Hori­zon snaps up rare dis­ease drugs, pipeline, in $800M Rap­tor buy­out

Hori­zon Phar­ma has bagged Rap­tor Phar­ma­ceu­ti­cals $RPTP and a pair of rare dis­ease drugs on the mar­ket — along with a mul­ti-drug pipeline — in an $800 mil­lion cash deal. Hori­zon is pay­ing $9 a share for Rap­tor, which al­so has been pur­su­ing a trou­bled de­vel­op­ment path for a new drug for Hunt­ing­ton’s drug, now in a piv­otal tri­al.

Hori­zon $HZNP is look­ing for some quick re­turns on Rap­tor’s two ap­proved drugs, Pro­cys­bi and Quin­sair, used to treat a rare meta­bol­ic dis­or­der called nephro­path­ic cysti­nosis and chron­ic pul­monary in­fec­tions due to Pseudomonas aerug­i­nosa in adult pa­tients with cys­tic fi­bro­sis. Pro­cys­bi has U.S. and Eu­ro­pean ap­provals, while Quin­sair is OK’s in Eu­rope and Cana­da.

The com­pa­ny trum­pet­ed the move for beef­ing up its or­phan drug port­fo­lio, which will now in­clude 6 drugs for rare dis­eases. It’s al­so chuffed that it can now ex­tend its op­er­a­tions in­to Eu­rope.

That news sat well with in­vestors, who bid up Hori­zon’s shares by 5% Mon­day morn­ing.

Rap­tor’s shares closed at $7.45 on Fri­day af­ter climb­ing back from a $3.32 low in Jan­u­ary. The biotech’s rev­enue hit a quar­ter­ly high of $32 mil­lion in Q2. That’s not block­buster ter­ri­to­ry by any means, but it al­lows Hori­zon an op­por­tu­ni­ty to grow the com­pa­ny while ex­pand­ing in a rare dis­ease field that has at­tract­ed wide­spread in­ter­est in bio­phar­ma in re­cent years.

A few weeks ago, Rap­tor was the sub­ject of con­sid­er­able mar­ket chat­ter from Bloomberg and oth­ers, which cit­ed Retrophin and Shire as like­ly ac­quir­ers. Those re­ports were right about the in­ter­est, even if they failed to iden­ti­fy the buy­er. But it is note­wor­thy that de­spite the buzz, Rap­tor did not fetch a big pre­mi­um.

Rap­tor’s top drug in the pipeline is RP103, which failed a Phase II Hunt­ing­ton’s study in late 2015 but was shoved in­to a piv­otal tri­al in any case, as in­ves­ti­ga­tors say they were per­suad­ed by enough clin­i­cal­ly sig­nif­i­cant da­ta to push ahead. The set­back on RP103 fol­lowed a com­plete fail­ure on NASH at the biotech. Rap­tor al­so has drugs in the clin­ic for cys­tic fi­bro­sis, bronchiec­ta­sis and non tubu­lar my­obac­te­ria.

“The pro­posed ac­qui­si­tion of Rap­tor fur­thers our com­mit­ment to help­ing peo­ple with rare dis­eases and is a sig­nif­i­cant step in ad­vanc­ing our strat­e­gy to ex­pand our rare dis­ease busi­ness,” said Tim­o­thy P. Wal­bert, chair­man, pres­i­dent and chief ex­ec­u­tive of­fi­cer, Hori­zon Phar­ma plc.  “Along with the po­ten­tial for ac­cel­er­at­ed rev­enue growth, the ad­di­tion of Rap­tor strength­ens our U.S. or­phan busi­ness and pro­vides a plat­form to ex­pand our or­phan busi­ness in Eu­rope and oth­er key in­ter­na­tion­al mar­kets.  We look for­ward to work­ing with new pa­tient com­mu­ni­ties and build­ing on the suc­cess of the Rap­tor team.”

Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

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Jeff Albers, Blueprint CEO

Di­ag­nos­tic champ Roche buys its way in­to the RET ti­tle fight with Eli Lil­ly, pay­ing $775M in cash to Blue­print

When Roche spelled out its original $1 billion deal — $45 million of that upfront — with Blueprint to discover targeted therapies against immunokinases, the biotech partner’s RET program was still preclinical. Four years later, pralsetinib is on the cusp of potential approval and the Swiss pharma giant is putting in much more to get in on the commercial game.

Roche gains rights to co-develop and co-commercialize the drug, with sole marketing responsibility for places outside the US and China (where CStone has staked its claim).

