On May 4, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the first quarter of 2022, raised 2022 financial guidance and provided business updates (Press release, Jazz Pharmaceuticals, MAY 4, 2022, View Source [SID1234613574]).

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"We’re pleased to raise our top- and bottom-line guidance, driven by our continued execution and significant progress across commercial and R&D in the first quarter, which positions us well for the rest of the year and to achieve Vision 2025," said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals. "Our recent launches of Xywav, in both narcolepsy and idiopathic hypersomnia (IH), and Rylaze in acute lymphoblastic leukemia (ALL), continue to generate increased prescriber and patient adoption, and demonstrate our ability to deliver innovative new medicines to improve the lives of patients and their families. On the corporate development front, our three recent transactions are aligned with our broader strategy, allowing us to focus on our highest priorities, enhance our pipeline in areas of key interest in neuroscience and oncology and drive long-term shareholder value."

"We’ve had a highly productive start to 2022 with the submission of two Rylaze Supplemental Biologics License Applications, the first patient enrolled in our Phase 2 basket trial for Zepzelca and the first presentation of preclinical data for JZP815, an investigational, next-generation pan-RAF kinase inhibitor," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "I’m also excited about the addition of DSP-0187, a potent and highly selective oral orexin-2 receptor agonist, now called JZP441, further strengthening our leadership in sleep medicine, and WTX-613, a differentiated, conditionally activated interferon alpha (IFNα) INDUKINE molecule, now called JZP898, which has demonstrated anti-tumor activity in preclinical models. These recent transactions reinforce our commitment to enhancing our pipeline and leveraging our productive R&D engine to develop novel medicines for people with serious diseases."

Key Highlights
Business and Execution
Robust early launch momentum in first full quarter of Xywav for IH
Submitted a Rylaze Supplemental Biologics License Application (sBLA) for Monday/Wednesday/Friday (M/W/F) intramuscular (IM) dosing and an sBLA for intravenous (IV) administration; both are being reviewed under the Real-Time Oncology Review (RTOR) program
First patient enrolled in Zepzelca EMERGE-201 Phase 2 basket trial
Strengthened leadership in sleep medicine with addition of a potent, highly selective oral orexin-2 receptor agonist, JZP441 (DSP-0187)
Expanded oncology pipeline with JZP898 (WTX-613), a differentiated, conditionally activated IFNα INDUKINE molecule
Strategic divestiture of Sunosi allows increased investment and sharpens focus on highest strategic priorities
Financial
Growing and durable commercial franchises drove 1Q22 total revenues of $813.7 million; 34% increase compared to the same period in 2021
Raising top- and bottom-line guidance; 2022 total revenue guidance increased to $3.5 to $3.7 billion
Net leverage ratio of 3.9×1 as of March 31, 2022, demonstrating rapid deleveraging following the close of the GW Pharmaceuticals (GW) acquisition; on-track for target of less than 3.5x by the end of 2022
Substantial revenue diversification continues as newer products continue to grow and the Company optimizes its commercial portfolio

On a non-GAAP adjusted basis. Non-GAAP net leverage ratio is a non-GAAP financial measure. For further information, see "Non-GAAP Financial Measures."

