On October 1, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting significant unmet needs in the treatment of tumors and viruses, reported preliminary first cohort data from the Emory University physician-sponsored clinical trial being conducted at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta by Dr. Tobey MacDonald, Professor of Pediatrics and Director of the Pediatric Neuro-Oncology Program (Press release, Moleculin, OCT 1, 2020, View Source [SID1234567908]). He is studying the use of WP1066 (AflacST1901), a proprietary Moleculin drug candidate, as a potential treatment for childhood brain tumors. The first three patients in the trial received treatment at a dose level of 4 mg/kg with no adverse events related to WP1066 and the study will now proceed to the next higher dose of 6 mg/kg. One of these patients with diffuse intrinsic pontine glioma (DIPG), showed an apparent response to the treatment with both clinical improvement and radiologic reduction of tumor size.

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

Dr. MacDonald stated, "We are very pleased that this trial has successfully completed the first cohort without any safety issues and will now progress to the second cohort at an escalated dose level. We must, of course, be very careful not to draw any conclusions from such preliminary data, but to have an objective response in a DIPG patient is frankly, unexpected."

Mr. Walter Klemp, Chairman and CEO of Moleculin, "When you look at the clinical trial history of DIPG, despite approximately 200 clinical trials, no drug has shown significant activity in this disease, so we find this initial activity particularly encouraging. WP1066 is an immuno-stimulating p-STAT3 inhibitor and has been shown to stimulate immune responses that successfully modulate oncogenic transcriptional activity in tumor cells and repress their ability to drive tumor growth. Coupled with the activity we have recently seen with WP1220, a close analog to WP1066, in its proof of concept clinical trial for the topical treatment of cutaneous t-cell lymphoma, we are more committed than ever to determine the full potential of this new class of p-STAT3 inhibitors. We now have six drug candidates, with three of them showing human activity, so we need to be careful not to confuse this p-STAT3 inhibitor pipeline with the recent announcement regarding our antimetabolites and their potential to treat viruses. We have placed a high priority on reducing risk for our investors by creating what we call ‘multiple shots on goal,’ and the events of this week are showing just how effective that strategy has been."

Mr. Klemp concluded, "Consistent with our history of providing clinical trial updates on a cohort-by-cohort basis, we look forward to updating investors on the continued progress of this trial as additional cohorts are completed. For more information regarding the design of this study, please refer to View Source;

On October 1, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported it has completed its tender offer for all outstanding shares of Pfenex Inc. for $437.5 million in cash, plus one non-transferable contingent value right (CVR) per share representing the right to receive a contingent payment of $78 million in cash if a certain specified milestone is achieved (Press release, Ligand, OCT 1, 2020, View Source [SID1234567896]). The acquired company will cease trading on the NYSE American under the symbol PFNX effective as of October 1, 2020.

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"This is a transformative acquisition that provides a highly valuable technology platform and a portfolio of royalty-bearing collaborations with leading pharmaceutical companies for treatments and vaccines. The business is well established with an attractive growth outlook that is expected to add significantly to Ligand’s financial growth and performance," said John Higgins, Chief Executive Officer of Ligand. "The expertise we acquired in the expression of complex proteins is highly complementary to Ligand’s industry-leading antibody and drug enabling technologies, which together comprise a comprehensive discovery and early stage platform. We welcome our new colleagues to Ligand and look forward to growing the integrated business with our expanded platform supporting the pharmaceutical industry."

The acquired protein expression technology platform is utilized to develop next-generation and novel protein therapeutics to improve existing therapies and create new therapies for biological targets linked to critical, unmet diseases. The proprietary platform uses P. fluorescens bacterium, which are especially well-suited for complex, large-scale protein production that cannot be made by more traditional host systems. The technology can contribute significant value to biopharmaceutical development programs by reducing development timelines and costs for manufacturing human therapeutics and vaccines.

