December 12, 2019: BioCentury Publications, Inc.: Leo's IL-13 mAb hits in atopic dermatitis; how it stacks up against Dupixent remains a question mark 15 December 2019


Over a year after the primary completion of tralokinumab's Phase III studies, Leo Pharma has said the anti-IL-13 mAb met the primary endpoint in all three trials to treat moderate-to-severe atopic dermatitis without divulging details.

When the data are announced, the efficacy bar to beat will be the benefit seen with Dupixent dupilumab from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and Sanofi (Euronext:SAN; NASDAQ:SNY).

According to Leo Pharma A/S, tralokinumab met the primary endpoint of Investigator Global Assessment (IGA) score of clear or almost clear skin at week 16 and at least a 75% or greater change from baseline in Eczema Area and Severity Index (EASI) score at week 16 in ECZTRA 1, ECZTRA 2 and ECZTRA 3. The mAb also met the trial's secondary endpoints: change from baseline to week 16 in SCORing of Atopic Dermatitis (SCORAD), Pruritus Numeric Rating Scale (NRS) of at least 4, and Dermatology Life Quality Index (DLQI).

The "actual primary completion date" for the trials were in August, September and October 2018, according to However, Leo Pharma told BioCentury that the full data set was not available for analyses until the "actual study completion" date listed on, which was in August-October of this year.

Leo said it plans to announce detailed results and submit regulatory applications next year for the therapy, which it licensed from AstraZeneca plc (LSE:AZN; NYSE:AZN) in 2016.

In three Phase III trials of Dupixent in atopic dermatitis, IGA score at week 16 was the primary endpoint; EASI75 at week 16 was a secondary endpoint. Among patients treated with Dupixent in the studies, the proportion that achieved clear or almost clear skin range from 36% to 39% as measured by IGA. The proportion of patients who achieved EASI75 was 44-69% across the three trials.

Targets: IL-4RA - The alpha subunit of the interleukin-4 (IL-4) receptor (IL-4R; IL-4RA; CD124); IL-13 - interleukin-13 (IL-13).






BioCentury Publications, Inc.

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BioCentury Publications, Inc. provides informed business intelligence solutions for biotech and pharma companies. It offers a journal, which provides analysis, interpretation, and commentary on industry development, corporate performance, clinical and commercial product development, emerging technology, public policy, and financial markets; newspaper and online newspaper for biotech and pharma companies; quarterly stock roundup, which tracks various public biotech companies in the United States, Europe, Canada, and the Asia-Pacific; online intelligence; online library for articles, charts, and tables covering collection of publications, and analysis and reporting; data solutions; and conferences. The company was founded in 1993 and is based in San Carlos, California. It has locations in Redwood City, California; Chadds Ford, Pennsylvania; Chicago, Illinois; Oxford, the United Kingdom; and Washington, District of Columbia.


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December 14: BioCentury Publications, Inc.: Management Tracks: Alector hires new president, COO and appoints R&D committee co-chairs; plus Pyxis, Translate Bio, Canaan, PMV, Janpix, Kiadis and more

Neuroimmunology and immuno-oncology company Alector Inc. (NASDAQ:ALEC) hired Shehnaaz Suliman as president and COO and made Richard Scheller and Thomas Sudhof co-chairs of its strategic portfolio advice and review committee. Suliman was SVP of corporate development and strategy at Theravance Biopharma Inc. (NASDAQ:TBPH). Scheller, who won the 2013 Lasker Prize, is chairman of R&D at BridgeBio Pharma Inc. (NASDAQ:BBIO) and was head of therapeutics and CSO at 23andMe Inc. from 2015-2019. Sudhof, a Nobel laureate, is a professor at Stanford University School of Medicine.

Immuno-oncology play Pyxis Oncology Inc. said Lara Sullivan will become CEO, succeeding founding CEO David Steinberg, and appointed her to the board. Steinberg, a general partner at Longwood Fund, will become chairman. He succeeds founding chairman John Flavin, who will remain as an independent director. Sullivan was founder and president of SpringWorks Therapeutics Inc. (NASDAQ:SWTX).

mRNA therapeutics company Translate Bio Inc. (NASDAQ:TBIO) said SVP of platform technology Frank DeRosa has succeeded scientific founder Michael Heartlein as CTO, and Chief Legal Officer Paul Burgess will take on the additional role of COO. Heartlein will become EVP and founder, a newly created position.

Canaan promoted Julie Grant to general partner. Grant's portfolio includes pain company Nocion Therapeutics Inc. and two newcos in stealth: a Stanford spinout and a targeted oncology play, of which Grant is founding CEO. Previous portfolio companies include Cellular Research, which Becton Dickinson and Co. (NYSE:BDX) acquired, and Protagonist Therapeutics Inc. (NASDAQ:PTGX). The firm said that to its knowledge, Grant is the only female GP promoted internally from within a healthcare VC firm in the last 15 years.