Business Updates
Key Commercial Products
Oxybate (Xywav and Xyrem ):
Net product sales for the combined oxybate business increased 6% to $433.6 million in 1Q22 compared to the same period in 2021.
Average active oxybate patients on therapy was approximately 16,650 in 1Q22, an increase of approximately 6% compared to the same period in 2021.
Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution:
Xywav net product sales increased 147% to $186.1 million in 1Q22 compared to the same period in 2021.
There were approximately 7,800 active Xywav patients exiting 1Q22.
Xywav has broad patent protection to 2033.
Xywav for Narcolepsy:
There were approximately 7,050 narcolepsy patients taking Xywav exiting 1Q22.
The benefits of lowering sodium intake continues to resonate with patients and prescribers. In June 2021, U.S. Food and Drug Administration (FDA) recognized seven years of Orphan Drug Exclusivity (ODE), through July 2027, for Xywav and published its summary of clinical superiority findings stating that "Xywav is clinically superior to Xyrem by means of greater safety because Xywav provides a greatly reduced chronic sodium burden compared to Xyrem." Further, FDA stated that "the differences in the sodium content of the two products at the recommended doses will be clinically meaningful in reducing cardiovascular morbidity in a substantial proportion of patients for whom the drug is indicated."
Xywav for Idiopathic Hypersomnia (IH):
Positive early launch momentum with approximately 750 IH patients taking Xywav exiting 1Q22.
The Company launched Xywav for IH in November 2021, with initial launch efforts focused on the approximately 37,000 currently diagnosed patients in the U.S. who are actively seeking healthcare. Healthcare providers are excited to have a treatment option with positive and compelling clinical trial results that address IH and not just its symptoms.
FDA recognized ODE for IH in January 2022, extending regulatory exclusivity to August 2028.
Xyrem (sodium oxybate) oral solution:
Xyrem net product sales decreased 26% to $247.5 million in 1Q22 compared to the same period in 2021, reflecting the continued adoption of Xywav by patients with narcolepsy.
Epidiolex/ Epidyolex (cannabidiol):
Epidiolex/Epidyolex net product sales increased 6% to $157.9 million in 1Q22 compared to the same period in 2021, on a proforma basis.
Epidiolex/Epidyolex net product sales in 4Q21 were favorably impacted by approximately $18 million, due to a temporary increase in specialty pharmacy inventory levels at the end of 2021. The majority of this increase reversed in 1Q22, reducing 1Q22 revenues.
Excluding this impact, we saw double-digit percentage revenue growth in 1Q22 compared to 1Q21, and sequential growth in underlying demand, despite challenges posed by the Omicron variant.
Epidyolex is now commercially available and fully reimbursed in four of the five key European markets: United Kingdom, Germany, Italy and Spain, with an anticipated launch in France in 2022. The Company has made significant progress on its European rollout with launches in Spain, Italy and Switzerland in 3Q21 and Ireland and Norway in 1Q22.
The Company expects to initiate a Phase 3 pivotal trial of Epidiolex for Epilepsy with Myoclonic-Atonic Seizures (EMAS), the fourth target indication for Epidiolex, in 1H22.
The Company continues to strengthen the durability of Epidiolex. Patent US 11,207,292 is Orange Book listed and extends through 2039. This patent covers the composition of the botanically derived cannabidiol (CBD) preparation used in Epidiolex and the treatment of indicated disorders using that CBD preparation.
Zepzelca (lurbinectedin):
Zepzelca net product sales increased 9% to $59.3 million in 1Q22 compared to the same period in 2021.
The Company is pleased to have established Zepzelca as the treatment of choice in the second-line small cell lung cancer (SCLC) setting after only eighteen months on the market.
Zepzelca development program updates:
In March 2022, the first patient was enrolled in the EMERGE-201 Phase 2 basket trial evaluating Zepzelca as monotherapy in select relapsed/refractory solid tumors.
Jazz and collaborator F. Hoffmann-La Roche Ltd (Roche) have initiated a Phase 3 trial to evaluate first-line use of Zepzelca in combination with Tecentriq (atezolizumab), compared to Tecentriq alone, as maintenance therapy in patients with extensive-stage SCLC after induction chemotherapy. The first patient was enrolled in the trial in November 2021.
The Company’s partner, PharmaMar, initiated a confirmatory trial, LAGOON, in second-line SCLC in December 2021. If positive, this trial could confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following first-line treatment with a platinum-based regimen.
Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn):
Rylaze net product sales were $54.2 million in 1Q22.
The continued strong launch of Rylaze reflects the significant unmet patient need for a high-quality, reliable supply of Erwinia asparaginase for patients with ALL.
In January 2022, the Company completed the submission of an sBLA to FDA seeking approval for a M/W/F IM dosing schedule for Rylaze. In April 2022, the Company also completed the submission of an sBLA to FDA seeking approval for IV administration of Rylaze. Both submissions are being reviewed under the RTOR program.