Financial and Business Highlights

The acquired business is forecasted to generate $30 million of total revenue in 2021 and double that amount, or $60 million, in 2023. Royalties are expected to be the major component of total revenue and are forecasted to increase Ligand’s royalty revenue by approximately 50% starting by the end of 2021 assuming approval of the programs partnered with Jazz and Merck. The newly acquired business is expected to be accretive to Ligand in 2021 and contribute $1.50 in adjusted earnings per share in 2023.
Regulatory submission is expected to be filed in the fourth quarter 2020 for Merck’s V114, with potential approval and launch in 2021. Jazz Pharmaceuticals anticipates submitting the JZP-458 biologics license application (BLA) as early as year-end 2020 and is targeting mid-2021 for U.S. launch following BLA submission and approval.
Serum Institute of India (SII) recently launched its PNEUMOSIL vaccine to low- and middle-income countries where price previously was a barrier to providing sufficient vaccination. SII is targeting an initial delivery of 150 million doses of the vaccine annually.
Ligand gains eight existing partner contracts and over $600 million of remaining milestone payments to potentially be paid to Ligand.
Ligand believes it can secure at least three new partnerships over the next 12 months whereby large pharma or leading biotech companies license Ligand’s newly acquired protein expression technology.
Partnership Highlights

The acquisition brings to Ligand major collaborations with Jazz Pharmaceuticals, Merck, Serum Institute of India and Alvogen. Each has the potential to contribute meaningfully to Ligand’s royalty revenue, as follows:

Jazz Pharmaceuticals: Under this collaboration Ligand will receive tiered royalties on net sales of JZP-458. JZP-458 is a recombinant Erwinia asparaginase product candidate that is being developed for the treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products. The Phase 2/3 study of JZP-458 continues to enroll and Jazz Pharmaceuticals anticipates submitting the JZP-458 BLA as early as year-end 2020 and is targeting mid-2021 for U.S. launch following BLA submission and approval. JZP-458 was granted Fast Track designation by FDA in October 2019 for the treatment of this patient population. The Pfenex Expression Technology platform is used to develop complex therapeutic proteins such as JZP-458.
Merck: Under this collaboration, Ligand will receive a low-single-digit royalty on net sales of Merck’s 15-valeant pneumococcal vaccine V114. V114 is expected to compete with Pfizer’s $6 billion Prevnar franchise. Merck plans to file a BLA by the end of 2020, with a standard 10-month FDA review. The CRM197 carrier protein protected via the Pfenex platform is utilized in the Merck V114 vaccine program.
Serum Institute of India: Under this collaboration Ligand will receive a low-to-mid-single-digit royalty on net sales of SII’s recently approved PNEUMOSIL vaccine and Phase 3 meningococcal vaccine. SII is initially targeting 150 million doses of the vaccines annually for low-and middle-income countries in the developing world. SII intends to sell the vaccine at $2 to $4 per dose. PNEUMOSIL utilizes the CRM197 platform.
Teriparatide: Teriparatide Injection is exclusively licensed to Alvogen for sale in the U.S. Under this collaboration, Ligand may be eligible to receive 50% gross profit share on sales of Teriparatide Injection (previously referred to as PF708 and Bonsity, filed with FDA as a 505(b)(2) application referencing Eli Lilly’s Forteo) if the product is rated by FDA as Therapeutic Equivalent to Forteo, and up to 40% if rated differently. Alvogen is currently marketing Teriparatide Injection in the U.S., and continues to seek a Therapeutic Equivalence rating from FDA. Eli Lilly’s Forteo had U.S. sales of $645 million in 2019.1 Alvogen recently received EU Marketing Authorization for Teriparatide Injection from the European Commission, to be marketed under the name Livogiva by Theramex. Important safety information about Teriparatide can be found here.
Expression Technology Platform

The Pfenex Expression Technology is a robust, validated, cost-effective and scalable platform for recombinant protein production, and is especially well-suited for complex, large-scale protein production where traditional systems are not suitable. Multiple global manufacturers have demonstrated consistent success with the platform and the technology is currently out-licensed for numerous commercial and development-stage programs. The versatility of the platform has been demonstrated in the production of enzymes, peptides, antibody derivatives and engineered non-natural proteins. Partners seek the platform as it can contribute significant value to biopharmaceutical development programs by reducing development timelines and costs for manufacturing therapeutics and vaccines. Given pharmaceutical industry trends toward large molecules with increasing structural complexities, the Pfenex Expression Technology is well positioned to meet these growing needs as the most comprehensive broadly available protein production platform in the industry.