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Cy­to­ki­net­ics nabs Chi­nese part­ner, up to $450M as CV be­comes com­pa­ny fo­cus

Cytokinetics’ ALS programs may have seen better days, but its cardiovascular division has attracted well-backed partners, including Amgen and Royalty Pharma, who, in 2017 paid $90 million in exchange for a 4.5% royalty on their lead heart drug.

Now a Chinese drugmaker and a major investor are signing onto their second cardiovascular candidate, an experimental drug known as CK-274 and designed to treat hypertrophic cardiomyopathies — genetic conditions that cause heart muscles to become abnormally and potentially dangerously thick. Ji Xing Pharmaceuticals will pay Cytokinetics $25 million upfront for the rights to commercialize the drug in China and certain neighboring regions, along with $200 million in potential milestones.

President Trump (left) and NIAID chief Anthony Fauci in the White House press room, April 22, 2020 (Michael Reynolds/Sipa via AP Images)

White House tries to dis­cred­it An­tho­ny Fau­ci — could he be on his way out?

For two months in late winter and early spring, Anthony Fauci and President Trump stood in uneasy co-existence at White House briefings — an unlikely truce between an infectious disease official who had helped combat AIDS and Ebola and a president who repeatedly denied the danger of a virus that would go on to kill 100,000 Americans, repeatedly rejected masks and certain social distancing efforts, and promoted a drug with little scientific basis.

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SPACs are rid­ing on the biotech IPO boom

Billionaire hedge fund manager Bill Ackman’s push to raise $4 billion for his blank check company, Pershing Square Tontine Holdings, is casting a spotlight on the SPACs. And amid a historic SPAC boom, biotechs are placing several milestones on what some observers say is shaping up to be a third major track — besides IPO and M&A — to go public.

“SPACs were approximately 3% of the IPO market back in 2014, now they are almost 35% of all new listings,” Jay Heller, the Nasdaq’s head of capital markets, told Endpoints News.

Stéphane Bancel, Moderna CEO (Andrew Harnik/AP Images)

A top an­a­lyst turns the spot­light on Mod­er­na, fu­el­ing a fast-and-fu­ri­ous Street race over the fu­ture of mR­NA

Bioregnum Opinion Column by John Carroll

Four months ago, one of the favorite talking points on the biopharma social media wave length was whether Moderna shares $MRNA were priced right or were wildly inflated.

After all, said the naysayers, the company had never actually pushed a treatment to an approval. Did messenger RNA really work, coding cells to make a drug or a vaccine? And how about all that chatter about how ‘secretive’ they are, or were?

Now, as CEO Stéphane Bancel and the top execs push the company to the forefront of a frantic race to develop the first vaccine to fight against the reignited wildfire spread of Covid-19, all those questions have been magnified — along with the stock price.

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En­gi­neer­ing an on/off switch for CAR-T out of yeast and Juras­sic Park

Almost as soon as CAR-T emerged in the mid-2010s as a near-cure for some cancers, so did a question: How do you give this without risking killing patients?

At the time, James Patterson was wrapping an MD-PhD at a yeast lab at London’s Francis Crick Institute. Yeast may seem an unlikely place to find a fix for cancer therapy, but reading through other researchers’ solutions to CAR-T’s toxicity, Patterson wondered if a method long used by yeast biologists called auxotrophy might be useful. You genetically modify cells to make them dependent on a particular nutrient. Then you can make them live or die — proliferate or deplete — by giving or taking away that nutrient.

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FDA re­jects Ver­ri­ca’s skin warts pitch, a month af­ter flag­ging ap­pli­ca­tion ‘de­fi­cien­cies’

Two years ago, Verrica Pharma landed a $75 million IPO on the promise they could develop the first ever-FDA approved drug for molluscum contagiosum, an infection that causes warts. Now that won’t happen, at least not this summer.

The FDA today rejected Verrica’s application to have their lead topical drug VP-102 approved for molluscum contagiosum. The news likely came as little surprise to Verrica or its investors; the biotech disclosed in late June that the FDA sent a letter saying “deficiencies” in their application precluded a discussion around labeling or post-marketing commitments.

Andrew Allen, Gritstone Oncology CEO

A neoanti­gen pi­o­neer says its tech is work­ing great. So what wrecked the share price?

Gritstone Oncology was one of the original neoantigen upstarts, raising cash and planning to disrupt the immuno-oncology field with a bold new approach to fighting cancer with a new brand of vaccines.

On Monday, the crew in charge ran out a full display of what they’ve been seeing in a Phase I study. And everything seems to be working perfectly with one big exception: It didn’t significantly shrink tumors, let alone eradicate them.

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