The Company anticipates that data from the current development program will support regulatory filings in Europe in mid-2022, including IV administration, with potential for approval in 2023. The Company is also working with a partner to advance the program for potential submission, approval and launch in Japan.
Corporate Development
JZP441 (DSP-0187) Agreement:
On May 4, 2022, the Company and Sumitomo Pharma Co., Ltd. announced an exclusive license agreement for DSP-0187, now called JZP441, a potent, highly selective oral orexin-2 receptor agonist designed to activate orexin signaling.
Sumitomo Pharma initiated a Phase 1 clinical trial in Japan in November 2021 to evaluate safety, tolerability, and pharmacokinetics in healthy volunteers.
The collaboration will leverage the Company’s substantial experience and leadership in sleep disorders to advance this therapy with the potential to improve patient care.
Financial terms included a $50 million upfront payment to Sumitomo Pharma, and Sumitomo Pharma is eligible to receive development, regulatory and commercial milestone payments of up to $1.09 billion. Pending approval, Sumitomo Pharma is eligible to receive a tiered, low double-digit royalty on the Company’s future net sales of JZP441.
JZP898 (WTX-613) Agreement:
On April 7, 2022, the Company and Werewolf Therapeutics entered into a licensing agreement under which the Company acquired exclusive global development and commercialization rights to Werewolf’s investigational molecule, WTX-613, now called JZP898, a differentiated, conditionally activated IFNα INDUKINE molecule.
JZP898 is an engineered IFNα cytokine pro-drug that is activated specifically within the tumor microenvironment where it can stimulate IFNα receptors on cancer-fighting immune effector cells. The aim of JZP898 is to minimize the severe toxicities that have been observed with systemically active recombinant IFNα therapy and maximize clinical benefit when administered as monotherapy or in combination with other agents.
Jazz expects to file an Investigational New Drug (IND) application in the U.S. in 2023.
Financial terms included a $15 million upfront payment to Werewolf, and Werewolf is eligible to receive development, regulatory and commercial milestone payments of up to $1.26 billion. Pending approval, Werewolf is eligible to receive a tiered, mid-single-digit percentage royalty on the net sales.
Sunosi (solriamfetol) Strategic Divestiture:
On March 28, 2022, Jazz entered into a definitive agreement to divest Sunosi to Axsome Therapeutics.
The Company will receive an upfront payment of $53 million, a high single-digit royalty on Axsome’s U.S. net sales of Sunosi in current indications and a mid-single-digit royalty on Axsome’s U.S. net sales of Sunosi in future indications.
The Company and Axsome are committed to ensuring that patients receive uninterrupted access to Sunosi throughout the transition.
The companies expect the U.S. transaction to close in the second quarter of 2022 and the ex-U.S. transaction close to occur within 60 days following the close of the U.S. transaction.
Key Pipeline Highlights
Nabiximols:
There are currently three ongoing Phase 3 trials in multiple sclerosis (MS)-related spasticity. The Company anticipates data from its first Phase 3 trial, NCT04657666, in 2Q22; supportive findings may enable a New Drug Application submission to FDA in 2022.
Suvecaltamide (JZP385):
Suvecaltamide, a highly selective modulator of T-type calcium channels, is in clinical development for the treatment of essential tremor.
The Company initiated a Phase 2b trial in 4Q21 and announced that the first patient was enrolled in December 2021. Top-line data read-out is anticipated in 1H24.
JZP150:
JZP150, a selective fatty acid amide hydrolase, or FAAH, inhibitor, is in clinical development for the potential treatment of post-traumatic stress disorder (PTSD).
The Company initiated a Phase 2 trial in 4Q21 and announced that the first patient was enrolled in December 2021. Top-line data read-out is anticipated in late 2023.
The Company received Fast Track Designation for JZP150 development in PTSD from FDA in 4Q21, underscoring the significant unmet medical needs of patients.
JZP815:
JZP815 is an investigational, preclinical stage pan-RAF kinase inhibitor that targets specific components of the mitogen-activated protein kinase (MAPK) pathway, which when activated by oncogenic mutations, can be a frequent driver of human cancer.
The pan-RAF inhibitor program is part of a novel class of next-generation precision oncology therapies that has the potential to benefit cancer patients with high unmet needs in multiple different solid tumors.
The Company, together with our preclinical collaboration partner, Redx Pharma, presented its first preclinical data in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.
JZP815 inhibited tumor growth in several RAS- and BRAF-mutated solid tumor models, and demonstrated enhanced activity when combined with other MAPK pathway inhibitors.
The Company plans to submit an IND for JZP815 this year.
Other Products
Sunosi (solriamfetol):

