Biotech M&A is back. Ipsen set to fork out up to $1.3B for rare dis­ease spe­cial­ist Clemen­tia

Merg­er Mon­day is back in full swing. Along with Roche buy­ing Spark Ther­a­peu­tics $ONCE, Parisian biotech Ipsen (Eu­ronext: $IPN) is beef­ing up its rare dis­ease ar­se­nal by ac­quir­ing Cana­da’s Clemen­tia Phar­ma­ceu­ti­cals, less than two years af­ter the Mon­tre­al-based drug de­vel­op­er’s $120 mil­lion IPO.

Un­der the terms of the deal, Ipsen will pay $25 per Clemen­tia share — a rough­ly 68% pre­mi­um to its Fri­day close — for a to­tal of $1.04 bil­lion, in ad­di­tion to an­oth­er $6/share if Clemen­tia’s lead ex­per­i­men­tal drug, palo­varotene, can be used to treat a rare bone dis­ease called mul­ti­ple os­teo­chon­dro­mas (MO), which could trans­late to an­oth­er $263 mil­lion.

Clemen­tia’s shares $CM­TA sky­rock­et­ed 73.5% in pre­mar­ket trad­ing.

Palo­varotene has been re­pur­posed by Clemen­tia for a pair of rare dis­eases, af­ter Roche failed to show the retinoic acid re­cep­tor gam­ma (RARγ) se­lec­tive ag­o­nist could work against em­phy­se­ma, a form of chron­ic ob­struc­tive pul­monary dis­ease (COPD).

The Swiss drug­mak­er had plans to shelve the drug, but re­searchers un­der­scored the po­ten­tial of the class of drugs in pre­vent­ing bone for­ma­tion and fi­bro­sis (retinoic acid sig­nal­ing is be­lieved to play a role in mus­cle cell gen­er­a­tion). Clemen­tia took the drug off Roche’s shelf, for two bone dis­or­ders: fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP) and MO.

A mar­ket­ing ap­pli­ca­tion for the drug to treat episod­ic flare-ups as­so­ci­at­ed with FOP is ex­pect­ed to be sub­mit­ted to the FDA in the sec­ond half of this year — and if ap­proved the drug is set for launch mid-2020. For the FOP in­di­ca­tion, the FDA has laid out a red car­pet for the drug, with or­phan drug des­ig­na­tion, break­through ther­a­py sta­tus, fast track sta­tus and rare pe­di­atric dis­ease des­ig­na­tion, so the odds are in its fa­vor for an ac­cel­er­at­ed re­view, if the NDA is ac­cept­ed.

In an ini­ti­a­tion note pub­lished last month Baird an­a­lysts cheered the po­ten­tial of the drug: “(With) strik­ing ef­fi­ca­cy and a po­ten­tial ac­cel­er­at­ed NDA fil­ing in an ul­tra-rare con­di­tion, we have con­vic­tion that pa­lo can dom­i­nate the FOP ther­a­peu­tic land­scape.”

A Phase III study test­ing the treat­ment as a chron­ic dos­ing reg­i­men for FOP, a Phase II tri­al for MO, and a Phase I tri­al for dry eye dis­ease are al­so on­go­ing.

In a note on Mon­day, Leerink’s Joseph Schwartz said the on­go­ing phase III study test­ing palo­varotene in FOP pa­tients could fur­ther demon­strate and sup­ple­ment its ef­fi­ca­cy in the pa­tient pop­u­la­tion, es­ti­mat­ing peak sales of more than $400 mil­lion for the in­di­ca­tion by 2030.

Schwartz ex­pects in­ter­im da­ta from on­go­ing MO study in pe­di­atric pa­tients by the end of next year, and topline da­ta by 2021. On the ba­sis of a “sig­nif­i­cant­ly high­er pro­ject­ed preva­lence of MO”, he fore­cast peak sales of over $1 bil­lion by 2030.

The deal is ex­pect­ed to be con­sum­mat­ed in the sec­ond quar­ter, and will have a lim­it­ed di­lu­tive im­pact on Ipsen’s core op­er­at­ing mar­gin for 2019 and 2020, the com­pa­ny said on Mon­day.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; 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.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.