CRM197

CRM197 is a non-toxic mutant of diphtheria toxin having a single amino acid substitution of glutamic acid to glycine at position 52. CRM197 is a well-defined protein and functions as a carrier for polysaccharides and haptens, making them immunogenic. It is utilized as a carrier protein in several approved conjugate vaccines for diseases such as Streptococcus pneumoniae, Haemophilus influenzae B and Neisseria meningitidis. CRM197 is produced by the Pfenex Expression Technology platform and is currently being used in vaccine development by multiple partners, including Merck and the Serum Institute of India Private Ltd.

Integration Plan

Ligand plans to operate the newly acquired protein expression technology platform business with approximately 50 specialized employees based in San Diego, CA. By mid-2021, Ligand expects to consolidate its multiple San Diego locations into one facility at the current Pfenex company location.

Transaction Details

On October 1, 2020, Ligand completed the acquisition through a tender offer and subsequent merger of Pfenex with Pelican Acquisition Sub Inc., a wholly owned subsidiary of Ligand (Buyer). Pfenex is now a wholly owned subsidiary of Ligand. The tender offer for all of the outstanding shares of common stock of Pfenex at a price of $12.00 per share, in cash, plus a CVR, which represents the right to receive a contingent payment of $2.00 in cash, without interest and less any applicable withholding taxes, if a specified milestone is achieved, expired as scheduled, at midnight (New York City Time), at the end of the day on Tuesday, September 29, 2020. American Stock Transfer & Trust Company, LLC, the depositary and paying agent for the tender offer, has advised Ligand that 24,744,327 shares of Pfenex common stock (excluding shares with respect to which Notices of Guaranteed Delivery were delivered) were validly tendered and not properly withdrawn in the tender offer, representing approximately 72.0% of the shares outstanding as of the expiration of the tender offer. In addition, Notices of Guaranteed Delivery had been delivered with respect to approximately 2,847,227 shares that had not yet been tendered, representing approximately 8.3% of the outstanding shares. All of the conditions to the tender offer having been satisfied, on September 30, 2020, Buyer accepted for payment and will promptly pay for all shares tendered. The transaction will be funded with cash on hand.

Ligand completed its acquisition of Pfenex through the merger of Buyer with and into Pfenex without a vote of Pfenex’s shareholders pursuant to Section 251(h) of the Delaware General Corporation Law. In connection with the merger, all shares of Pfenex common stock outstanding immediately prior to the effective time (other than shares owned by Ligand, Buyer, Pfenex, any other subsidiary of Ligand’s or any subsidiary of Pfenex, or shares that are held in Pfenex’s treasury, or shares held by any Pfenex stockholder who has properly demanded and perfected appraisal rights under Delaware law) have been converted into the right to receive $12.00 per share, in cash, plus the CVR, without interest (less any required withholding taxes), the same amount paid for all shares validly tendered and not validly withdrawn in the tender offer. As a result of the merger, as of October 1, 2020, Pfenex common stock will cease to be traded on the NYSE American market.

Adjusted Financial Measures

Ligand reports adjusted earnings per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. Ligand’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of Ligand’s press release reporting its results of operations for the period ended June 30, 2020. However, other than with respect to total revenues, Ligand only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing Ligand’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by Ligand’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

On October 1, 2020 ONCODESIGN (Paris:ALONC) (ALONC – FR0011766229), a biopharmaceutical group specialized in precision medicine, reported its results for the first half of 2020 and issues a business update (Press release, Oncodesign, OCT 1, 2020, View Source [SID1234567894]).