Sunosi net product sales increased by 37% to $15.9 million in 1Q22 compared to the same period in 2021.
Vyxeos (daunorubicin and cytarabine) liposome for injection:

Vyxeos net product sales increased 2% to $33.8 million in 1Q22 compared to the same period in 2021.
Defitelio (defibrotide sodium) / defibrotide:

Defitelio/defibrotide net product sales of $49.5 million in 1Q22 were consistent with the same period in 2021.
Financial Highlights

Adjusted EPS in 1Q22 was impacted by $0.44 per share following the adoption of ASU 2020-06.

The Company adopted ASU No. 2020-06, "Debt—Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging—Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity", (ASU 2020-06) on January 1, 2022. Following adoption, diluted EPS must be calculated using the if-converted method which assumes full conversion of our Exchangeable Senior Notes.

GAAP net income in 1Q22 was $1.6 million, or $0.03 per diluted share, compared to $121.8 million, or $2.09 per diluted share, for 1Q22. Non-GAAP adjusted net income in 1Q22 was $261.9 million, or $3.73 per diluted share, compared to $228.8 million, or $3.92 per diluted share, for 1Q22. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Net product sales for Epidiolex/Epidyolex and Sativex are included from the acquisition of GW on May 5, 2021.

Total revenues increased 34% in 1Q22 compared to the same period in 2021.

Neuroscience net product sales in 1Q22 increased 45% to $612.1 million compared to the same period in 2021 primarily driven by Epidiolex/Epidyolex net product sales in the first quarter of 2022 of $157.9 million following the acquisition of GW. In 1Q22, oxybate net product sales increased 6% to $433.6 million.
Oncology net product sales in 1Q22 increased 10% to $196.8 million compared to the same period in 2021 primarily driven by Rylaze net product sales in 1Q22 of $54.2 million following product launch in July 2021, partially offset by Erwinaze/Erwinase net product sales in 1Q21 of $41.1 million.
Operating Expenses and Effective Tax Rate

Operating expenses increased over the prior year period primarily due to the following:

Cost of product sales increased in 1Q22 compared to the same period in 2021, on a GAAP and on a non-GAAP adjusted basis, due to increased net product sales as a result of the acquisition of GW. In addition, acquisition accounting inventory fair value step-up expense of $63.9 million in 1Q22 impacted GAAP cost of product sales.
Selling, general and administrative (SG&A) expenses increased in 1Q22 compared to the same period in 2021, on a GAAP and on a non-GAAP adjusted basis, primarily due to an increase in compensation-related expenses driven by higher headcount as a result of the acquisition of GW.
Research and development (R&D) expenses increased in 1Q22 compared to the same period in 2021, on a GAAP and on a non-GAAP adjusted basis, primarily due to the addition of costs related to clinical programs for Epidiolex, nabiximols and cannabinoids, an increase in costs related to JZP150 and suvecaltamide (JZP385) and an increase in compensation-related expenses due to higher headcount primarily driven by the acquisition of GW.
Cash Flow and Balance Sheet
As of March 31, 2022, cash and cash equivalents were $490.8 million, and the outstanding principal balance of the Company’s long-term debt was $6.2 billion compared to $6.4 billion as of December 31, 2021. In addition, the Company had undrawn borrowing capacity under a revolving credit facility of $500.0 million. For the three months ended March 31, 2022, the Company generated $209.0 million of cash from operations. In 1Q22 the Company repaid in full the $251.0 million remaining aggregate principal amount of the Euro Term Loan B.

Excludes $305-$340 million of amortization of acquisition-related inventory fair value step-up, $13-$15 million of share-based compensation expense and $2 million of transaction and integration related expenses relating to the acquisition of GW from estimated GAAP gross margin.

Excludes $148-$168 million of share-based compensation expense and $31-$41 million of transaction and integration related expenses relating to the acquisition of GW and $40-$50 million of costs related to the disposal of a business from estimated GAAP SG&A expenses.

Excludes $59-$67 million of share-based compensation expense and $2 million of transaction and integration related expenses relating to the acquisition of GW from estimated GAAP R&D expenses.

Excludes the income tax effect of adjustments between GAAP net income and non-GAAP adjusted net income.

