to potentially develop new targeted protein degradation therapies."

On May 4, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported that the company will hold a conference call and live webcast on Wednesday, May 11, 2022 at 4:30 p.m. Eastern Time (ET) to review its first quarter 2022 financial results and provide a business update (Press release, Ascendis Pharma, MAY 4, 2022, View Source [SID1234613513]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Conference Call Details

A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.