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Philippe GENNE, Chairman and Chief Executive Officer of Oncodesign, says: "Within a context marked by an unprecedented global public health crisis in the first semester, Oncodesign has deployed a business continuity plan that has enabled to remain operational to serve clients, drive projects forward and continue the strategic development. The Group has shown resilience, with operating revenue of €18.1 million and a net profit at June 30, 2020. Our total revenue, on a like-for-like basis (excluding upfront and milestone payments), of €11.8 million in the first half of 2020 has held up, with a record cash position of €19.9 million. The first half was also marked by the success of our collaboration with Servier’s laboratories on the LRRK2 project that provided us with a €1 million milestone payment. The RIPK2 project with its First-in-class ODS 101 inhibitor, is following its development plan to the letter, and its IND is still scheduled for June 2021. We are now equipped to meet the targets we have set for 2023: i.e. pursue the clinical development of our drug candidates, support the development of Drug Discovery while consolidating our multi-year Drug Development service partnership offer (DDSA) and Integrated Drug Discovery (IDDS) offer to drive Service revenue and EBITDA growth. The Oncodesign teams and I are more than ever focusing on the Group’s future, as illustrated by the stakes recently acquired by members of the Company’s Senior Management team".

Arnaud LAFFORGUE, Chief Financial Officer of Oncodesign, continues: "From the start of the year, we implemented the growth plan for the next five years and structured our company into three Business Units. Lastly, despite this unprecedented context in which Business Development has been directly penalized, we have recorded a 14% increase in incoming orders compared to last year, to €11.6 million, driven by the signing of a number of multi-year contracts in Asia and Europe. Also, in the first half of the year, we have launched the integrated Drug Discovery service offer, DRIVE, including in partnership with Chinese company Hitgen, and a new service offer dedicated to COVID-19 in association with IDMIT, providing a response to the high level of demand on this market, the latter leading to a substantial order book with revenue prospects on this offer in the second half of 2020. In addition, for the first time, the income statements of 2 main BUs are presented, namely the Service BU and the Biotech BU for the first half of 2020 compared to the homogeneous figures for the first half of 2019 in order to better highlight the performance and challenges of its activities."

First-half 2020 financial results

The Group generated revenue of €11.8 million in the first half of 2020, down 16% compared with H1 2019, due to both the decrease in Biotech revenue, with a milestone payment of €1 million in H1 2020 versus an upfront of €3 million over the same period last year, and, for the Service revenue, resulting from the public health crisis.

Other operating revenue, which totaled €6.29 million in the first half, was up by 5.5% compared with the first half of 2019, and consisted primarily of the following:

the subsidy received from GSK, within the framework of the acquisition of the François Hyafil center, which amounted to €3.96 million in the first half of 2020 (6 months pro rata temporis). The final payment was carried out in January 2020;
the 12% increase in French and Canadian Research Tax Credit to €1.66 million, directly associated with investments in internal programs (RIPK2, MNK1/2) and on AI.
Operating expenses totaled €18.42 million, down because of the reduction in variable costs such as purchases of external subcontracting services and business trips because of the pandemic. Personnel expenses were stable at €9.2 million (+1.3%) as a result of the decision taken at the start of the year, given the first effects of the pandemic on the economy, to freeze all wage increases that normally take place at this time of the year. However, it is worth noting the effect of the full presence over the first half of the year of the new staff recruited during the second half of 2019, i.e. an additional 9 people.

Research & Development investments amounted to €5.41 million in the first half of 2020, versus €4.0 million at June 30, 2019. This acceleration in spending (+35%) was primarily associated with the RIPK2 program’s regulatory preclinical phase, entirely financed by Oncodesign.

Thanks to good management of operating expenses, down €1.1 million (-6%), there was an operating loss of -€320k, a controlled decrease of €830k compared with the first half of 2019 in view of the investment efforts undertaken over the period.

The net profit remained close to zero (+€40k), despite a financial income deteriorated due to the effect of exchange rate fluctuations (US$) for €125k. In addition, the restatement of goodwill, following the sell and lease-back of the Les Ulis building, enabled income of €500k to be recognized in consolidated accounts.

Cash position

The Company had cash and cash equivalents of €19.9 million at June 30, 2020, a substantial increase of +50% compared with June 30, 2019 (€13.3 million) despite the Group’s considerable R&D investments. This figure includes the €1.0 million milestone payment received from Servier, the €7.92 million GSK subsidy received for 2020 and the Research Tax Credit for 2018 (€3.5 million not redeemed at the end of 2019 and regularized in April 2020) and 2019, €3.2 million and usually received at the end of the following year. Lastly, it should be noted that this cash position was achieved without, as yet, incorporating the State-Guaranteed Loan.