Cancer company PMV Pharmaceuticals Inc.

December 13: BioCentury Publications, Inc.: Sarepta wins approval for second exon-skipping DMD therapy after disputing August CRL

FDA reversed an August decision that rebuffed Sarepta's Vyondys 53 golodirsen, granting accelerated approval to the therapy to treat Duchenne muscular dystrophy in patients amenable to exon 53 skipping. The decision came as a surprise late Thursday, when the company revealed for the first time that it had appealed a complete response letter in which the agency flagged infection and toxicity concerns.

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) was up $28.03 (28%) to $128.50 during after-hours trading.

If the move holds, it would represent a gain in Sarepta's market cap of about $2.1 billion and help the stock return to levels not seen since before the summer setback, which shaved $1.1 billion off the company's valuation (see "Sarepta Sinks After FDA Rejects DMD Therapy").

Sarepta made a formal dispute resolution request to FDA; the Office of New Drugs then evaluated and granted the appeal, allowing the company to resubmit the NDA.

The indication represents about 8% of DMD patients.

The company said Vyondys 53 will be available immediately for the same price as Exondys 51. The company's first exon-skipping DMD therapy has a wholesale acquisition cost of $8,000 per 10 ml vial.

The accelerated approval is based on the surrogate endpoint of increased dystrophin production in skeletal muscle. FDA noted that the therapy's clinical benefit has not been established and will require a post-marketing trial to confirm its ability to improve motor function.

Vyondys 53 is in the Phase III ESSENCE trial; its primary endpoint is the 6-minute walk test. The confirmatory trial is expected to conclude by 2024.

Sarepta received a rare pediatric disease Priority Review voucher.

Separately Thursday, FDA authorized GSP Neonatal Creatine Kinase-MM from PerkinElmer Inc. (NYSE:PKI) as the first test for newborn screening of DMD.

December 11: BioCentury Publications, Inc.: Perceptive launches $210M early-stage fund tied to Garabedian's accelerator Xontogeny

Two and a half years after Perceptive Advisors' Chris Garabedian launched Xontogeny to foster biotech start-ups' growth, the firm has created a $210 million fund that will invest in companies the accelerator is nurturing through the clinical proof-of-concept stage.

Perceptive Xontogeny Venture Fund (PXV) will aim to be the lead or sole investor in about 8-10 series A rounds of $10-$20 million, and will participate in subsequent series B rounds. LPs include endowments, foundations, family offices and institutional investors.

PXV is Perceptive's inaugural VC fund. The firm has historically favored investments in later-stage and publicly traded companies; it said PXV will complement its Life Sciences Master and Credit Opportunity Funds.

Garabedian, a former CEO of Sarepta Therapeutics Inc. (NASDAQ:SRPT) who joined Perceptive in 2017, created Xontogeny to provide seed funding, managerial and operational support, and consulting services to preclinical life sciences companies. Garabedian will manage the fund for Perceptive, which is the accelerator's majority owner, and he will continue to be Xontogeny's chairman and CEO (see "Xontogeny Raises $25M in Series A").

PXV closed earlier this week, but it has already invested in two therapeutics companies: Quellis Biosciences Inc. and Landos Biopharma Inc. About 80% of the fund's investments will be channeled toward drug development companies, with the balance aimed at non-drug technologies.

Perceptive will invest the fund in some of Xontogeny's more advanced companies as they progress to the next stage of development. "We want companies that have a product within two years of the clinic and within five years of clinical proof of concept," said Garabedian. He added the fund was particularly interested in investing in companies working on products for CNS, cardio-metabolic, rare disease, or immuno-inflammatory conditions.

According to Garabedian, Xontogeny companies typically spend 9-18 months in the accelerator, with three to four incubating at once.

December 03: BioCentury Publications, Inc.: IFM Therapeutics gets $55.5M to launch incubator and new subsidiaries

IFM Therapeutics is doubling down on its asset-centric subsidiary model, raising $55.5 million to launch an incubator and up to three new subsidiaries focused on developing small molecules targeted at the innate immune system for oncology and inflammatory disorders.

New investor Omega Funds led the financing, joined by existing investors Atlas Venture and Abingworth.

The new funds will enable IFM Therapeutics LLC to move two candidates into the clinic within the next three years. The company said it will be pursuing a basket of innate immune targets, including a number that fall into the inflammasome and pattern recognition receptor space.

Inflammasome complexes have come to the fore as attractive targets because they sit upstream of cytokines, meaning interfering with them can cut off inflammation at the source. IFM is one of the few companies pursuing novel inflammasome targets beyond NLRP3 (see "Biotechs Explore the Next Generation of Inflammasome Targets, with Caution").

The first candidate to emerge from the new IFM Discovery incubator and advance to preclinical testing will be moved into IFM Quattro, the company's third subsidiary, for further development. The latest financing will also enable IFM Therapeutics to create up to two more subsidiaries to house additional programs.