Non-GAAP adjusted EPS guidance for 2022 reflects dilution of $2.05, at the midpoint, post adoption of ASU 2020-06. Diluted EPS calculations for 2022 include 9 million shares related to the assumed conversion of the Exchangeable Senior Notes and the associated interest expense add-back to net income of $29 million, on a GAAP basis, when dilutive, and $25 million on a non-GAAP basis, under the "if converted" method.

See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to non-GAAP Adjusted 2022 Net Income Guidance" at the end of this press release.

Conference Call Details
Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET (9:30 p.m. IST) to provide a business and financial update and discuss its 2022 first quarter results. The live webcast may be accessed from the Investors section of the Company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 7492554.

A replay of the conference call will be available through May 11, 2022 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 7492554. An archived version of the webcast will be available for at least one week in the Investors section of the Company’s website at www.jazzpharmaceuticals.com.

On May 4, 2022, Turning Point Therapeutics, Inc. (the "Company") reported that entered into a license agreement (the "LaNova License Agreement") with LaNova Medicines Limited ("LaNova") for an exclusive, royalty-bearing license to intellectual property related to LM-302, a clinical stage anti-Claudin18.2 antibody drug conjugate (the "Product"), on a worldwide basis excluding Greater China and South Korea (the "Company Territory") (Filing, 8-K, Turning Point Therapeutics, MAY 4, 2022, View Source [SID1234613640]). Under the LaNova License Agreement, the Company has the exclusive right to research, develop, use, register, offer for sale, import and otherwise commercialize the Product in the Company Territory and non-exclusive rights to manufacture the Product worldwide in support of activities in the Company Territory.

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Pursuant to the LaNova License Agreement, the Company will pay LaNova an upfront cash payment of $25.0 million and may be obligated to pay milestone payments, which include up to $195.0 million in development and regulatory milestones and up to $880.0 million in sales milestones, and tiered royalty payments based on percentages (ranging from the mid-single digits to the mid-teens) of net sales (subject to customary deductions).

Subject to specified exceptions, for a period of time, the Company has agreed that neither it nor its controlled affiliates or sublicensees will engage in any clinical development, use or commercialization of specified products that would compete with the Products in the Company Territory, and LaNova has agreed that neither it nor its affiliates, licensees and its sublicensees will conduct any clinical development, use or commercialization of specified products that would compete with the Products in the Company Territory, other than expressly permitted activities.

The LaNova License Agreement will continue in effect until expiration of the last royalty term for the Product in any country in the Company Territory, where the royalty term for a Product in a given country in the Company Territory continues until the later of (i) the date of the last-to-expire valid claim within the Company’s patent rights that covers the Product in such country; (ii) the expiry of the regulatory exclusivity for such Product in such country; or (iii) 10 years after the date of the first commercial sale of the Product in such country. Subject to the terms of the LaNova License Agreement, the Company may terminate the LaNova License Agreement for convenience by providing written notice to the Company, which termination will be effective following a prescribed notice period. In addition, either party may terminate the LaNova License Agreement for the other party’s uncured material breach of the LaNova License Agreement, with a customary notice and cure period, or for the other party’s insolvency. LaNova may also terminate the agreement if the Company is acquired by a third party and the acquired party is engaged in activities with competing products that are not divested or discontinued, upon notice of termination to the Company within a specific period following closing of such acquisition. In addition, LaNova may terminate the LaNova License Agreement under specified circumstances if the Company or certain other parties challenge LaNova’s patent rights. If the Company terminates the agreement for convenience, the Company will grant to LaNova a non-exclusive, worldwide license, which may be royalty-bearing in certain circumstances, to intellectual property owned by the Company that is necessary for and was used by the Company to commercialize the Product.

The Company is responsible for conducting the development and commercialization activities in the Company Territory related to the Products at its own expense. The Company and LaNova will collaborate on a global development plan under which both parties will conduct global clinical studies in their respective territories. The Company and LaNova will each be responsible for the costs allocated to them in accordance with the agreed budget under the global development plan. Both the Company and LaNova have the ability to conduct local studies outside of the global development plan at their own expense.

Both the Company and LaNova have the obligation to use commercially reasonable efforts to conduct development activities under the agreed-upon global development plan. The Company has the obligation to use commercially reasonable efforts to perform local studies independent of the global development plan and to obtain regulatory approvals for the Product in the Company Territory.