First-half 2020 business update and outlook

Following completion of the new organization, the Service BU, which has 203 employees, realizes a total revenue of 14M€ by cumulating the "External" revenues corresponding to the sales realized with Oncodesign’s customers, and the "Internal" revenues1 carried out for the execution of work related to our therapeutic projects for the Biotech BU (RIPK2, LRRK2, MNK1/2). It is stable compared to last year at the same date (+260 K€).

External revenues amounted to €9.03 million over the period, a decrease of 10% in the first half of the year, following the economic slowdown associated with the global health crisis. Oncodesign SA (worldwide clients excluding the USA and Canada) recorded a 13.6% decrease in revenue to €7.71 million, while revenue generated in North America (USA and Canada) remained dynamic, increasing by 18.4% to €1.4 million. The latter’s growing weight – with it now accounting for 16% of revenue compared to 10% in 2019 – demonstrates its strategic role for the Service Business Unit, thus justifying its development over the coming years. Moreover, activity recorded in Asia (Japan and South Korea) has also grown rapidly over the last 3 years, with this region now accounting for 7% of total first-half 2020 revenue compared to just 2% in 2018. This international development reflects the substantial investments undertaken by the Company, and notably the strengthening of the sales teams.
Internal revenues increased by 30% to almost €5 million following the acceleration of our programs for the Biotech BU.
In terms of EBITDA, the BU achieves an increase of €1.8 million to reach €0.67 million in the first half of 2020, representing an improvement in EBITDA margin of 13.1% from -8.3% to +4.8% (thanks to good cost control and increased productivity as a result of the new BU organization). This performance illustrates the Service BU’s ability to improve its operational performance by optimizing its productivity and internal organization. These figures give us confidence in the Service BU’s ability to achieve its profitability objectives at term.

The objectives of the Service BU are to accelerate the development of the sale of integrated and long-term service contracts for growth in Service revenues and EBITDA.

A COVID-19 offer has been developed in association with IDMIT. This offer is seeing very high demand in the current context, particularly as few companies have the infrastructure and expertise to undertake this type of service. This offer has promising revenue potential, both for the short term in H2 and for the coming years.

Impact of the Covid pandemic: the activation of our Business Continuity Plan and the continuation of activity on our sites and in our laboratories allows us to undertake the work entrusted to us by our clients. To date, we have not recorded widespread order cancellations from our clients. The procurement of supplies is still possible for most of our consumables/raw materials; delivery times are occasionally longer but this isn’t blocking our work. Lastly, the nature of our portfolio of offers, notably multi-year programs, guarantees us a level of recurrent activity scheduled over the long term. In contrast, the booking of orders for one-off and non-recurrent offers could be penalized, with the cancellation of conferences and ongoing travel restrictions impeding the fieldwork of our sales staff, without us currently being able to assess the precise impact.

Oncodesign’s 2023 targets are notably, for the Service BU, to achieve revenue of €50 million and EBITDA of between 15% and 20%.

Biotech revenues consist mainly of the sale of partnerships and licensing resulting from Nanocyclix: so in the short term from Up-fronts / Milestones and the coverage of research costs of the Servier Partnership for LRRK2. Thus, its sales reached €2.8 million in the first half of 2020, versus last year’s figure of €4 million that included the initial €3 million payment by Servier, within the framework of the strategic partnership sealed in March 2019 to develop LRRK2 kinase inhibitors as a treatment for Parkinson’s Disease. Revenue relating to work on LRRK2 increased by +78% to €1.8 million and the program is progressing rapidly in accordance with its initial schedule.

Costs in the Biotech BU are under control thanks to our efforts to rationalize our expenses, in connection with the new allocation of scientific staff and the control of our purchases.

However, the medium-term trend of this BU, whose programs are progressing according to their development plan, should enable it to achieve milestones in the coming semesters.

The objectives of the Biotech BU are to continue to ensure the ramping up of our pipeline via the selection of, on the one hand, kinase inhibitor drug candidates resulting from Nanocyclix technology and, on the other hand, external opportunities on other targets.

