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.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.