LaNova has an initial obligation to supply the Company with the Product. After a specified period, the Company will assume responsibility for supply of the Product.

As part of the LaNova License Agreement, the Company also obtained the right of first negotiation for an exclusive license to develop, use, manufacture and commercialize other products containing components of the Product. In addition, the Company has the option to collaborate with LaNova on up to three additional antibody drug conjugate programs, initiated or proposed by either the Company or LaNova.

The foregoing description of the material terms of the Agreement does not purport to be complete and is qualified in its entirety by reference to the complete text of the Agreement, a copy of which the Company intends to file, with confidential terms redacted, with the Securities and Exchange Commission as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022.

On May 4, 2022 Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, reported first quarter 2022 financial results and provided business updates (Press release, Seres Therapeutics, MAY 4, 2022, View Source [SID1234613474]).

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"Seres continued to make important progress toward bringing SER-109 to patients suffering with recurrent C. difficile infection (rCDI). Given the robust profile of SER-109 observed in our Phase 3 study, we expect the product to have a significant impact on rCDI, a serious disease with approximately 170,000 cases annually in the US. We look forward to meaningful additional clinical data, including in first recurrence CDI patients, from our SER-109 open label study later this quarter, supporting a mid-2022 completion of our BLA submission with the U.S. Food and Drug Administration (FDA)," said Eric Shaff, President and Chief Executive Officer at Seres. "We are also advancing our commercialization efforts alongside our collaborator, Aimmune Therapeutics, Inc., a Nestlé Health Science company, in anticipation of a potential SER-109 product launch in the first half of 2023."

"In tandem, we are excited about our earlier stage pipeline and continue to enroll our Phase 1b clinical study of SER-155 designed to evaluate safety, microbiome alterations, and the impact on infections and/or graft versus host disease (GvHD) in adult subjects who are undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Preclinical activities are also underway for additional Infection Protection development candidates, leveraging our prior compelling clinical data in this therapeutic category, for indications such as cancer neutropenia, solid organ transplant, and antimicrobial resistant infections more broadly," continued Mr. Shaff.

Program and Corporate Updates

SER-109 Phase 3 ECOSPOR III study in recurrent C. difficile infection: SER-109, an investigational oral, live microbiome therapeutic, achieved its primary endpoint of superiority to placebo in reducing CDI recurrence in patients with rCDI.

Seres has achieved target enrollment in an open-label study of SER-109 in patients with rCDI (ClinicalTrials.gov identifier: NCT03183141), which also enrolled over 25% of patients with a single recurrence of rCDI, to expand the SER-109 database in support of the planned BLA filing. Study results are anticipated in Q2 2022. The FDA recently endorsed Seres’ plans regarding a rolling BLA submission for SER-109. Seres intends to begin the BLA rolling submission in the coming weeks, and to complete the BLA submission, including data from the open-label study, in mid-2022.

Seres has an active SER-109 expanded access program at various sites across the U.S. The program is designed to enable eligible adults with rCDI to obtain access to SER-109 prior to a potential FDA product approval.

The Company continues to prepare for a successful product launch with Aimmune Therapeutics, Inc., a Nestlé Health Science company, who will be leading commercialization efforts. The Company believes that a substantial commercial opportunity exists for SER-109. The cost of a patient with recurrence of CDI has been estimated to result in approximately $34,000 in annual direct healthcare expenses; this does not include the substantial indirect costs associated with this disease. There are approximately 170,000 cases of rCDI annually in the U.S. and CDI results in over 20,000 deaths.

Seres continues to execute pre-commercialization activities in collaboration with Aimmune Therapeutics, including market education and data dissemination to the medical community. Medical Affairs led engagement of key opinion leaders has increased following the publication of the SER-109 Phase 3 data in the New England Journal of Medicine. In addition, activities have been initiated to engage payers in accordance with FDA guidance on pre-approval information exchange.

In January 2022, the New England Journal of Medicine (NEJM) published data from the SER-109 Phase 3 ECOSPOR III study evaluating SER-109 for the treatment of rCDI. The publication highlights key results including that SER-109 was superior to placebo in reducing CDI recurrence, with 88% of SER-109 patients achieving a sustained clinical response compared to 60% on placebo. SER-109 was well tolerated, with a side effect profile comparable to placebo and no serious drug-related adverse events observed.