Biotech’s prime focus is to develop the maturity of our therapeutic pipeline by taking our molecules to the clinical development stage: RIPK2, LRRK2 and MNK1/2 inhibitors in oncology.

At the end of 2019, the substantial investment efforts undertaken in R&D enabled Oncodesign to select a First-in-Class drug candidate, a RIPK2 kinase inhibitor, for autoimmune and inflammatory diseases, which represents a first step in the creation of expected value. As a reminder, Oncodesign initially planned to finance the development of ODS 101 up to the IND (significant risk-limiting step on the inhibitor by regulatory multi-species toxicity studies), programmed in June 2021, while looking for a Pharma partner.

In February 2020, Servier and Oncodesign announced that they had reached a major milestone several months ahead of schedule, within the framework of their strategic partnership in the research and development of drug candidates to treat Parkinson’s disease (LRRK2 program). Oncodesign received a first milestone payment of €1 million associated with the program’s first success.

Moreover, discussions are ongoing with new partners to enable the resumption of the clinical development of the mutated EGFR radiotracer. Regarding the partnership with Bristol-Myers Squibb (BMS), after the internalization of the program at the end of 2018, the company decided to stop the program for toxicity reasons related to the target. The intellectual property concerning the molecules has been returned to Oncodesign, which is free to exploit them. Lastly, the MNK1/2 program is continuing to move forward, the aim being to reach the pre-candidate stage by the end of 2020.

Impact of the Covid pandemic: for the Biotech Business Unit, as a result of our autonomy on work programs, the pandemic has had little impact on our activity or the ramping up of our pipeline. However, we are observing a slowing down of Big Pharma decision-making and therefore partner research processes for RIPK2.

Oncodesign is aiming to take 3 products to the clinical phase by 2023.

ARTIFICIAL INTELLIGENCE BU

The objective of the AI BU is to continue the development of our strategic technological pillars using AI: structure the Precision Medicine platform of the 21st century

On the basis of the OncosnipeTM project, whose launch 3 years ago initiated the application of AI technologies to the detection of new therapeutic targets and enabled the creation of an internal center of expertise on this subject, a third Business Unit dedicated to Artificial Intelligence was created in April 2020, under the direction of Stéphane Gérart.

Impact of the Covid pandemic: the OncosnipeTM project was penalized by the public health crisis in the first half, as it relies on a clinical trial whose patient enrollments had to be suspended during this period. In return, we opened 5 new clinical centers and received the associated BPI subsidy of €0.4 million at the end of June within the framework of the PSPC competitive cluster structuring project.

This BU is aiming to build, by 2023, a platform to identify and validate new therapeutic targets and to increase the Drug Discovery process’ reliability and reduce its development times, while developing revenue streams by providing research services to industry.

Oncodesign’s first-half 2020 financial report is available in French on the Company’s website:
www.oncodesign.com

Next investor meeting: Investir – Direct Dirigeants event in Paris on Tuesday October 6, 2020

On October 1, 2020 Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions utilizing the latest biotechnology, and MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing potential new therapies for cancer, reported the first patient has been dosed in the pivotal Phase 2 study of zandelisib (formerly called ME-401), an oral, once-daily, investigational drugcandidate selective for phosphatidylinositol 3-kinase delta (PI3Kδ) in patients with indolent B-cell nonHodgkin’s lymphoma (iNHL) without Small lymphocytic lymphoma, lymphoplasmacytic lymphoma (LPL), and Waldenström’s macroglobulinemia (WM) in Japan.

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This Phase 2, multicenter, open-label, single-arm clinical study is conducted by Kyowa Kirin to evaluate zandelisib as monotherapy for treatment of Japanese patients with relapsed or refractory iNHL with at least two prior systemic therapies.

Yoshifumi Torii, Ph.D., Vice President, Head of R&D Division of Kyowa Kirin, said "I am pleased that we have initiated the clinical study to seek regulatory approval in Japan. We are committed to delivering zandelisib as a new potential treatment option for Japanese patients and their physicians. And we continue to work closely with MEI Pharma to expand the global development program for zandelisib."

