Gene ther­a­py de­vel­op­er Or­chard prices IPO at $14 to raise $200M

UK gene ther­a­py de­vel­op­er Or­chard Ther­a­peu­tics $OR­TX priced its ini­tial pub­lic of­fer­ing at $14 (ADS) per share, and is of­fer­ing about 14.3 mil­lion shares, it said on Tues­day. Shares of the Lon­don-based start­up, which ac­quired the rights to GSK’s $GSK “bub­ble boy” dis­ease drug Strimvelis ear­li­er this year, are ex­pect­ed to com­mence trad­ing on the Nas­daq on Wednes­day. The of­fer­ing comes at a time when the rate of bio­phar­ma IPOs has hit a fever­ish pace,with more such IPOs in the first half of this year than all of 2017.

The gross pro­ceeds from Or­chard’s of­fer­ing —  in which JP Mor­gan, Gold­man Sachs and Cowen are act­ing as joint book-run­ning man­agers — are ex­pect­ed to be in the range of about $200 mil­lion.

Or­chard is de­vel­op­ing an ex vi­vo gene ther­a­py in which a pa­tient’s stem cells are ex­tract­ed and fixed out­side the body be­fore be­ing trans­plant­ed back in with an aim to treat a va­ri­ety of rare dis­eases. GSK in April trans­ferred the rights to its port­fo­lio of rare dis­ease gene ther­a­pies, in­clud­ing Strimvelis, to Or­chard in ex­change for a 19.9% stake af­ter Britain’s biggest drug­mak­er de­cid­ed the slate of drugs were too niche for its ar­se­nal of treat­ments.

Strimvelis was ap­proved in Eu­rope in 2016, and is an ex­pen­sive but cor­rec­tive treat­ment for adeno­sine deam­i­nase se­vere com­bined im­mun­od­e­fi­cien­cy (ADA-SCID), but has on­ly been used in a hand­ful of pa­tients.

Be­fore it an­nounced plans to go pub­lic, the young biotech, led by in­di­vid­u­als who worked on Strimvelis, raised $150 mil­lion in a Se­ries C fund­ing round in ad­di­tion to the $110 mil­lion raised in a Se­ries B round late last year.

The funds raised will be used to shep­herd the de­vel­op­ment of Or­chard’s three late-stage rare-dis­ease pro­grams: ADA-SCID, metachro­mat­ic leukody­s­tro­phy (MLD), and Wiskott–Aldrich syn­drome (WAS). The com­pa­ny is al­so de­vel­op­ing drugs for X-linked chron­ic gran­u­lo­ma­tous dis­ease (X-CGD) and be­ta-tha­lassemia, in ad­di­tion to a clutch of pre­clin­i­cal tar­gets.

The FDA ap­proval of Spark Ther­a­peu­tics’ $ONCE Lux­tur­na for an in­her­it­ed form of blind­ness late in 2017 brought the field of gene-ther­a­pies in­to the spot­light, with a grow­ing list of of drug­mak­ers cur­rent­ly look­ing for such one-time so­lu­tions for ge­net­ic dis­or­ders that are not prop­er­ly treat­ed by small mol­e­cule drugs and en­zyme re­place­ment ther­a­pies — treat­ments that are not de­signed to tar­get the root cause of the dis­or­ders.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Yanay Ofran (L) and Anat Binur (Ukko)

Leaps by Bay­er backs a pro­tein en­gi­neer­ing start­up tak­ing on Aim­mune — and Nestlé — in peanut al­ler­gy

Little capsules of peanut powder drove Nestlé’s $2.6 billion buyout of Aimmune. Now, with $40 million in new funding, a fledgling biotech is promising to bring a more sophisticated version of that protein therapy that can go much, much further.

Ukko’s goal is two-pronged — with the initial products spanning therapeutic and food — but it’s grounded in the same protein engineering platform, co-founder and CEO Anat Binur told Endpoints News.

As tar­get­ed ther­a­pies get ever more pre­cise, Deer­field un­veils $50M bet on an Har­vard pro­fes­sor's chem­istry in­sights

Behind the seemingly simple concept of targeted cancer therapies is the drug developer’s headache that the target is always changing. Each generation of kinase inhibitors may be ostensibly hitting the same oncogene, but in addition to blocking the wildtype oncogene, they must now also address the mutations that have developed along the way, spurring resistance to current drugs.

The more those target kinases evolve, too, the more they could resemble off-target kinases you don’t want to bind. So each iteration requires more selectivity — sometimes down to differences of a few atoms.

Vincent Sandanayaka (file photo)

UP­DAT­ED: Ex-MD An­der­son chief De­Pin­ho is help­ing launch an­oth­er biotech — and he's stick­ing with fa­mil­iar ground

Years after co-founding SINE-focused Karyopharm and stirring up controversy at MD Anderson, Ronald DePinho is helping uncloak a new biotech targeting solute carrier transporter proteins — and Karyopharm’s former head of chemistry is leading the charge.

Nirogy Therapeutics emerged from stealth mode on Tuesday with a $16.5 million Series A round and plans to hit the clinic by 2022. The financing should be enough to carry the startup’s lead program, a small molecule lactate transport inhibitor, through Phase I, CEO Vincent Sandanayaka said.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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