SER-155 Phase 1b clinical study activities: In December 2021, Seres announced the enrollment of the first patient in the Company’s Phase 1b clinical study of SER-155 designed to evaluate safety, microbiome alterations, and the impact on infections and/or GvHD associated with SER-155 in adult subjects who are undergoing allo-HSCT. In April 2022, the SER-155 Data and Safety Monitoring Committee met as part of a planned data review, and approved a recommendation to continue with enrollment in Cohort 1 based on an evaluation of available safety data. The study is being conducted with Memorial Sloan Kettering Cancer Center and the University of Chicago with additional sites expected to be added soon.

SER-155 is an investigational oral, rationally designed, cultivated microbiome therapeutic designed to reduce the incidence of gastrointestinal infections, bloodstream infections, and GvHD in patients receiving allo-HSCT. SER-155 is a consortium of bacterial species selected using Seres’ reverse translation discovery and development platforms. The design incorporates microbiome biomarker data from human clinical data and nonclinical human cell-based assays and in vivo disease models. The SER-155 composition aims to decrease the colonization and translocation of antibiotic-resistant bacteria in the gastrointestinal tract to decrease the incidence of bloodstream infections and additionally to modulate host immune responses to decrease GvHD. In addition to SER-109, SER-155 represents Seres’ second active development program in its Infection Protection franchise.

Infection Protection investor event: In January 2022, Seres held a webcast event that highlighted development of microbiome therapeutics as a novel approach for Infection Protection in medically compromised individuals. Building upon SER-109, and in addition to SER-155, Seres is also evaluating additional preclinical stage programs in indications such as cancer neutropenia, solid organ transplant, and antimicrobial resistant infections more broadly.

Ulcerative colitis (UC) research: The Company previously reported clinical, microbiome and metabolomic data from the SER-287 Phase 2b study and the first cohort of its SER-301 Phase 1b study. Available data suggest that there may be an opportunity to utilize biomarker-based patient selection and stratification for future studies. Research activities remain ongoing to inform potential further development activities.

Financial Results

Seres reported a net loss of $56.6 million for the first quarter of 2022, as compared with a net loss of $35.5 million for the same period in 2021.

Research and development expenses for the first quarter of 2022 were $39.6 million, compared with $29.3 million for the same period in 2021. The research and development expenses were primarily related to Seres’ late-stage SER-109 clinical development program and manufacturing costs, as well as personnel expenses.

General and administrative expenses for the first quarter of 2022 were $18.6 million, compared with $11.7 million for the same period in 2021. General and administrative expenses were primarily related to personnel expenses, professional fees, including SER-109 commercial readiness and pre-launch expenses, and facility costs.

Seres ended the first quarter of 2022 with approximately $248.0 million in cash, cash equivalents and investments as compared with $291.2 million at the end of 2021.

Conference Call Information

Seres’ management will host a conference call today, May 4, 2022, at 8:30 a.m. ET. To access the conference call, please dial 877-270-2148 (domestic) or 412-902-6510 (international) and reference the Seres Therapeutics conference call. To join the live webcast, please visit the "Investors and News" section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

On May 4, 2022 Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo for patients with solid and hematologic malignancies, reported that it will have an oral presentation at the International Society for Cell and Gene Therapy (ISCT) Annual Meeting 2022, to be held May 4-7, 2022 in San Francisco, California (Press release, Umoja Biopharma, MAY 4, 2022, View Source [SID1234613506]).

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On Friday, May 6th, Dave Vereide, Ph.D., Senior Scientist at Umoja Biopharma, will give an oral presentation titled "A synthetic cytokine receptor platform for producing cytotoxic innate lymphocytes as ‘off-the-shelf’ cancer therapeutics." The presentation will highlight Umoja’s rapamycin-activated cytokine receptor (RACR) platform which seeks to address current challenges in the cellular therapy space. Current approaches for differentiating induced pluripotent stem cells (iPSCs) into therapeutic immune cell types often requires expensive exogenous gamma-chain cytokines for expansion and survival, and often, the presence of feeder cells. The RACR platform permits the production of immune effector cells, termed RACR-induced cytotoxic innate lymphocytes (iCILs), in the absence of exogenous cytokines and feeder cells by genetically modifying iPSC-derived progenitor cells to express the RACR synthetic cytokine receptor. A synthetic small molecule ligand then binds RACR to drive the differentiation and expansion of the iCILs. RACR technology can be employed in both a manufacturing setting to generate iCILs and in vivo to enable the selective expansion and survival of iCILs.