"The initiation of the Japanese Phase 2 pivotal study is an important addition to the zandelisib global development program," stated Richard Ghalie, M.D., senior vice president, clinical development of MEI Pharma. "We are excited to continue working diligently in close partnership with Kyowa Kirin to build on the potential for zandelisib inside and outside the U.S., and to expand development into indications beyond follicular lymphoma."

In April 2020, MEI and Kyowa Kirin entered a global license, development, and commercialization agreement to further develop and commercialize zandelisib. MEI and Kyowa Kirin will co-develop and co-promote zandelisib in the U.S., with MEI booking all revenue from the U.S. sales. Kyowa Kirin has exclusive commercialization rights outside of the U.S.

Indication iNHL* *SLL, WM and LPL are excluded
Phase Phase 2
Design Multicenter, open-label and single-arm study
Administration group Zandelisib monotherapy
Primary Endpoint Objective response rate (ORR)
Sample size 60
Estimated study completion September 2024
Countries/Regions Japan

About Zandelisib
Zandelisib (formerly called ME-401) is an investigational cancer treatment being developed as an oral, once-daily, selective PI3Kδ inhibitor for the treatment of B-cell malignancies. In March 2020 the U.S. FDA granted zandelisib Fast Track designation.

In April 2020, MEI and Kyowa Kirin entered a global license, development, and commercialization agreement to further develop and commercialize zandelisib. MEI and Kyowa Kirin will co-develop and co-promote zandelisib in the U.S., with MEI booking all revenue from the U.S. sales. Kyowa Kirin has exclusive commercialization rights outside of the U.S.

Ongoing studies evaluating zandelisib include TIDAL (Trials of PI3K DeltA in Non-Hodgkin’s Lymphoma) a Phase 2 clinical trial evaluating zandelisib as a monotherapy for the treatment of adults with follicular lymphoma after failure of at least two prior systemic therapies including chemotherapy and an antiCD20 antibody. Subject to the results, upon completion TIDAL is intended to be submitted to FDA to support an accelerated approval marketing application under 21 CFR Part 314.500, Subpart H. Ongoing zandelisib studies also include a Japanese Phase 2 pivotal study in patients with iNHL without Small lymphocytic lymphoma, LPL and WM being conducted by Kyowa Kirin. (Press release, Kyowa Hakko Kirin, OCT 1, 2020, View Source [SID1234567893])

On October 1, 2020, Protalix BioTherapeutics, Inc., a Delaware corporation (the "Company"), reported that it entered into an ATM Equity OfferingSM Sales Agreement (the "Sales Agreement") with BofA Securities, Inc., as the Company’s sales agent (the "Agent") (Filing, 8-K, Protalix, OCT 1, 2020, View Source [SID1234567892]). Pursuant to the terms of the Sales Agreement, the Company may sell from time to time through the Agent shares of the Company’s common stock having an aggregate offering price of up to $30 million (the "Shares"). The Shares will be issued pursuant to the Company’s shelf registration statement on Form S-3 (Registration No. 333-230604). The Company intends to use the net proceeds from the offering, after deducting the Agent’s commissions and the Company’s offering expenses, for general corporate purposes.

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In accordance with the terms of the Sales Agreement, the Company may offer and sell the Shares at any time and from time to time through the Agent. Sales of the Shares, if any, will be made by means of transactions that are deemed to be "at the market" offerings as defined in Rule 415 under the Securities Act of 1933, as amended, including block trades and sales made in ordinary brokers’ transactions on the NYSE American or otherwise at market prices prevailing at the time of the sale, at prices related to prevailing market prices or at negotiated prices. Under the terms of the Sales Agreement, the Company may also sell Shares to the Agent as principal for its own account at a price to be agreed upon at the time of sale. Any sale of Shares to the Agent as principal would be pursuant to the terms of a separate terms agreement between the Company and the Agent.

The foregoing description of the Sales Agreement in this report does not purport to be complete and is qualified by reference to the full text of the Sales Agreement, which is filed as Exhibit 1.1 hereto. The legal opinion and consent relating to the Shares are included as Exhibits 5.1 and 23.1, respectively, hereto.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy any Shares, nor shall there be any sale of Shares in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.