IFM itself spun out of IFM Therapeutics Inc. to develop the parent company's remaining assets after it was acquired in 2017 by Bristol-Myers Squibb Co. (NYSE:BMY).

After the BMS sale, IFM regrouped and created two new subsidiaries, IFM Due and IFM Tre, both of which entered deals with Novartis AG (NYSE:NVS) less than a year after they launched.

In April, Novartis paid $310 million up front and up to $1.3 billion in milestones for IFM Tre, gaining IFM-2427, an NLRP3 antagonist in Phase I testing (see "IFM Tre Deal Offers Glimpse Into Novartis' M&A Future").

In September, Novartis entered a deal to finance IFM Due's R&D costs in exchange for an option to acquire the subsidiary for up to $840 million in upfront and milestone payments.

November 28: BioCentury Publications, Inc.: Incyte wins Priority Review for cholangiocarcinoma therapy

Two months after presenting Phase II data at ESMO, Incyte Corp. (NASDAQ:INCY) said FDA has accepted and granted Priority Review to an NDA for pemigatinib to treat the rare bile duct cancer cholangiocarcinoma. The candidate's PDUFA date is May 30.

Pemigatinib's target population is patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. The candidate is an oral inhibitor of FGFR isoforms 1, 2 and 3.

Incyte reported data in the indication at the European Society for Medical Oncology meeting in September showing that pemigatinib led to an overall response rate of 36% among 107 patients with FGFR2 fusions or rearrangements. The median duration of response was 7.5 months and the median progression-free survival was 6.9 months. According to Incyte, patients on later lines of treatment typically have response rates of less than 10% and a median PFS of about three months (see "Incyte Lining up Pemigatinib Filing on ESMO Data").

After cholangiocarcinoma, Incyte is looking to take the program into larger indications, including FGFR-positive urothelial carcinoma and FGFR-positive solid tumors regardless of histology. Pemigatinib is in several Phase II trials for bladder cancer and solid tumors.

One FGFR inhibitor has already made it to the market for bladder cancer. Balversa erdafitinib from Johnson & Johnson (NYSE:JNJ) was approved in April to treat second-line urothelial carcinoma with susceptible FGFR2 or FGFR3 mutations (see "J&J's Balversa Gets Accelerated Approval").

Source: Company Website

November 28: BioCentury Publications, Inc.: Incyte wins Priority Review for cholangiocarcinoma therapy

Two months after presenting Phase II data at ESMO, Incyte Corp. (NASDAQ:INCY) said FDA has accepted and granted Priority Review to an NDA for pemigatinib to treat the rare bile duct cancer cholangiocarcinoma. The candidate's PDUFA date is May 30.

Pemigatinib's target population is patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. The candidate is an oral inhibitor of FGFR isoforms 1, 2 and 3.

Incyte reported data in the indication at the European Society for Medical Oncology meeting in September showing that pemigatinib led to an overall response rate of 36% among 107 patients with FGFR2 fusions or rearrangements. The median duration of response was 7.5 months and the median progression-free survival was 6.9 months. According to Incyte, patients on later lines of treatment typically have response rates of less than 10% and a median PFS of about three months (see "Incyte Lining up Pemigatinib Filing on ESMO Data").

After cholangiocarcinoma, Incyte is looking to take the program into larger indications, including FGFR-positive urothelial carcinoma and FGFR-positive solid tumors regardless of histology. Pemigatinib is in several Phase II trials for bladder cancer and solid tumors.

One FGFR inhibitor has already made it to the market for bladder cancer. Balversa erdafitinib from Johnson & Johnson (NYSE:JNJ) was approved in April to treat second-line urothelial carcinoma with susceptible FGFR2 or FGFR3 mutations (see "J&J's Balversa Gets Accelerated Approval").

Source: Company Website

November 27: BioCentury Publications, Inc.: Nov. 26 Company Quick Takes: Kyowa, Ardelyx expand partnership; plus ICER, Celltrion, Asahi-Veloxis and Merck

Kyowa, Ardelyx expand collaboration with new targets and $20M investment Under an expanded partnership, Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) will pay Ardelyx Inc. (NASDAQ:ARDX) $10 million in research support over the next two years to identify and develop programs against two undisclosed targets, after which Kyowa has the option to license resulting products for $10.5 million in upfront payments and up to $500 million in development and sales milestones. In a second deal, Kyowa invested $20 million in Ardelyx through the purchase of 2.9 million shares at $6.96. Kyowa gained Japanese rights in 2017 to develop and commercialize Ardelyx's Ibsrela tenapanor for cardiorenal diseases. FDA approved Ibsrela in September for irritable bowel syndrome with constipation, and Ardelyx plans to submit an NDA to FDA in 2020 for the candidate to treat hyperphosphatemia in end-stage renal disease patients. Ardelyx gained $1.16 (18%) to $7.54 on Tuesday (see "Ardelyx Gains on Phase III Hyperphosphatemia Combination Data").