"iPSCs are an excellent source of renewable, modifiable, and scalable material for cell therapy manufacturing, yet several limitations exist with the current manufacturing processes for differentiating iPSCs into therapeutic cell types, including the need for exogenous growth factors and feeder cells." said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. "At Umoja we are excited about our RACR-iCIL iPSC platform and its capacity to generate immune effector cells in the absence of these limitations. Due to the selective growth advantage provided by our RACR platform, a highly pure iCIL population can be produced during manufacturing. We are evaluating iCILs in pre-clinical studies to assess their in vivo anti-tumor activity, and are optimistic about their potential for development as "off-the-shelf" cell therapies for diverse hematologic malignancies and solid tumors."

Presentation details:

Presentation Title: A synthetic cytokine receptor platform for producing cytotoxic innate lymphocytes as ‘off-the-shelf’ cancer therapeutics
Presenting Author: Dave Vereide, Ph.D., Senior Scientist at Umoja Biopharma
Abstract Number: 44
Oral Presentation Session: Gene Editing/Gene Therapies
Oral Presentation Date, Time: Friday May 6, 2022; 4:45-5:45 p.m. PT
Poster Presentation Session: Poster Networking Reception 2
Poster Presentation Date, Time: Thursday May 5, 2022; 5:45-7:15 p.m. PT

On May 4, 2022 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, reported financial results for the quarter ended March 31, 2022 (Press release, Adaptive Biotechnologies, MAY 4, 2022, View Source [SID1234613522]).

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"We started the year strong and completed a strategic reorganization of our company around two key business areas: MRD and Immune Medicine," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "I am encouraged by the solid momentum across both business areas as we optimize our resource allocation to capitalize on the multiple opportunities ahead."

Recent Highlights

Revenue of $38.6 million for the first quarter 2022, representing a 0.5% increase from the first quarter 2021.

clonoSEQ test volume in the first quarter 2022 grew 45% versus the first quarter of prior year and 12% over the fourth quarter of 2021.

Signed expanded MRD pan-portfolio agreement with major partner in MM and CLL for the use of MRD status as a clinical endpoint.

Recognized $3.0 million in MRD regulatory milestone revenue resulting from a biopharmaceutical partner who used data from our MRD assay to support its drug approval.

Completed analysis from immuneSense Lyme blinded data from 990 participants, confirming T-Detect Lyme is nearly twice as sensitive as the current standard of care (54% T-Detect Lyme vs 30% STTT sensitivity, both at 99% specificity). T-Detect Lyme offering to be made available during 2022 Lyme season.

Entered a new T-MAP collaboration with the Janssen Pharmaceutical Companies of Johnson & Johnson to map T cell responses to RSV to support Janssen’s RSV vaccine program.

Announced the appointment of Tycho Peterson as chief financial officer, who brings several decades of financial leadership and experience within the life science and diagnostic industries.

First Quarter 2022 Financial Results

Revenue was $38.6 million for the quarter ended March 31, 2022, representing a 0.5% increase from the first quarter in the prior year. Immune Medicine revenue was $20.8 million for the quarter, representing a 4% increase from the first quarter in the prior year. MRD revenue was $17.8 million for the quarter, representing a 3% decrease from the first quarter in the prior year.

Operating expenses were $101.7 million for the first quarter of 2022, compared to $79.7 million in the first quarter of the prior year, representing an increase of 28%.

Net loss was $62.8 million for the first quarter of 2022, compared to $40.6 million for the same period in 2021.

Cash, cash equivalents and marketable securities was $500.7 million as of March 31, 2022.

2022 Financial Guidance

Adaptive Biotechnologies reiterates full year 2022 revenue to be in the range of $185 million to $195 million.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its first quarter 2022 financial results after market close on Wednesday, May 4, 2022 at 4:30 PM Eastern Time. The conference call can be accessed at View Source The webcast will be archived and available for replay at least 90 days after the event.