Leo Phar­ma lines up an op­tion to buy Bridge­Bio's Pelle­Pharm and its PhI­II rare skin can­cer drug

Der­ma­tol­ogy com­pa­ny Leo Phar­ma’s vo­ra­cious ap­petite for part­ner­ships has been fu­eled with yet an­oth­er deal, this time with Bridge­Bio’s Pelle­Pharm. The Dan­ish drug­mak­er has tak­en a mi­nor­i­ty stake and agreed to pro­vide R&D sup­port to the Bay Area com­pa­ny, which is set to test its rare skin can­cer drug, patideg­ib, in a Phase III tri­al.

The cen­tu­ry old drug­mak­er, which con­sum­mat­ed a deal to swal­low Ger­man gi­ant Bay­er’s pre­scrip­tion der­ma­tol­ogy busi­ness in the Unit­ed States in ad­di­tion to forg­ing pacts with Mor­phoSys and Zymeworks $ZYME ear­li­er this year, has ini­tial­ly com­mit­ted $70 mil­lion com­prised of eq­ui­ty fi­nanc­ing and fi­nan­cial R&D sup­port for a late-stage tri­al eval­u­at­ing Pelle­Pharm’s patideg­ib for Gor­lin syn­drome – a rare, ge­net­ic skin dis­ease that af­fects 10,000 in the Unit­ed States.

Sanuj Ravin­dran

Un­der the terms of the deal, Leo Phar­ma has the op­tion to buy Pelle­Pharm, with the lat­ter and its stock­hold­ers el­i­gi­ble to re­ceive up to an ad­di­tion­al $690 mil­lion in­clud­ing merg­er con­sid­er­a­tion and mile­stone pay­ments. If the drug is ap­proved, Pelle­Pharm stock­hold­ers are al­so el­i­gi­ble to re­ceive a dou­ble-dig­it roy­al­ty up­on achiev­ing cer­tain com­mer­cial mile­stones.

Patideg­ib is a top­i­cal gel for­mu­la­tion de­signed to treat Gor­lin syn­drome, char­ac­ter­ized by mu­ta­tions in the tu­mor sup­pres­sor gene en­cod­ing Patched1 (PTCH1), which acts as the pri­ma­ry in­hibitor of the hedge­hog sig­nal­ing path­way that is known to play a role in cel­lu­lar func­tion and tis­sue de­vel­op­ment; this cul­mi­nates in the for­ma­tion of hun­dreds of basal cell car­ci­no­mas (BCCs), es­pe­cial­ly on on the face and sun-ex­posed ar­eas. With no FDA-ap­proved treat­ments for the dis­ease, the stan­dard-of-care is surgery, with pa­tients suf­fer­ing from a se­vere form of Gor­lin syn­drome un­der­go­ing as many as 30 pro­ce­dures an­nu­al­ly.

Ervin Ep­stein

Pelle­Pharm is ex­pect­ed to be­gin re­cruit­ing pa­tients for the Phase III in the first quar­ter of next year, and the com­pa­ny ex­pects to have a da­ta read­out by the sec­ond half of 2020, chief Sanuj Ravin­dran told End­points News, adding that if all goes well, he an­tic­i­pates ap­proval by 2021. Mid-stage da­ta on the drug, which has se­cured the or­phan drug sta­tus and break­through ther­a­py des­ig­na­tion from the FDA, have shown promise in mit­i­gat­ing BCC tu­mors in pa­tients with Gor­lin syn­drome by block­ing the dis­ease at its source with­in the hedge­hog sig­nal­ing path­way. The gel for­mu­la­tion is be­ing de­vel­oped to elim­i­nate the need for surg­eries.

Pelle­Pharm’s founders de­vel­oped a top­i­cal gel for­mu­la­tion af­ter li­cens­ing the drug from In­fin­i­ty Phar­ma­ceu­ti­cals, to help al­le­vi­ate cer­tain sys­temic ad­verse events ob­served with oral hedge­hog in­hibitors, such as Roche’s $RHB­BY Erivedge. Al­though Ravin­dran did not dis­close pric­ing plans for the drug, he not­ed oral HHIs are priced at around $100,000 an­nu­al­ly.

Pelle­Pharm was con­ceived by Ervin Ep­stein, Jean Tang, and Phil Beachy in 2012, as they had worked with oral HHIs and saw the need to de­vel­op such treat­ments with a more tol­er­a­ble safe­ty pro­file. Michael Hen­der­son, who co-found­ed Pelle­Pharm while at med­ical school aged 22, is now in charge of hunt­ing down po­ten­tial as­sets for Bridge­Bio to snap up.

In 1908, phar­ma­cists Au­gust Kong­st­ed and An­ton An­tons bought the Leo Phar­ma­cy in Copen­hagen, Den­mark. From start­ing out in the base­ment of the phar­ma­cy, to­day Leo Phar­ma’s prod­ucts — main­ly with­in der­ma­tol­ogy and throm­bo­sis — are used in over 100 coun­tries.

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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EMA rec­om­mends re­vok­ing au­tho­riza­tion of No­var­tis' sick­le cell drug

The European Medicines Agency’s committee for medicinal products for human use (CHMP) on Friday recommended revoking the marketing authorization for Novartis’ treatment for a painful complication related to sickle cell, after a recent study did not confirm its clinical benefit.

CHMP’s review looked at results of the STAND study, finding that Adakveo (crizanlizumab) did not reduce the number of painful crises leading to a healthcare visit, and patients treated with Adakveo had slightly more painful crises on average, with a subsequent healthcare visit, over the first year of treatment, compared with those on placebo.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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