AbbVie's RA successor to Humira is cost-effective, ICER finds ICER released an evidence report Tuesday that found Rinvoq upadacitinib from AbbVie Inc. (NYSE:ABBV) leads to higher rates of disease remission for rheumatoid arthritis and offers a small to substantial net health benefit over the pharma's Humira adalimumab. The Institute for Clinical and Economic Review concluded that, after accounting for assumed manufacturer rebates of 25-26% to its list price of $59,860, the JAK-1 inhibitor's net price is in line with ICER's value-based price benchmark range. ICER's California Technology Assessment Forum (CTAF) will review the report at a public meeting on Dec. 9 (see "Rinvoq Approval Bolster's AbbVie's Autoimmune Portfolio").

Europe approves subcutaneous infliximab biosimilar Celltrion Healthcare Co.

November 26: Blackstone places $400M bet to launch Ferring gene therapy subsidiary

Ferring created a subsidiary to house registration-stage bladder cancer gene therapy nadofaragene firadenovec and drew a $400 million investment from Blackstone on Monday.

Ferring Pharmaceuticals A/S and Blackstone Life Sciences launched FerGene to commercialize nadofaragene in the U.S. and further its global development. Ferring will also invest up to $170 million in the subsidiary.

A BLA for nadofaragene is under Priority Review; its PDUFA date is undisclosed.

The therapy is being developed to treat patients with high grade, Bacillus Calmette-Guerin unresponsive, non-muscle invasive bladder cancer (NMIBC). Phase III data for nadofaragene will be presented at the Society of Urologic Oncology meeting on Dec. 5.

Ferring will assume exclusive, worldwide rights to nadofaragene upon FDA approval. The biotech received an option to the candidate from Finland-based FKD Therapies Oy last year for an undisclosed sum. Nadofaragene originated from Merck & Co. Inc. (NYSE:MRK); FKD got rights to nadofaragene under a 2011 deal.

The gene therapy comprises an adenovirus serotype 5 vector containing interferon alfa-2b. Administered by catheter every three months, the gene is taken up by the cells of the bladder wall, which then secrete high levels of the naturally occurring antitumor protein.

Source: Company Website

November 23: BioCentury Publications, Inc.: Sanofi adds in home-grown trispecific antibody technology to grow oncology franchise

Promising preclinical data from a trispecific mAb platform developed by Sanofi could provide a new growth platform for its cancer pipeline, as the pharma seeks to wean off its reliance on Regeneron.

Sanofi (Euronext:SAN; NYSE:SNY) has been striving to expand its cancer franchise as its relationship with Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) winds down.

Last year, Sanofi hired John Reed as EVP and global head of R&D, pulling him from Roche (SIX:ROG; OTCQB:RHHBY), which has led pharmas in cancer sales for years, where he was global head of the Pharma Research and Early Development unit.

Much of Sanofi's oncology pipeline stems from a 2007 discovery collaboration and a 2015 immuno-oncology deal with Regeneron.

Source: Company Website

November 22: BioCentury Publications, Inc.: Longwood launches Immunitas with $39M and single-cell genomics platform

With $39M series A, Immunitas is harnessing single-cell analysis to identify new immune targets for cancer.

Immunitas Therapeutics emerged from stealth mode on Thursday with a $39 million series A round and a single-cell genomics platform to identify novel targets for mAbs that boost antitumor immunity.

Momentum is growing around use of single-cell analysis and human tissue samples, rather than animal models, to identify targets that are relevant to human biology and might be overlooked by conventional approaches (see "The Next Frontier for Single-Cell Analysis").

"Single-cell analysis allows us to home in on relevant cell types that would otherwise be challenging to study as they would be 'averaged out' in the noise," said President Lea Hachigian, who is also a principal at Longwood Fund.

Immunitas, which is based in Cambridge, Mass., was founded in July by Longwood along with Hachigian and three researchers: Kai Wucherpfennig of the Dana-Farber Cancer Institute and Harvard Medical School; Mario Suva of the Broad Institute and Massachusetts General Hospital; and Dane Wittrup of the Massachusetts Institute of Technology (MIT).

Leaps by Bayer and Novartis Venture Fund led the A round with participation from Evotec SE, M Ventures, Alexandria Venture Investments and other undisclosed institutional investors.

"We are leveraging the deep immunology expertise of our co-founders to ask highly targeted questions about the innate and adaptive immune systems in tumors," said Hachigian.

She said the biotech's process starts with cells from human tumor samples, employs single-cell sequencing to identify all the proteins that are upregulated or downregulated in the cells, and then applies computational biology tools developed by Immunitas' scientific co-founders to identify the most relevant targets and screen out the background noise.

November 19: BioCentury Publications, Inc.: NIBR Shanghai exits discovery to refocus on clinical development, local collaborations

Novartis AG doesn't have a better solution than its multnational peers for how to fruitfully leverage discovery science in China. The pharma is closing down discovery operations at its Shanghai-based Novartis Institutes for BioMedical Research (NIBR) in favor of expanding clinical development and forming collaborations with domestic Chinese biotechs.

The move falls squarely in line with the trend among MNCs to use external rather than internal innovation to build its early-stage activities in China.

In an emailed statement to BioCentury, a Novartis AG (NYSE:NVS; SIX:NOVN) spokesperson said NIBR's Shanghai center "will exit early discovery research" and focus on "expanding the scale and scope of our early clinical development and later stage clinical trial operations" in order to support simultaneous near-term submissions in China. By 2023, the company plans to file 50 NDAs in the country.

She said NIBR's Shanghai team also plans to "increase connectivity with a growing community of life science innovators in China" and "seek strategic collaboration opportunities with early stage biotech companies in China through investments in local venture capital firms."

As innovative domestic biotechs have emerged to capitalize on regulatory changes from China's NMPA and abundant domestic venture capital, multinational pharmas have struggled to retain R&D talent in China. In the last two years, several pharmas including Johnson & Johnson (NYSE:JNJ), Merck KGaA (Xetra:MRK), Pfizer Inc. (NYSE:PFE) and AstraZeneca plc (LSE:AZN; NYSE:AZN) have opened innovation hubs in China in order to create ties with local companies and build R&D activities focused on external innovation (see "Major Multinationals Open, Close, Open R&D Centers in China").

While the restructuring will impact 150 personnel working in early discovery, the Shanghai site will continue to house more than 1,000 staffers, and NIBR plans to add 340 jobs in its global drug development and commercial divisions in China by 2023.

"We will transition research programs that are aligned with our portfolio to other NIBR sites and look to out-license or retire others that are not a strategic fit," said the Novartis spokesperson.

Although cybersecurity and IP reforms such as patent linkage and data exclusivity remain challenges for innovative drug developers in China, Novartis said NIBR's pivot to clinical development was not due to concerns over IP risks (see "China and the U.S.

November 15: BioCentury Publications, Inc.: Sarepta partners with StrideBio to enable viral gene therapy re-dosing

To enable repeat dosing of adeno-associated virus gene therapies, Sarepta is partnering with StrideBio to use the latter's capsid technology in up to eight CNS and neuromuscular programs.

The immunogenicity of viral antigens limits the number of times a patient may receive any given gene therapy. StrideBio, which raised a $15.7 million series A in June 2018, is designing AAV capsids that can evade neutralizing antibodies (see "AAV Plasmid Company StrideBio Raises $15.7M Series A").

StrideBio exclusively licensed its technology to Sarepta Therapeutics Inc. (NASDAQ:SRPT) for gene therapies targeting MECP2 for Rett syndrome, Nav1.1 for Dravet syndrome, UBE3A for Angelman syndrome and NPC1 for Niemann-Pick disease. StrideBio will receive $48 million up front in cash and Sarepta stock for the four programs and is eligible for undisclosed milestones and royalties.

StrideBio will conduct preclinical R&D and work with Sarepta in early clinical development. Sarepta is responsible for late-stage development and commercialization.

Sarepta also gained the option to exclusively license StrideBio's technology for four more programs. If exercised, StrideBio would gain up to $42.5 million up front plus undisclosed milestones, and has an option to co-commercialize and co-develop one of the four additional programs.

Sarepta will invest in StrideBio's next financing round. Both companies declined to disclose additional financial details.

For Sarepta, the collaboration complements several new gene therapy programs outside muscular dystrophy that the company has disclosed over the past 15 months. These include partnerships with the University of Massachusetts for Rett syndrome and the University of Florida for cardiomyopathy and multiple sclerosis; a deal with Lysogene S.A. (Euronext:LYS) for mucopolysaccharidosis Type IIIa; and a collaboration with Lacerta Therapeutics Inc.

November 15: BioCentury Publications, Inc.: Minerva on the verge in neuropsychiatry with two compounds close to verdict

Minerva Neurosciences is on the precipice of two clinical readouts that could determine whether its founding bet on a pair of neuropsychiatry compounds from Mitsubishi Tanabe has panned out. Neither molecule represents a shift from the long tradition of targeting neurotransmitter systems, but each has a differentiated target profile that could address unserved patients.

It's also well-positioned to move a third candidate into Phase III, this time with a program that takes the opposite tack, targeting a validated mechanism and differentiating based on selectivity.

Minerva Neurosciences Inc. (NASDAQ:NERV) was founded in 2013 with licenses to two compounds from Mitsubishi Tanabe Pharma Corp. (TOKYO:4508) and funding from Index Ventures (now Medicxi). The company went public in 2014 and has a market cap of $202.5 million.

Chairman and CEO Remy Luthringer, who was an adviser to Mitsubishi and remains an adviser to Medicxi, saw opportunity for both molecules to treat symptoms left behind by marketed agents.

"We've had antipsychotics for 60 years. The percentage of patients with a job hasn't changed," said Luthringer.

Rather than aiming to treat the positive symptoms of schizophrenia, Minerva is focusing on the negative symptoms. Its lead compound roluperidone, according to Luthringer, would become the first molecule specifically for negative

Source: Company Website

November 14: BioCentury Publications, Inc.: Management tracks: Longtime Abbott CEO resigning; plus Otsuka, Bavarian Nordic, Progenics and Rakuten

Miles White will step down as CEO of Abbott Laboratories (NYSE:ABT) on March 31, after 21 years in the role while remaining executive chairman. President and COO Robert Ford, who joined the company in 1996, will become CEO and has been elected to the board.

Otsuka Pharmaceutical Development & Commercialization Inc. President and CEO William Carson will retire, effective Jan. 1, and become its chairman. A spokesperson for company told BioCentury it has no plans at this time to appoint his successor. OPDC is a subsidiary of Otsuka Pharmaceutical Co. Ltd., itself a subsidiary of Otsuka Holdings Co. Ltd. (Tokyo:4578).

Infectious disease and cancer company Bavarian Nordic A/S (CSE:BAVA; Pink:BVNRY) hired Jean-Christophe May was EVP and CCO, effective January 2020. He was VP and global vaccines commercialization leader at GlaxoSmithKline plc (LSE:GSK; NYSE:GSK).

Cancer company Progenics Pharmaceuticals Inc. (NASDAQ:PGNX) disclosed in an SEC filing that CEO Mark Baker resigned. CFO Patrick Fabbio will assume Baker's responsibilities until the board names an interim or permanent CEO. Last month, Lantheus Holdings Inc. (NASDAQ:LNTH) proposed to acquire the company, which has been under pressure from activist investor Velan Capital (see "Lantheus Enters Therapeutics with Progenics Takeout").

Cancer precision therapeutics company Rakuten Medical Inc. hired Brenton Keath as CFO and Dana Johnson as general counsel and appointed David Chao and Kentaro Hyakuno to the board. Keath was head of finance at Rakuten Commerce; Johnson was VP, head of legal affairs and corporate compliance at Gritstone Oncology Inc. (NASDAQ:GRTS); Chao is co-founder and general partner of DCM Ventures; and Hyakuno is COO and a group EVP at Rakuten Inc.

Source: Company Website

November 13: BioCentury Publications, Inc.: Three late-stage wins shift the clinical landscape in SLE

A trio of companies reported positive data from late-stage systemic lupus erythematosis trials, raising hopes that new treatments are on the horizon for an indication that has seen only one new therapy approved in the past 60 years.

At the 2019 American College of Rheumatology (ACR) / Association for Rheumatology Professionals (ARP) annual meeting this week, SLE candidates from AstraZeneca plc (LSE:AZN; NYSE:AZN), Idorsia Ltd. (SIX:IDIA) and RemeGen Ltd., each with different mechanisms, had positive data in Phase II or III trials.

The data are encouraging to the lupus community and a welcome change from the spate of recent late-stage failures. Last year, seven compounds failed to meet primary endpoints in Phase II or III studies (see "Target Wide, Test Narrow in Lupus").

Progress has been made in treating other autoimmune diseases, but SLE has been particularly challenging because of inconsistencies in standard of care and high placebo effect, which complicate trial design.

The three companies behind this week's positive readouts took varied strategies to overcome those hurdles, including endpoint selection and focusing on a proven mechanism.

AstraZeneca presented detailed results from its Phase III TULIP 2 trial for anifrolumab after announcing positive top-line results in August. SVP Late Stage R&D Richard Marshall told BioCentury that AZ aims to submit a BLA for the mAb against the type I interferon receptor 2H20.

After the mAb missed the primary endpoint based on SLE Responder Index 4 (SRI4) in the Phase III TULIP 1 trial last year, the pharma amended the TULIP 2 protocol to base its primary endpoint on another widely used composite score, British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) (see "New Endpoint Key to Phase III Success for Anifrolumab").

In the randomized study of 365 patients receiving anifrolumab or placebo on top of SOC, 47.8% of patients given an IV infusion of 300 mg of the mAb every four weeks showed a reduction in disease activity as measured by BICLA at 52 weeks, compared with 31.5% of patients given placebo.

November 06: BioCentury Publications, Inc.: Sandoz to launch third Neulasta biosimilar

Sandoz has received FDA approval for Ziextenzo pegfilgrastim-bmez and plans to price it at a monthly wholesale acquisition cost of $3,925, a 37% discount to the WAC for Amgen neutropenia drug Neulasta.

The entry of Ziextenzo sets the stage for competition among three biosimilars for the U.S. market for Neulasta pegfilgrastim. Sandoz, a unit of Novartis AG (NYSE:NVS; SIX:NOVN), plans to launch the drug this year.

Prior to Ziextenzo's launch, biosimilar competition had already cut into Neulasta's market share and forced Amgen Inc. (NASDAQ:AMGN) to cut pricing. Amgen reported a 32% year-over-year decline in Neulasta sales in 3Q19, with a 31% decline in the U.S.

FDA approved Udenyca pegfilgrastim-cbqv from Coherus Biosciences Inc. (NASDAQ:CHRS) in November 2018, and in June 2018 it approved Fulphila pegfilgrastim-jmdb from Mylan N.V. (NASDAQ:MYL) and Biocon Ltd. (NSE:BIOCON; BSE:BIOCON).

Fulphila and Udenyca launched at discounts of about 33% to Neulasta's WAC.

Udenyca had captured about 13% of the U.S. Neulasta market at the end of June, Coherus Chairman, President and CEO Dennis Lanfear reported on an August earnings call. He said net sales in 1H19 were about $120 million.

Mylan did not break out sales of its biosimilar.

Source: Company Website

November 06: BioCentury Publications, Inc.: Allogene sets sights on iPS cell-based allogeneic therapies via Notch partnership

Allogene has staked its claim in iPS cell-based allogeneic therapies by partnering with Notch to develop T cell and NK cell therapies to treat non-Hodgkin lymphoma, leukemia and multiple myeloma.

Moving beyond healthy donor cells to use iPS cells for allogeneic therapies is "what we see as the evolution of the field and the future of cell therapy," Allogene Therapeutics Inc. (NASDAQ:ALLO) President and CEO David Chang told BioCentury. "This is a space we have to have a play in."

Notch Therapeutics Inc. will be responsible for preclinical research while Allogene will be in charge of clinical development. Chang estimated the partners will have an IND-ready product in a couple of years.

Allogene will have exclusive, worldwide rights to any resulting products and gains a 25% equity stake in Notch. The company will also have a seat on Notch's board. Notch will receive $10 million up front and is eligible for $294.3 million in milestones.

Helmed by former executives of CAR T company Kite Pharma Inc., Allogene has a pipeline of off-the-shelf allogeneic CAR T products spun out from Pfizer Inc. (NYSE:PFE). The company, which launched in November 2017, had amassed $420 million in private funding before raising $324 million in an October 2018 IPO -- the biggest of that year (see "Allogeneic CARs on the Horizon").

iPS cells can be clonally expanded after genetic engineering, leading to a more homogeneous allogeneic therapy than one based on donor cells, which are edited in bulk, Chang said. Potential edits include removing risks of both graft-versus-host and host-versus-graft diseases, as well as increasing the cells' potency.

While standard iPS cell platforms require support cells to induce differentiation -- which can limit scaleability -- Notch's Engineered Thymic Niche (ETN) platform relies on synthetic reagents instead.

Chang said he isn't aware of another company using an ETN platform, which he cited as one of Notch's main draws, along with the company's co-founders, who are the pioneers of iPS cell differentiation.

Notch was formed in 2018 to develop research from the labs of Juan Carlos ZuNiga-Pflucker and Peter Zandstra.

November 02: BioCentury Publications, Inc.: Foresite launches incubator aimed at meshing data science with healthcare

With its newly launched incubator, Foresite will provide its companies access to a holistic data set and analytical tools to explore how data can drive the development of personalized medicine across broad diseases.

The incubator, Foresite Labs, is led by Foresite Capital Managing Director Vik Bajaj. Before joining the firm, he was CSO of Grail Inc. and co-founded and served as CSO of Verily Life Sciences LLC.

Bajaj told BioCentury that while at Grail, he met Foresite founder and CEO Jim Tananbaum. He left Grail to join the firm in 2017.

"We both came to the conclusion that healthcare is in the middle of a transformation driven by the economic reality that we're spending an increasing fraction of our GDP on healthcare, but the outcomes that we're achieving are in decline," he said.

Despite the prevalence of data and the availability of computational tools to analyze human biology and diseases, the healthcare industry has been slower than other industries to embrace them, Bajaj said, leading him to ask why.

What he found were high barriers for entrepreneurs to enter the data science and healthcare space. Foresite Labs' sole focus, Bajaj said, is to remove those barriers: having access to the right data, having the right analytical tools, and forming the right team to know how to piece data and life sciences together.

Existing data sets can be incomplete or biased, Bajaj said, but generating completely new data can be burdensome for a new company.

Foresite Labs will provide its companies access to a platform "that amalgamates all of the existing public data in a way that allows for a unified analysis across all these data sets, as well as the prospective generation of new proprietary data sets," Bajaj said.

The incubator will also provide statistical genetics and causal inference tools so the companies can draw conclusions from the data.

Bajaj said Foresite Labs is interested in companies working on developing personalized medicines for subsets of broad, complex diseases.

October 31: BioCentury Publications, Inc.: Polaris' Pandion partners with Astellas on diabetes bispecifics

Formed by Polaris Partners to develop therapies that restore immune homeostasis at the site of disease, Pandion has entered its first large pharma partnership under which it will work with Astellas to discover and develop bispecific antibodies to treat pancreatic autoimmune diseases, including Type I diabetes.

Pandion Therapeutics Inc. will initially receive up to $45 million, which comprises an upfront payment and preclinical milestones. The company is also eligible for about $750 million in development and commercial milestones, plus royalties.

For the five-year collaboration, Pandion will be responsible for the discovery and design of up to three bispecifics, while Astellas Pharma Inc. (Tokyo:4503) will be in charge of preclinical and clinical development, as well as commercialization.

Pandion CEO Rahul Kakkar told BioCentury that while there are effective antibody therapies for some autoimmune diseases, others, like Type I diabetes or inflammatory bowel syndrome, don't get the same durable disease control. That's why the company thinks "the next frontier in autoimmune diseases will go beyond knocking down signals to balancing the immune system," he said.

Pandion's bispecifics pair an immune effector to rebalance T cell activity with a "tissue-tethering" antibody that binds a specific protein in the desired tissue.

Kakkar said the collaboration will at first focus on using a PD-1 agonist on the immune modulation side, which stimulates an anti-effective signal to tamp down on the activity of invading T cells that destroy pancreatic tissue.

The partners will also explore IL-2 mutein, which is the other side of the T cell coin, Kakkar said. The protein expands Tregs to induce immunosuppression.

Source: Company Website

October 23: BioCentury Publications, Inc.: Oct. 22 Company Quick Takes: Zealand acquires Encycle; plus Novartis, Vitalis, Roche-Spark

Zealand expands peptide platform with Encycle acquisition Zealand Pharma A/S (CSE:ZEAL; NASDAQ:ZEAL) said it will expand its peptide therapeutics platform through the acquisition of Encycle Therapeutics Inc. In return for all its outstanding shares, access to its screening library of peptide macrocycles, IP and lead asset ET3764, Encycle will be eligible to receive up to $80 million in earn-out payments linked to ET3764 milestones plus single-digit royalties on the oral peptide inhibitor of integrin α4β7. ET3764 is in preclinical testing for inflammatory bowel disease.

Novartis plans label expansion for Entresto after Phase III miss Novartis AG (NYSE:NVS; SIX:NOVN) said it will seek a label expansion for Entresto sacubitril/valsartan to treat heart failure in patients with preserved ejection fraction in 4Q19. Entresto is marketed to treat chronic heart failure patients with reduced ejection fraction and pediatric heart failure in patients with left ventricular systolic dysfunction. In July, the drug missed its primary endpoint of reducing cardiovascular death and total heart failure hospitalizations in the Phase III PARAGON-HF trial in patients with preserved ejection fraction. However, June FDA guidance said that heart failure therapies do not need to show a mortality benefit or reduction in hospitalizations for approval (see "Entresto's Heart Failure Miss. May Not Signal The End").

Vitalis plans pivotal study for MS therapy Vitalis LLC emerged from stealth to announce plans to start a pivotal trial in 2020 for lead candidate VTS-72 for relapsing-remitting multiple sclerosis in patients experiencing flushing side effects on dimethyl fumarate. The company will submit an NDA to FDA under the 505(b)(2) pathway as early as 2020. The company said the formulation of aspirin and fumarate reduced flush vs.

October 19: BioCentury Publications, Inc.: Biotech industry urges CFIUS to scale back regulations

Biopharma companies and investors are calling on the Treasury Department to scale back plans to restrict foreign investment in U.S.companies that collect genetic information.

About 60 comments to Treasury on the regulations, which implement the Foreign Investment Risk Review Modernization Act of 2018 (FIRRMA), were recorded on as of Oct. 18, a day after the deadline for comments.

Under the regulations, companies collecting or maintaining sensitive personal data, including health data and genetic information would be subject to CFIUS review.

The definition of sensitive data is "unnecessarily broad to address specific national security concerns," BIO said in its comments. The trade group warned that the regulations could weaken the U.S. biotech industry by diverting investment to other countries and providing an incentive for U.S. businesses to relocate overseas.

While the committee's new jurisdiction over sensitive data would be limited to companies that collect personal data on more than 1 million U.S. individuals, companies that collect genetic information would have no minimum threshold (see "CFIUS's Genetic Data Criteria Could Ensnare More Biotech Investments").

BIO, the National Venture Capital Association (NVCA) and the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) were among those that proposed that CFIUS should limit the definition of genetic information to unique U.S. citizen data that could not be obtained from foreign sources and that genetic data de-identified through aggregation or anonymization should not be considered sensitive. NVCA added that only whole-genome genetic information should be considered sensitive, not information from individual genetic tests.

In an Oct. 4 commentary, BioCentury's Editorial Board said, "The definition would blanket a huge portion of clinical trials today, and the vast majority of studies that will be conducted in the future.

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